TAMPA, Fla.--(BUSINESS WIRE)--OSSIO Inc., a fast-growing medical technology company focused on evolving orthopedic surgery with bio-integrative, metal-free fixation implants, has selected Florida — specifically, the City of Palmetto in Manatee County, near Tampa — as the location of its new U.S headquarters, a move that is expected to create about 100 new jobs over the next five years.

The addition of OSSIO to our health technology sector will directly elevate Florida's life sciences job and innovation outcomes. - Florida Secretary of Commerce, J. Alex Kelly

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“Florida welcomes OSSIO as part of our effort to make the Sunshine State a global life sciences hub, with multiple centers of life sciences investment, research and development, and public-private collaboration,” said Florida Secretary of Commerce, J. Alex Kelly. “Aligned with the manufacturing vision of Gov. Ron DeSantis, Florida now ranks second for medical device and pharmaceutical manufacturing, and Florida supports these industries with the nation’s fifth largest investment in biotech research and development activities. The addition of OSSIO to our health technology sector will directly elevate Florida's life sciences job and innovation outcomes.”

OSSIO recently started converting 30,000 square feet of leased space in Palmetto into a state-of-the-art manufacturing operation and surgeon training center.

“Florida fosters a culture of innovation in health care and life sciences that OSSIO intends to strengthen by locating our U.S. headquarters here,” OSSIO CEO Brian Verrier said. “The Tampa region in particular offers medical technology companies like ours an attractive combination of technical talent and economic incentives to support sustainable growth.”

Recruiting set to start

OSSIO plans to begin recruiting for manufacturing technicians, clinical trainers and support staff in the fall and to start producing OSSIOfiber® implants at the new site in the spring.

“We are thrilled to have OSSIO join the Palmetto community,” the city’s mayor, Dan West, said. “Their presence reflects the ongoing growth and innovation in our city, and we look forward to supporting their success. Palmetto is a city that values strong partnerships, and OSSIO is a wonderful addition to our local business family.”

OSSIO HQ also promises to benefit the Tampa Medical & Research District, which encompasses several organizations associated with Tampa General Hospital and the University of South Florida.

“As the Tampa Medical & Research District develops,” explained Tampa General Hospital SVP Jennifer Crabtree, the organization’s newly appointed president, “I envision involving innovative life sciences companies with nearby operations like OSSIO in our work, which centers on making Florida’s southwest coast a center of excellence for health care innovation.”

In addition, OSSIO recently joined BioFlorida, the state’s trade association for the health care and medical technology industry.

“OSSIO represents exactly the kind of innovative company that strengthens Florida’s life sciences ecosystem,” said BioFlorida President and CEO Mark Glickman. “Their orthopedic implant technology is a true game-changer by offering patients a metal-free, more natural way to heal after surgery. We are proud to welcome OSSIO to BioFlorida and look forward to supporting the company’s growth and impact in advancing medical technology.”

About OSSIOfiber implants

Based on Intelligent Bone Regeneration Technology, OSSIOfiber implants combine unparalleled mechanical strength and natural healing in a bio-integrative, metal-free design. Their proprietary polymeric mineral fiber matrix enables rapid bone in-growth, regeneration and replacement — a new way for orthopedic surgeons to restore their patients’ stability and mobility while leaving nothing permanent behind.

OSSIOfiber implants represent the first credible alternative to metal, absorbable and allograft fixation devices. The most significant advance in orthopedic fixation technology in more than 100 years, this novel medical technology has the potential to address an increasingly wide array of surgical applications due to the proprietary “know-how” to manufacture limitless implant platforms. Looking ahead, the company intends to continue developing additional applications for OSSIOfiber implants in all major segments of orthopedics.

Notably for U.S. healthcare providers and payors, OSSIOfiber implants utilize existing reimbursement codes and surgical techniques. Their uptake continues to grow because of strong patient and surgeon preference to avoid the concerns and complications of metal fixation implants. Through September 2025, more than 60,000 OSSIOfiber implants have been used to treat orthopedic patients in the U.S.

About OSSIO Inc.

OSSIO is an orthopedics company committed to transforming the surgical experience for patients, physicians, providers and payors. The company’s vision is to provide the first credible replacement for metal implants in the multibillion-dollar global orthopedic fixation market with OSSIOfiber Intelligent Bone Regeneration Technology. Founded in 2014, the company’s vision is to provide the first credible replacement for metal implants in the multibillion-dollar global orthopedic fixation market with OSSIOfiber Intelligent Bone Regeneration Technology. OSSIO conducts product development in Caesarea, Israel, and commercial operations from Woburn, Mass. For more information about the company and OSSIOfiber implants, visit www.ossio.io.

Disclaimer: Forward-looking statements in this news release are based on estimates and assumptions of OSSIO management and are believed to be reasonable, though they are inherently uncertain and difficult to predict.


Contacts
Joe McGrath
Scope Strategic Communications
contactmcgrath@scopestrategic.com
+1-612-819-6421

Tampa, Florida, US / Gothenburg, Sweden – June 23, 2025

Decisionexus LLC is pleased to announce a new strategic agreement with Captario, a leading innovator in portfolio analysis for the pharmaceutical industry.

Founded and led by former senior executives from leading global pharma companies, Decisionexus brings a deep understanding of the complex realities faced by drug development organizations. With this agreement, Decisionexus will take a leading role in customer engagements, helping clients fully leverage Captario SUM®, an AI-powered platform for Strategic Uncertainty Management. By enabling real-time Monte Carlo simulations, AI- enhanced predictive modeling, and dynamic scenario planning, Captario SUM® allows users to make faster, smarter, and more transparent decisions across the entire drug development lifecycle.

This partnership brings together Captario’s advanced technology with Decisionexus’s deep expertise in decision analysis, stakeholder alignment, and strategic facilitation. Together, the firms will deliver tailored, high-impact engagements that accelerate value creation and improve outcomes for clients navigating complex, uncertain environments.

“We’re excited to collaborate with Captario and its world class platform to deliver even greater impact for clients,” said Terrence Karner, Principal at Decisionexus. “This agreement enables us to help organizations to drive strategic agility, reduce decision latency, and maximize portfolio value.”

For more information, please contact:

Terrence Karner
Principal, Decisionexus LLC terrence.karner@decisionexusllc.com
+1 847-338-9791

Magnus Ytterstad
Head of Customer Success & Portfolio Analytics, Captario magnus.ytterstad@captario.com
+1 551 755 1239

Adam Anderson 

Genome sequencing and the future of medicine in Florida | Column

Genome sequencing in infants saves lives and improves health outcomes.

Genome sequencing provides answers quickly, providing the opportunity for early access to treatment. [ FRANK AUGSTEIN | AP ]

Published April 11

The American Dream is built on opportunity — the opportunity to lead, innovate and change lives. Nowhere is that opportunity more profound than in medicine, where scientific breakthroughs can mean the difference between life and death. Florida stands on the precipice of becoming a national leader in medical research and innovation, and we are seizing this moment to transform health care for generations to come.

More than 30 million Americans suffer from rare diseases — many of them children who never get the chance to live full, healthy lives. But we have the power to change that. Advances in genetic testing offer the opportunity to diagnose and treat diseases at birth, yet too many families still endure years of uncertainty, misdiagnoses, and unnecessary suffering. Florida is stepping up to end that diagnostic odyssey.

We have a moral and economic imperative to act. That’s why, on National Rare Disease Day 2025, I announced HB 907, the Sunshine Genetics Act, at the Florida State University Institute for Pediatric Rare Diseases Symposium. This landmark legislation establishes a free, opt-in newborn genetic testing program capable of diagnosing over 600 disorders at birth. It also unites Florida’s top research institutions, specialty children’s hospitals, and biotech leaders in a first-of-its-kind consortium dedicated to advancing genetic research and finding cures. This is how we turn Florida into a hub for scientific discovery, where the best and brightest come to tackle the most demanding medical challenges of our time.

The science is clear: genome sequencing in infants saves lives and improves health outcomes. It takes, on average, four to five years and seven or more trips to specialists plus overnight states in the NICU to diagnose a child with a rare disease. Genome sequencing provides answers in a few short days, providing the opportunity for early access to treatment. Turning away from that opportunity would be inexcusable. Beyond the moral imperative, this is also a sound investment — genetic testing could save our health care system several $100s of millions annually by reducing the need for costly specialist visits and NICU stays. The future of medicine is coming to Florida, and we are ensuring our state leads the way.

Our commitment to this vision is already being recognized. At last year’s Florida State University Institute for Pediatric Rare Diseases Symposium, the FSU Center received a $5 million award, a testament to Florida’s dedication to medical research. This investment signals that we are ready to take things to the next level.

However, government alone cannot meet this challenge. We are building an ecosystem where the private sector and research institutions work hand-in-hand to push the boundaries of what’s possible. Regeneron Pharmaceuticals has invested $119.5 million in the Truveta Genome Project, leveraging AI and big data to advance genetic research. Baylor College of Medicine was awarded a $3.5 million grant to bring genomic medicine to NICUs in underserved communities. Florida must attract these types of investments. By fostering a cutting-edge research environment, we will bring the best minds, the most ambitious projects, and the most significant scientific advancements to our state.

Florida has a once-in-a-generation opportunity to lead. We are not just investing in science; we are investing in hope — the hope that no parent will have to endure years of unanswered questions, the hope that the next significant medical breakthrough will come from our labs, and the hope that Florida will stand at the forefront of the most transformative advancements in medicine in history.

To every scientist, investor, and innovator looking for the next frontier — Florida welcomes you. The future of medicine is being written right here, and together, we will change lives.

Rep. Adam Anderson represents parts of Pinellas County and serves as the Chairman of the Health Care Facilities & Systems Subcommittee.

Earlier this Week the District Court Granted Summary Judgement to Catalyst in Its Lawsuit Against the FDA and this FDA Action is the Result of that Decision

February 03, 2022 08:03 ET | Source: Catalyst Pharmaceuticals, Inc.

CORAL GABLES, Fla., Feb. 03, 2022 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare diseases, today reported that the District Court which heard Catalyst's Lawsuit against the FDA has entered summary judgment in favor of Catalyst. The District Court's order implements the prior decision of the U.S. Court of Appeals for the 11th Circuit, which ruled that the United States Food and Drug Administration's ("FDA") approval of Ruzurgi® (Jacobus Pharmaceutical Company, Inc.'s amifampridine product) for the treatment of pediatric patients with Lambert-Eaton myasthenic syndrome ("LEMS") violated Catalyst's exclusivity for FIRDAPSE® (amifampridine) Tablets 10 mg under the Orphan Drug Act. As a result of the District Court's order, the FDA marketing approval previously granted for Ruzurgi® is no longer valid.

Patrick J. McEnany, Catalyst's Chairman and CEO stated, "The entire Catalyst patient assistance team has been preparing for this likely outcome since the decision of the U.S. Circuit Court of Appeals for the 11th Circuit was reported. We have added additional care coordinators to our Catalyst Pathways® team, and our entire patient-focused team is ready to assist LEMS patients that are currently being treated with Ruzurgi® to provide a smooth, uninterrupted transition to FIRDAPSE."

Information for Prescribers and Patients on the options available to determine how to transition patients from Ruzurgi® are available at 1-833-422-8259 or www.yourcatalystpathways.com.

About Catalyst Pharmaceuticals

Catalyst Pharmaceuticals is a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare diseases. With exceptional patient focus, Catalyst is committed to developing a robust pipeline of cutting-edge, first- or best-in-class medicines for other rare diseases. Catalyst's New Drug Application for FIRDAPSE® (amifampridine) Tablets 10 mg for the treatment of adults with Lambert-Eaton myasthenic syndrome ("LEMS") was approved in 2018 by the U.S. Food & Drug Administration ("FDA"), and FIRDAPSE is commercially available in the United States as a treatment for adults with LEMS. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS.

Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Jacobus Pharmaceutical Company, Inc. will appeal the ruling of the U.S. Court of Appeals for the 11th Circuit to the U.S. Supreme Court, whether the U.S Supreme Court will agree to hear the appeal, and, if the U.S. Supreme Court agrees to hear the appeal, whether the U.S. Supreme Court will overturn the decision of the 11th Circuit, and (ii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2020 and Catalyst's other filings with the U.S. Securities and Exchange Commission ("SEC"), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

Source: Catalyst Pharmaceuticals, Inc.

Today, the U.S. Food and Drug Administration took action to expand the use of a booster dose for COVID-19 vaccines in eligible populations. The agency is amending the emergency use authorizations (EUA) for COVID-19 vaccines to allow for the use of a single booster dose as follows:

• The use of a single booster dose of the Moderna COVID-19 Vaccine that may be administered at least 6 months after completion of the primary series to individuals:
  • 65 years of age and older
  • 18 through 64 years of age at high risk of severe COVID-19
  • 18 through 64 years of age with frequent institutional or occupational exposure to SARS-CoV-2
• The use of a single booster dose of the Janssen (Johnson and Johnson) COVID-19 Vaccine may be administered at least 2 months after completion of the single-dose primary regimen to individuals 18 years of age and older.
• The use of each of the available COVID-19 vaccines as a heterologous (or “mix and match”) booster dose in eligible individuals following completion of primary vaccination with a different available COVID-19 vaccine.
• To clarify that a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine may be administered at least 6 months after completion of the primary series to individuals 18 through 64 years of age with frequent institutional or occupational exposure to SARS-CoV-2.

“Today’s actions demonstrate our commitment to public health in proactively fighting against the COVID-19 pandemic,” said Acting FDA Commissioner Janet Woodcock, M.D. “As the pandemic continues to impact the country, science has shown that vaccination continues to be the safest and most effective way to prevent COVID-19, including the most serious consequences of the disease, such as hospitalization and death. The available data suggest waning immunity in some populations who are fully vaccinated. The availability of these authorized boosters is important for continued protection against COVID-19 disease.”

“The amendments to the emergency use authorizations to include a single booster dose in eligible populations are based on the available data and information and follows the input from the members of our advisory committee who were supportive of the use of a booster dose of these vaccines in eligible populations,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We are also taking action today to include the use of mix and match boosters to address this public health need. We will work to accrue additional data as quickly as possible to further assess the benefits and risks of the use of booster doses in additional populations and plan to update the healthcare community and public with our determination in the coming weeks.”

Authorization of Moderna COVID-19 Vaccine Booster Dose

To support the authorization for emergency use of a single booster dose of the Moderna COVID-19 Vaccine, the FDA analyzed immune response data from 149 participants 18 years of age and older from the original clinical studies who received a booster dose at least 6 months after their second dose and compared it to the immune responses of 1,055 study participants after completing their two-dose series. The antibody response of the 149 participants against SARS-CoV-2 virus 29 days after a booster dose of the vaccine demonstrated a booster response. 

The FDA also evaluated an additional analysis from Moderna comparing the rates of COVID-19 accrued during the Delta variant surge during July and August 2021, which suggest that there is a waning of vaccine effectiveness over time.

Safety was evaluated in 171 participants 18 years of age and older who were followed for an average of approximately six months. The most commonly reported side effects by the clinical trial participants who received the booster dose of the vaccine were pain at the injection site, tiredness, headache, muscle and/or joint pain, chills, swollen lymph nodes in same arm as the injection, nausea and vomiting, and fever. Of note, swollen lymph nodes in the underarm were observed more frequently following the booster dose than after the primary two-dose series.

Ongoing analyses from the FDA and the Centers for Disease Control and Prevention (CDC) safety surveillance systems have identified increased risks of inflammatory heart conditions, myocarditis and pericarditis, following vaccination with the Moderna COVID-19 vaccine, particularly following the second dose. Typically, onset of symptoms has been a few days following vaccination. The observed risk is higher among males under 40 years of age, particularly males 18 through 24, than among females and older males. 

The Moderna COVID-19 single booster dose is half of the dose that is administered for a primary series dose and is administered at least six months after completion of a primary series of the vaccine. 

Authorization of Janssen (Johnson and Johnson) COVID-19 Vaccine Booster Dose

The authorization for emergency use of a single booster dose of the Janssen COVID-19 Vaccine is based on the FDA’s evaluation of immune response data in 39 participants from a clinical trial including 24 participants who were 18 through 55 years of age and 15 participants who were 65 years of age and older.  The study participants received a booster dose approximately 2 months after their first dose, and the results demonstrated a booster response.

Overall, approximately 9,000 clinical trial participants have received two doses of Janssen COVID-19 Vaccine administered at least two months apart and of these, approximately 2,700 have had at least two months of safety follow-up after the booster dose. Janssen’s safety analyses from these studies have not identified new safety concerns.

Earlier analyses from the FDA and CDC safety surveillance systems suggest an increased risk of a serious and rare type of blood clot in combination with low blood platelets following administration of the Janssen COVID-19 vaccine. This serious condition is called thrombocytopenia syndrome (TTS). People who developed TTS after receiving the vaccine had symptoms that began about one to two weeks after vaccination. Reporting of TTS has been highest in females ages 18 through 49 years. In addition, safety surveillance suggests an increased risk of a specific serious neurological disorder called Guillain Barré syndrome, within 42 days following receipt of the Janssen COVID-19 Vaccine.  

Authorization of “Mix and Match” Booster Dose

Today, the FDA is also authorizing the use of heterologous (or “mix and match”) booster dose for currently available (i.e., FDA-authorized or approved) COVID-19 vaccines. Following a presentation of clinical trial data from the National Institute of Allergy and Infectious Diseases, the Vaccines and Related Biological Products Advisory Committee’s discussion of information submitted for consideration, along with the agency’s evaluation of the available data, the FDA has determined that the known and potential benefits of the use of a single heterologous booster dose outweigh the known and potential risks of their use in eligible populations.

A single booster dose of any of the available COVID-19 vaccines may be administered as a heterologous booster dose following completion of primary vaccination with a different available COVID-19 vaccine. The eligible population(s) and dosing interval for a heterologous booster dose are the same as those authorized for a booster dose of the vaccine used for primary vaccination.

For example, Janssen COVID-19 Vaccine recipients 18 years of age and older may receive a single booster dose of Janssen COVID-19 Vaccine, Moderna COVID-19 Vaccine (half dose) or Pfizer-BioNTech COVID-19 Vaccine at least two months after receiving their Janssen COVID-19 Vaccine primary vaccination. 

In another example, Moderna COVID-19 Vaccine and Pfizer-BioNTech COVID-19 vaccine recipients falling into one of the authorized categories for boosters (65 years of age and older, 18 through 64 years of age at high-risk of severe COVID-19, and 18 through 64 years of age with frequent institutional or occupational exposure to SARS-CoV-2) may receive a booster dose of Moderna COVID-19 Vaccine (half dose), Pfizer-BioNTech COVID-19 Vaccine or Janssen COVID-19 Vaccine at least six months after completing their primary vaccination.

The agency recognizes that health care providers and COVID-19 vaccine recipients will have questions about booster doses. The individual fact sheets for each available vaccine provide relevant information for health care providers and the vaccine recipients. The agency encourages health care providers to also follow the recommendations that will be provided by the CDC following a meeting of their Advisory Committee on Immunization Practices and formal recommendations signed by the CDC director.

Related Information

###

For Immediate Release: Friday, September 24th, 2021
Contact: Media Relations
(404) 639-3286

Today, CDC Director Rochelle P. Walensky, M.D., M.P.H., endorsed the CDC Advisory Committee on Immunization Practices’ (ACIP) recommendation for a booster shot of the Pfizer-BioNTech COVID-19 vaccine in certain populations and also recommended a booster dose for those in high risk occupational and institutional settings. The Food and Drug Administration’s (FDA) authorization and CDC’s guidance for use are important steps forward as we work to stay ahead of the virus and keep Americans safe.

This updated interim guidance from CDC allows for millions of Americans who are at highest risk for COVID-19 to receive a Pfizer-BioNTech COVID-19 booster shot to help increase their protection.

CDC recommends:

• people 65 years and older and residents in long-term care settings should receive a booster shot of Pfizer-BioNTech’s COVID-19 vaccine at least 6 months after their Pfizer-BioNTech primary series,
• people aged 50–64 years with underlying medical conditions should receive a booster shot of Pfizer-BioNTech’s COVID-19 vaccine at least 6 months after their Pfizer-BioNTech primary series,
• people aged 18–49 years with underlying medical conditions may receive a booster shot of Pfizer-BioNTech’s COVID-19 vaccine at least 6 months after their Pfizer-BioNTech primary series, based on their individual benefits and risks, and
• people aged 18-64 years who are at increased risk for COVID-19 exposure and transmission because of occupational or institutional setting may receive a booster shot of Pfizer-BioNTech’s COVID-19 vaccine at least 6 months after their Pfizer-BioNTech primary series, based on their individual benefits and risks.

Many of the people who are now eligible to receive a booster shot received their initial vaccine early in the vaccination program and will benefit from additional protection. With the Delta variant’s dominance as the circulating strain and cases of COVID-19 increasing significantly across the United States, a booster shot will help strengthen protection against severe disease in those populations who are at high-risk for exposure to COVID-19 or the complications from severe disease.

CDC will continue to monitor the safety and effectiveness of COVID-19 vaccines to ensure appropriate recommendations to keep all Americans safe. We will also evaluate with similar urgency available data in the coming weeks to swiftly make additional recommendations for other populations or people who got the Moderna or Johnson & Johnson vaccines.

The following is attributable to Dr. Walensky:

As CDC Director, it is my job to recognize where our actions can have the greatest impact. At CDC, we are tasked with analyzing complex, often imperfect data to make concrete recommendations that optimize health. In a pandemic, even with uncertainty, we must take actions that we anticipate will do the greatest good.

I believe we can best serve the nation’s public health needs by providing booster doses for the elderly, those in long-term care facilities, people with underlying medical conditions, and for adults at high risk of disease from occupational and institutional exposures to COVID-19. This aligns with the FDA’s booster authorization and makes these groups eligible for a booster shot. Today, ACIP only reviewed data for the Pfizer-BioNTech vaccine. We will address, with the same sense of urgency, recommendations for the Moderna and J&J vaccines as soon as those data are available.

While today’s action was an initial step related to booster shots, it will not distract from our most important focus of primary vaccination in the United States and around the world. I want to thank ACIP for their thoughtful discussion and scientific deliberation on the current data which informed my recommendation.

###
U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES

CDC works 24/7 protecting America’s health, safety and security. Whether disease start at home or abroad, are curable or preventable, chronic or acute, or from human activity or deliberate attack, CDC responds to America’s most pressing health threats. CDC is headquartered in Atlanta and has experts located throughout the United States and the world.

Contacts

Lisa M. Wilson
In-Site Communications, Inc.
212-452-2793
lwilson@insitecony.com

Regulatory Oversight in Gene Therapy: How Far Have We Come?

July 21, 2021

Gene therapy has finally emerged as a therapeutic reality delivering meaningful benefits to human health.

G&L’s Vice President of Regulatory Affairs, Jessica Kokosinski, and Senior Director of Regulatory Affairs, Christine Piscitelli take an in-depth look at the journey of this revolutionary therapy over the last five decades.

Introduction

What was once viewed as a theoretical concept over 50 years ago, gene therapy has finally emerged as a therapeutic reality delivering meaningful benefits to human health. The U.S. Food and Drug Administration (FDA) defines gene therapy as a technique that “seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.”

To date, there are only four FDA approved gene therapy products that insert new genetic material into a patient’s cells. However, FDA reports that there are more than 900 active investigational new drug (IND) applications in this area, studying treatments for diseases such as cancer, genetic diseases, and infectious diseases [1].

The regulatory framework for the development and manufacturing of gene therapy products comprises reg­ulations and guidance documents, including the interpretation of these governing documents by regulators and sponsors as experience is gained in practice.

Today, gene therapy products are regulated by FDA’s Center for Biologics Evaluation and Research (CBER). FDA requires submission of an IND prior to initiating clinical studies, and an approved biologics license application (BLA) to market the product in the United States.

However, this regulatory framework did not exist when the potential to alter human genes first emerged in the 1960s. Since that time, regulators have ensured that the regulatory framework and oversight continually evolve to keep pace with each new scientific breakthrough and advancement in the field.

Yet, it has not always been a smooth journey to get to where we are today – advancing science and human health while accelerating the availability of safe and effective gene therapy products is a fine balance and can be fraught with obstacles.

Brief History of Gene Therapy and U.S. Regulatory Oversight

Although gene therapy is a seemingly new frontier for biotherapeutic products, its history dates to the earlier part of the last century. The field evolved out of two significant discoveries in the first half of the 1900s: Frederick Griffith’s discovery of the “transforming principle” while studying pneumococcal bacteria in 1928, followed by the Avery et al. discovery in 1944 that bacterial transformation occurred through DNA [2].

By 1961, the first DNA-mediated heritable genetic transformation was successfully demonstrated in mammalian cells by Waclaw Szybalski [3]. This study ultimately laid the groundwork for gene therapy as a potential strategy to correct defective DNA in human cells. Soon after, research into gene transfer and its potential for clinical application began to flourish.

The early 1970s brought the first direct human gene therapy study which unsuccessfully attempted to treat two pediatric subjects suffering from the genetic disorder hyperargininemia [2]. At the time, there was little regulatory oversight for human gene therapy studies. Members of the scientific community began expressing growing concern over safety, scientific, social, and ethical issues raised by human gene therapy.

Most notable was a 1972 opinion piece written by Theodore Friedmann and Richard Roblin. While Friedmann and Roblin believed in the potential of gene therapy to treat human genetic diseases and encouraged continued research in this field, they also advised the need for additional scientific understanding and regulation before allowing further clinical trials in humans [4].

By 1974, a gene therapy regulatory framework began to take shape in the United States. The National Institutes of Health (NIH) established the Recombinant DNA Advisory Committee (RAC) to bolster oversight of basic and clinical research involving recombinant DNA and later expanded its scope to include human gene transfer research.

Regulators and the scientific community continued to make significant strides in addressing ethical, legal, and social implications of gene therapy and improving patient protections over the next 25 years. Several layers of federal and local oversight and regulations were established during this time. Namely, the Institutional Review Board (IRB) system was established, RAC developed biosafety guidelines, NIH published research guidelines for recombinant DNA molecules and established Institutional Biosafety Committees (IBC), and the FDA began regulating gene therapy products and issued its first guidance document on cell and gene therapy [5]. FDA and NIH continued to foster innovations in the development of gene therapy products while also eliminating overlaps in oversight efforts.

By 1997, the FDA assumed sole authority to approve gene transfer protocols and gene therapy products, while NIH eliminated director approval of individual protocols and shifted the responsibilities of the RAC to an advisory role [5,6].

The first U.S. gene therapy trial with a therapeutic attempt in humans occurred in September 1990 at the NIH Clinical Center [2]. This marked the first gene therapy trial with FDA oversight. Throughout the 1990s the number of trials increased over time, although not steadily. By the end of the decade, a total of 484 gene therapy clinical trials had been approved, were ongoing or completed worldwide with the peak occurring in 1999 (117 trials) [7].

While the outcome of many of these trials were unsuccessful, the research looked promising as no major safety concerns had been raised. The potential for gene therapy to treat and cure some of the most debilitating diseases remained encouraging.

Unfortunately, the boom experienced in the 1990s would be marred by several tragic events that underscored the many uncertainties that existed at the time regarding safety, efficacy, and long-term effects of human gene therapy.

The death of 18-year-old Jesse Gelsinger, who died of a massive immune response during a gene therapy safety trial in September 1999, accentuated the risks associated with viral vectors [2]. The risk of insertional mutagenesis was later observed in the early 2000s when five out of 20 pediatric subjects in a European trial developed gene transfer-related leukemia 2 to 5.5 years after gene therapy, resulting in the death of one child [8].

These events exemplified the potential for serious adverse events associated with gene therapy and highlighted the need for increased regulatory oversight and safety precautions. They led to the FDA and NIH taking additional individual and cooperative actions to strengthen protections for trial participants. Among these initiatives were the establishment of the Gene Therapy Clinical Trial Monitoring Plan and the Gene Transfer Safety Symposia [9].

ClinicalTrials.gov was also launched which provided a high level of transparency for gene therapy trials conducted by both public and private sponsors [6]. Additionally, the NIH and FDA jointly launched the Genetic Modification Clinical Research Information System (GeMCRIS), an information resource and analytical tool to coordinate adverse event reporting and expand public access to reports of serious events [5].

Unfortunately, despite these efforts, confidence in gene therapy appeared to falter. After hitting a new peak in 2008, the number of completed, ongoing or approved clinical trials worldwide declined between 2009 and 2012 [7].

A New Era in Gene Therapy and U.S. Regulatory Oversight

Within the last 8 years, the gene therapy field appears to have finally hit a major turning point in the United States. There has been a quantum leap forward with the discovery of new technologies and advancements in safer gene delivery systems.

In 2017, the first three gene therapy products were approved for use in the U.S. market: Kymriah (Novartis), Yescarta (Gilead), and Luxturna (Spark Therapeutics). Kymriah and Yescarta are cell-based gene therapies for the treatment of cancer (ex vivo treatments), whereas Luxturna is an adeno-associated virus (AAV) vector-based gene therapy indicated for treatment of an inherited form of blindness.

Luxturna was regarded as a landmark approval as it was the first approved gene therapy product to be administered in vivo and the first to target a specific genetic condition [6]. Another milestone was achieved in 2019 when FDA approved Zolgensma (AveXis), the second AAV vector-based gene therapy for a hereditary disease. Zolgensma is the first approved gene therapy for spinal muscular atrophy and for any chronic neurologic disease.

The U.S. has also not been alone in sharing such momentous achievements.  Gene therapies have previously been approved in Europe and China (Glybera, Stremelis, Gendicine).

With such recent historical successes, as well as significant advancements in gene therapy technologies, interest in the gene therapy sector has exploded and development of gene therapy products is now moving at an accelerated pace.

Today, there are many active gene therapy clinical trials underway to treat various diseases such as cancer, HIV/AIDS, and blood disorders. The FDA anticipates approving 10 to 20 cell and gene therapy products a year by 2025 and is working to expand its review group by about 50 additional reviewers dedicated to evaluation of gene therapy INDs [10].

The regulatory framework and oversight have also continued to evolve with rapidly advancing innovations in the development of gene therapy products. As the field matures and the risks are better understood, regulators continue to streamline duplicative and burdensome oversight efforts.

In 2019, the NIH and FDA jointly announced they were eliminating RAC review and reporting requirements. At the same time, NIH also announced it was renaming and refocusing the RAC into a role closer to its original mandate [11].

In January 2020, FDA announced the release of six final guidance documents on gene therapy manufacturing and clinical development of products and a draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations. In January 2021, the Agency released a draft guidance on human gene therapy for neurodegenerative disease, as well as a final guidance on manufacturing considerations for licensed and investigational cellular and gene therapy products during COVID-19 public health emergency.

This suite of documents not only signifies FDA’s contribution towards helping shape the modern era of gene therapy regulation and oversight but also their commitment to helping ensure such innovative products meet FDA’s standards for quality, safety and effectiveness.

The FDA also continues to encourage sponsors of new gene therapy products to make full use of the expedited programs and other novel regulatory pathways available for products intended to address unmet medical needs in the treatment of serious or life-threatening conditions.

There are currently five expedited approval designations available for gene therapy products: Fast Track; Breakthrough Therapy; Priority Review; Accelerated Approval; and Regenerative Medicine Advanced Therapy (RMAT). While a gene therapy product may qualify for one or more of the five expedited approval designations, the RMAT designation is the only accelerated pathway-focused specifically on regenerative medicine products inclusive of gene therapy.

It carries all of the benefits of Breakthrough and Fast Track designations, including early intensive interactions with the FDA, organizational commitment involving senior managers from the FDA, and actions to expedite product development and review as well as rolling review [12].

The Path Forward

Where do we go from here? While we cannot predict what will happen tomorrow in the field of gene therapy, it is exciting to imagine what is on the horizon.

The field is rapidly expanding with many promising new approaches being developed and studied. Already, there have been discoveries and advancements in new and existing gene-editing systems that have opened the door for treating diseases that might be a challenge to address with gene transfer technologies.

Significant improvements in viral vectors have also driven the recent resurgence in gene therapy efforts. Table 1, for example, provides a snapshot of the numerous gene therapies that were pursued in 2020 using viral vectors to address a missing or mutated endogenous gene [13].

Note: Developmental pipelines can change quickly and the above represents a list as of the date of this article.

With such incredible and complex science and technology behind these gene therapy products, regulators must continue adapting and tailoring the regulatory framework and oversight to meet the unique challenges created by this technology. Some key considerations and challenges that regulators must still address include:

• Continued efforts to standardize safety,
• Facilitating reimbursement and patient access, and
• Developing a practical approach for post-approval product lifecycle management.

These items lead to crucial questions that must be considered, such as:

• To what degree must critical quality attributes be understood for regulators to have confidence in the safety and clinical benefits of the product?
• How can regulators help ensure access to gene therapy products, especially when many come with a hefty price tag?
• Given that gene therapy can offer such personalized treatment, how can regulators offer a pathway to reasonably manage post-approval changes without creating a significant burden on industry?

Concluding Remarks

What is clear is that there is no shortage of questions surrounding gene therapy products and regulatory oversight. From the technical aspects of developing such products to the cost and everything in between, we are at a precipice.

The promise of gene therapy, inclusive of cures and one-time treatments for serious and life-threatening diseases, looms ahead of us. Will the regulatory constructs be able to keep pace with these scientific and clinical breakthroughs so as not to inhibit the staggering potential of gene therapy products? Only time will tell.

G&L Scientific – our role

G&L Scientific is a market-leading, multi-national consultancy firm with over 25 years of experience in Regulatory Affairs and Quality Assurance. G&L is recognized as a leader in Pharmaceutical and Biologics CMC serving clients globally from offices in North America, Europe, and Asia. We combine a unique and dynamic approach to providing collaborative Regulatory Affairs support to your organization ensuring success first time, every time. Client-focused. Industry Experts.

At G&L Scientific, we understand the changing pharmaceutical landscape – as new technologies are ushered in, global regulations and guidelines morph and adapt to ensure the quality, efficacy and safety of medications manufactured under these new modalities. And the pressure to do more with less is an ever-present reality of our industry.

It would be our pleasure to explore potential Regulatory Affairs solutions to the challenges faced within your organization. Please feel free to contact Christine Piscitelli or Jessica Kokosinski with your thoughts on this article or how we can help address your regulatory affairs challenges.

References

• S. Food and Drug Adminstration. (2020, January 28). FDA continues strong support of innovation in development of gene therapy products [Press release]. Retrieved from: https://www.fda.gov/news-events/press-announcements/fda-continues-strong-support-innovation-development-gene-therapy-products
• Wirth T, Parker N, Yla-Herttuala S. History of gene therapy. Gene. (2013) 525:162–9. doi: 10.1016/j.gene.2013.03.137
• Szybalska EH, Szybalski W. Genetics of human cess line. IV. DNA-mediated heritable transformation of a biochemical trait. Proc Natl Acad Sci USA. (1962) 48:2026–34. doi: 10.1073/pnas.48.12.2026
• Friedmann T, Roblin R. Gene therapy for human genetic disease? Science. (1972) 175: 949–955. doi: 10.1126/science.175.4025.949
• Institute of Medicine. (2014). Oversight and Review of Clinical Gene Transfer Protocols: Assessing the Role of the Recombinant DNA Advisory Committee. Washington, DC: The National Academies Press. https://doi.org/10.17226/18577.
• Collins FS, Gottlieb S. The next phase of human gene-therapy oversight. N Engl J Med. (2018) 379:1393-1395. doi: 10.1056/NEJMp1810628
• The Journal of Gene Medicine Clinical Trial site. Retrieved from: http://www.abedia.com/wiley/
• Cavazzana M, Six E, Lagresle-Peyrou C, André-Schmutz I, Hacein-Bey-Abina S. Gene therapy for x-linked severe combined immunodeficiency: where do we stand? Human Gene Therapy. (2016) 27:108-116. doi: 10.1089/hum.2015.137
• Sibbald, B. Death but one unintended consequence of gene-therapy trial. (2001) 164: 1612.
• S. Food and Drug Administration. (2019, January 15). Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies [Press release]. Retrieved from: https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics
• National Institutes of Health. (2019, April 25). NIH streamlines gene therapy oversight and charts a course for considering emerging technology [Press release]. Retrieved from: https://www.nih.gov/about-nih/who-we-are/nih-director/statements/nih-streamlines-gene-therapy-oversight-charts-course-considering-emerging-technology
• S. Food and Drug Administration. Guidance for Industry: Expedited programs for regenerative medicine therapies for serious conditions. Retrieved from: https://www.fda.gov/media/120267/download
• Curran, K. (2020, March 10). The gene therapy sector is experiencing an acceleration. Rising Tide Bio. Retrieved from: https://www.risingtidebio.com/what-is-gene-therapy-uses/
• S. Food and Drug Administration. (2018, July 25). What is gene therapy? Retrieved from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
• S. Food and Drug Administration. (2018, July 11). Statement from FDA Commissioner Scott Gottlieb, M.D. on agency’s efforts to advance development of gene therapies [Press release]. Retrieved from: https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-agencys-efforts-advance-development-gene-therapies
• Magers K. (2019, July 24). Addressing challenges in meeting chemistry, manufacturing and control regulatory requirements for gene therapy products. Cell & Gene Therapy Insights. Retrieved from: https://insights.bio/cell-and-gene-therapy-insights/journal/articles/addressing-challenges-in-meeting-chemistry-manufacturing-and-control-regulatory-requirements-for-gene-therapy-products/
• Sargent B, Glover C, Jaluria P, Madsen J, Moore Al, Tredenick T. (2018, September 12). Key considerations for gene therapy commercialization. Cell Culture Dish. Retrieved from: https://cellculturedish.com/key-considerations-for-gene-therapy-commercialization/
• Goswami R, Subramanian G, Silayeva L, Newkirk I, Doctor D, Chawla K, Chattopadhyay S, Chandra D, Chilukuri N, Betapudi V. Gene therapy leaves a vicious cycle. Frontiers in Oncology. (2019) 9:297. doi: 10.3389/fonc.2019.00297

[1] These trials rely on viral vectors to deliver genome editing technology (i.e., ZFN, CRISPR).

RAMSEY, N.J. and BOCA RATON, Fla., Jan. 07, 2019 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), a vertically integrated commercial biopharmaceutical and specialty immunoglobulin company that manufactures, markets and develops specialty plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious diseases, announces that the Company has submitted responses to the U.S. Food and Drug Administration (“FDA”) Complete Response Letter (“CRL”) received on December 19, 2018 for BIVIGAM®’s (Intravenous Immune Globulin [Human], 10%) (“IVIG”) Prior Approval Supplement (“PAS”). The Company anticipates receiving an acknowledgement letter from the FDA within 30 days, and plans to provide appropriate updates on the progress of the BIVIGAM® PAS review. 

About ADMA Biologics, Inc. (ADMA)
ADMA Biologics is a vertically integrated commercial biopharmaceutical company that manufactures, markets and develops specialty plasma-based biologics for the treatment of Primary Immune Deficiency Disease (“PIDD”) and the prevention and treatment of certain infectious diseases.  ADMA's mission is to develop and commercialize plasma-derived, human immune globulins targeted to niche patient populations for the treatment and prevention of certain infectious diseases. The target patient populations include immune-compromised individuals who suffer from an underlying immune deficiency disease, or who may be immune-compromised for other medical reasons. ADMA has received U.S. Patents 9,107,906, 9,714,283, 9,815,886 and 9,969,793 related to certain aspects of its lead product candidate, RI-002.  For more information, please visit www.admabiologics.com.

About BIVIGAM®
BIVIGAM® is an intravenous immune globulin indicated for the treatment of primary humoral immunodeficiency. This includes, but is not limited to, agammaglobulinemia, common variable immunodeficiency, Wiskott-Aldrich syndrome and severe combined immunodeficiency. These primary immunodeficiencies (“PI”) are a group of genetic disorders. Initially thought to be very rare, it is now believed that as many as 250,000 people in the U.S. have some form of PI. BIVIGAM® contains a broad range of antibodies similar to those found in normal human plasma. These antibodies are directed against bacteria and viruses, and help to protect PIDD patients against serious infections. BIVIGAM® is a purified, sterile, ready-to-use preparation of concentrated polyclonal Immunoglobulin (“IgG”) antibodies. Antibodies are proteins in the human immune system that work to defend against infections and disease. FDA’s initial approval for BIVIGAM® was received by Biotest Pharmaceuticals Corporation (“BPC” or “Biotest”) on December 19, 2012, and production of BIVIGAM® was halted by Biotest in December 2016.  ADMA Biologics obtained ownership and all rights, title and interest in BIVIGAM® on June 6, 2017 as part of the Biotest Therapy Business Unit (“BTBU”) asset acquisition and resumed the production of BIVIGAM during the fourth quarter of 2017.   

Cautionary Note Regarding Forward-Looking Statements
This press release contains "forward-looking statements" pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about ADMA Biologics, Inc. ("we“, “our” or the “Company”).  Forward-looking statements include, without limitation, any statement that may predict, forecast, indicate, or imply future results, performance or achievements, and may contain the words "estimate," “project,” "intend," “forecast,” "target,” ”anticipate,” “plan,” “planning,” “expect,” “believe,” “will," “is likely,” “will likely,” “should,” “could,” "would," "may," or, in each case, their negative, or words or expressions of similar meaning. These forward-looking statements also include, but are not limited to, statements concerning our plans to develop, manufacture, market, launch and expand our own commercial infrastructure and commercialize our current products and future products, the safety, efficacy and expected timing of, and our ability to, obtain and maintain regulatory approvals of our current products and product candidates, and the labeling or nature of any such approvals, the success of our work with our third party vendors and the U.S. Food and Drug Administration (the “FDA”) in furtherance of and progress towards an approval of our Biologics License Application for specialty plasma-based biologics and the ability of such third parties to respond adequately or in a timely manner to the issues raised by the FDA, our ability to successfully pursue commercialization and prelaunch activities, the timeframe within which we may receive approval from the FDA for specialty plasma-based biologics, if at all, the potential of our specialty plasma-based biologics to provide meaningful clinical improvement for patients living with Primary Immune Deficiency Disease or other indications, our ability to realize increased prices for plasma growth in the plasma collection industry and our expectations for future capital requirements. Actual events or results may differ materially from those described in this document due to a number of important factors. Current and prospective security holders are cautioned that there also can be no assurance that the forward-looking statements included in this press release will prove to be accurate. In light of the significant uncertainties inherent in the forward-looking statements included herein, the inclusion of such information should not be regarded as a representation or warranty by ADMA or any other person that the objectives and plans of ADMA will be achieved in any specified time frame, if at all. Except to the extent required by applicable laws or rules, ADMA does not undertake any obligation to update any forward-looking statements or to announce revisions to any of the forward-looking statements. Forward-looking statements are subject to many risks, uncertainties and other factors that could cause our actual results, and the timing of certain events, to differ materially from any future results expressed or implied by the forward-looking statements, including, but not limited to, the risks and uncertainties described in our filings with the U.S. Securities and Exchange Commission, including our most recent reports on Form 10-K, 10-Q and 8-K, and any amendments thereto.

COMPANY CONTACT:
Brian Lenz
Executive Vice President and Chief Financial Officer | 201-478-5552 | www.admabiologics.com

INVESTOR RELATIONS CONTACT:
Jeremy Feffer
Managing Director, LifeSci Advisors, LLC | 212-915-2568 |

Source: ADMA Biologics, Inc.

Released January 7, 2019

Organization celebrates research achievements and new partnerships - and shares easy ways to get involved

TAMPA, Fla., Sept. 24, 2018 /PRNewswire-PRWeb/ -- The National Pediatric Cancer Foundation – the top-rated cancer charity according to Charity Navigator – invites all to rise up for a faster cure during Childhood Cancer Awareness Month. Every day, 43 children are diagnosed with cancer and 43 families are faced with a future of uncertainty. Cancer is the number one cause of death by disease among children, yet only 4% of the billions of dollars spent annually on cancer research and treatment is directed toward pediatric cancer. Since 1980, fewer than 10 drugs have been developed for use in children with cancer, which means most children are treated with drugs designed specifically for adults, often leading to significant health-related issues later in life.

September is Childhood Cancer Awareness Month, and the National Pediatric Cancer Foundation is helping shine a light on pediatric cancer and the need for additional research and funding. The National Pediatric Cancer Foundation brings together leading doctors and researchers across the nation's top hospitals to fund research that fast-tracks less toxic, more targeted treatments by encouraging collaboration across hospitals.

Fueling a cure through partnerships:
The National Pediatric Cancer Foundation has announced new partnerships and research updates that are helping find a cure:

•     Biden Cancer Summit - On September 21, the National Pediatric Cancer Foundation joined Vice President Joe Biden and Dr. Jill Biden as they hosted the national Biden Cancer Summit in Washington, D.C. The Biden Cancer Initiative called upon public and private sector organizations to develop new innovative programs and partnerships. The National Pediatric Cancer Foundation (NPCF) is proud to embark on a (+/-) 2-year joint-effort to systematically address improvements for pediatric oncology (relapsed patient) treatments, guidelines and pathways driven through data and science. While formalized in the adult community, standards of care in pediatric oncology lag behind with resultant variation and possible recommendations for sub-optimal therapies instead of clinical trials. The outcome of this project will be to author evidence based, collaborative, treatment recommendations for "relapsed" pediatric and young adult oncology patients to ensure they receive the best diagnostic and therapeutic services to increase the chance of a positive outcome.
•     HOSA Partnership – HOSA-Future Health Professionals has partnered with the National Pediatric Cancer Foundation for a two-year national service project. Over 235,000 middle, high school and college students in the organization will support pediatric cancer research by conducting awareness, fundraising and career pathway activities now through 2020.
•     Clinical trials – The NPCF supports unique and innovative research through a collaborative model (The Sunshine Project) with 22 hospitals nationwide. In addition to multiple translational and lab projects, the NPCF's current trials include a brain tumor immunotherapy trial at the University of Florida, a phase one trial addressing Ewing Sarcoma – LSD1 inhibitor, and a phase two trial studying the drug combination of Gemcitabine and Nab-Paclitaxel.

Five Ways to "Rise Up" for a Faster Cure:
The National Pediatric Cancer Foundation needs your help to fight pediatric cancer. It's easy to start. Here are five ways you can fight pediatric cancer during Childhood Cancer Awareness Month, and all year round.

•     Brewing Funds the Cure — The National Pediatric Cancer Foundation has partnered with breweries across the United States to launch its "Brewing Funds the Cure" Campaign. Proceeds from the beer, dubbed "Rising Hope," will help fund pediatric cancer research. The "Rising Hope" recipe differs from brewery to brewery and is available at Cigar City Brewing (Tampa, FL), Brew Bus Brewing (Tampa, FL), Honolulu BeerWorks (Honolulu, HI), Flying Heart Brewery (Bossier City, LA), Remedy Brewing (Sioux Falls, SD), and Grey Sail Brewing (Westerly, RI). Find out more here.
•     Cooking Funds the Cure — Through the end of September at Metro Diner, diners can round-up their check to the nearest dollar in increments of $1, $2 and $5, or provide a custom amount to support the National Pediatric Cancer Foundation. Metro Diner has 62 locations nationwide and is known for serving serious diner food from an extensive menu of traditional diner dishes. Your restaurant can join the Cooking Funds the Cure by reaching out to cooking(at)nationalpcf(dot)org or you can sign up to participate as an individual in the online fundraising platform.
•     Fashion Funds the Cure — The NPCF will host its signature event "Fashion Funds the Cure" in 12 markets this coming year; see the full list of events, here. You can also purchase a bracelet from Lokai during Childhood Cancer Awareness Month, and the company will donate $1 to the National Pediatric Cancer Foundation. The yellow Lokai bracelets have helped raise over $80,000 to date.
•     Donate with a click — 43 children are diagnosed with cancer each day. Donate $43 during Childhood Cancer Awareness on the National Pediatric Cancer Foundation's website to join the fight. You can also set up a recurring or planned donation.
•     Fund the Cure your own way — From fishing outings, to haircuts, to active recreation, there are many ways to participate in a fundraiser to support the National Pediatric Cancer Foundation. Start planning, find an event or create your own fundraiser here.

About the National Pediatric Cancer Foundation:
The National Pediatric Cancer Foundation is a nonprofit organization dedicated to funding research to eliminate childhood cancer through the Sunshine Project, the Foundation's collaborative research initiative. By partnering with doctors and researchers from the country's top institutions, the Sunshine Project is fast-tracking the development of new drugs and therapies that will ultimately lead to the cure of childhood cancers. For more information, visit http://www.NationalPCF.org

SOURCE National Pediatric Cancer Foundation

MIAMI, May 23, 2018 /PRNewswire/ -- Longeveron LLC, a biopharmaceutical company that develops stem cell therapies for age-related diseases, announced that it is currently recruiting for a Phase 1 clinical trial to evaluate the safety and efficacy of its allogeneic Mesenchymal Stem Cells (MSCs) in patients with Alzheimer's disease.  The randomized, placebo-controlled, double-blinded trial is seeking up to 30 participants and is being conducted at the University of Miami Miller School of Medicine, Miami Jewish Health Systems, and Brain Matters Research in Delray Beach, Florida.

Eligible participants should be between 50 and 80 years old, be diagnosed with Alzheimer's in accordance with established criteria, and have an identified adult caregiver.  Longeveron's research is supported by the Alzheimer's Association Part the Cloud Challenge on Neuroinflammation.

Longeveron's MSC product is derived from the bone marrow of young, healthy donors and is currently being tested in a variety of indications in clinical trials, including Aging Frailty.  In 2017, the company published positive Phase I and Phase 2 Aging Frailty study results in the Journals of Gerontology.  Frail patients showed marked improvement in physical performance, lung function and inflammation biomarkers, with no serious adverse effects attributed to the treatment. 

"Regenerative medicine is the future.  Controlling inflammation in Alzheimer's disease is the key to a breakthrough," noted Mark Brody, M.D. of Brain Matters Research in Delray Beach, Florida, a site for the clinical trial. 

Marc Agronin, M.D., geriatric psychiatrist and vice president of Behavioral Health and Clinical Research at Miami Jewish Health, said, "Alzheimer's disease is one of the most pressing health care issues for aging individuals, and we need an effective treatment, if not a cure.  An innovative study such as the Longeveron stem cell clinical trial is an important and promising step forward."

In addition to the Alzheimer's trial, Longeveron is recruiting nationally for an expanded Phase 2b Aging Frailty study, as well as Phase 1 and Phase 2 trials to evaluate the safety and efficacy of its stem cells for improving flu vaccine immune response in aging frailty patients.  For more information about the Alzheimer's trial and other trials Longeveron is sponsoring, visit ClinicalTrials.gov or Longeveron's Web site www.longeveron.com

About Longeveron

Longeveron (www.longeveron.com) is a regenerative medicine therapy company founded in 2014.  Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life.  The company's research focus areas include Alzheimer's disease, Aging Frailty, and the Metabolic Syndrome, and gratefully acknowledges the generous support of the National Institutes of Health and the Alzheimer's Association.  Longeveron is also conducting a Phase 1 trial with the University of Maryland and Johns Hopkins University to study Hypoplastic Left Heart Syndrome, a rare indication that affects infants, and gratefully acknowledges the support and collaboration from the Maryland Stem Cell Research Fund. 

SOURCE Longeveron LLC

Related Links

http://longeveron.com

Parade float will highlight the life-changing power of tissue and organ donation

ALACHUA, Fla., Dec. 18, 2017 -- AxoGen, Inc. (NASDAQ:AXGN), a global leader in developing and marketing innovative surgical solutions for peripheral nerve injuries, today announced that Dylan Harvey was selected to ride on the 2018 Donate Life Rose Parade® float that will be featured in the annual Tournament of Roses® parade. The parade takes place in Pasadena on New Year's Day.

Dylan Harvey, 19, was a sophomore in high school when he was seriously injured in a motorcycle accident and spent several weeks in the ICU undergoing multiple surgeries to repair injuries to his left arm and right leg. Once stabilized, however, Dylan was unable to move most of his left arm due to significant nerve damage. Physicians suggested his arm may need to be amputated. In 2015, Dylan underwent nerve reconstruction including the implant of a processed nerve allograft from donated tissue. Today, Dylan helps his father run an electronics business and is currently pursuing acting and filmmaking classes. His participation in the parade is sponsored by AxoGen Corporation.

Damaged peripheral nerves can be surgically repaired, but the window of time for effective outcomes is limited, so expertise from a nerve specialist must be sought quickly. Fortunately, the Harvey family found David Adelson, M.D., the director of Barrow Neurological Institute and chief of pediatric neurosurgery at Phoenix Children's Hospital. Dr. Adelson performed surgery and utilized AxoGen's Avance® Nerve Graft to help repair the damaged nerve in Dylan's left arm.  

"If it weren't for Dr. Adelson and the generosity of a nerve tissue donor, I could be sitting here in a vastly different state," said Dylan. "I encourage everyone to become a tissue and organ donor, and am grateful for the opportunity to share my personal story by participating as a rider on the Donate Life Rose Parade® float."

Avance® Nerve Graft is an off the shelf processed human nerve allograft intended for the surgical repair of peripheral nerve discontinuities. Using Avance® Nerve Graft eliminates the comorbidities associated with a second surgical site.

"Nearly a million Americans undergo surgical procedures to repair peripheral nerve damage every year," said Karen Zaderej, president and chief executive officer of AxoGen. "There are many causes of peripheral nerve damage - from car accidents, to power tool accidents, to severe cuts. Nerve damage can result in loss of sensation or movement and reduced quality of life. AxoGen is dedicated to transforming nerve repair by providing innovative solutions to surgeons and their patients."

Dylan will ride alongside 16 other organ, eye, or tissue recipients, honoring their donors. The Donate Life Rose Parade® float is the centerpiece of a national effort of more than 50 organizations to reach a broad audience with the simple, life-giving message that organ, eye, and tissue donation saves and improves lives.

About Donate Life America
Donate Life America is a 501(c)3 not-for-profit alliance of national organizations and state teams across the United States committed to increasing organ, eye and tissue donation. Donate Life America manages and promotes the national brand for donation, Donate Life, and assists Donate Life State Teams and national partners in facilitating high-performing donor registries; developing and executing effective multi-media donor education programs; and motivating the American public to register now as organ, eye and tissue donors.

About AxoGen
AxoGen (AXGN) is the leading company focused specifically on the science, development and commercialization of technologies for peripheral nerve regeneration and repair. We are passionate about helping to restore nerve function and quality of life to patients with peripheral nerve injuries by providing innovative, clinically proven and economically effective repair solutions for surgeons and health care providers. Peripheral nerves provide the pathways for both motor and sensory signals throughout the body. Every day, people suffer traumatic injuries or undergo surgical procedures that impact the function of their peripheral nerves. Damage to a peripheral nerve can result in the loss of muscle or organ function, the loss of sensory feeling, or the initiation of pain.

AxoGen's platform for nerve repair features a comprehensive portfolio of products, including Avance® Nerve Graft, an off-the-shelf processed human nerve allograft for bridging severed nerves without the comorbidities associated with a second surgical site, AxoGuard® Nerve Connector, a porcine submucosa extracellular matrix (ECM) coaptation aid for tensionless repair of severed nerves, AxoGuard® Nerve Protector, a porcine submucosa ECM product used to wrap and protect injured peripheral nerves and reinforce the nerve reconstruction while preventing soft tissue attachments, and Avive® Soft Tissue Membrane, a minimally processed human umbilical cord membrane that may be used as a resorbable soft tissue covering to separate tissue layers and modulate inflammation in the surgical bed. Along with these core surgical products, AxoGen also offers AcroVal® Neurosensory & Motor Testing System and AxoTouch® Two-Point Discriminator. These evaluation and measurement tools assist health care professionals in detecting changes in sensation, assessing return of sensory, grip, and pinch function, evaluating effective treatment interventions, and providing feedback to patients on nerve function. The AxoGen portfolio of products is available in the United States, Canada, the United Kingdom, and several other European and international countries.

Cautionary Statements Concerning Forward-Looking Statements
This Press Release contains "forward-looking" statements as defined in the Private Securities Litigation Reform Act of 1995. These statements are based on management's current expectations or predictions of future conditions, events, or results based on various assumptions and management's estimates of trends and economic factors in the markets in which we are active, as well as our business plans. Words such as "expects," "anticipates," "intends," "plans," "believes," "seeks," "estimates," "projects," "forecasts," "continue," "may," "should," "will," and variations of such words and similar expressions are intended to identify such forward-looking statements. The forward-looking statements may include, without limitation, statements regarding our assessment on our internal control over financial reporting, our growth, our 2017 and 2018 guidance, product development, product potential, financial performance, sales growth, product adoption, market awareness of our products, data validation, our visibility at and sponsorship of conferences and educational events. The forward-looking statements are subject to risks and uncertainties, which may cause results to differ materially from those set forth in the statements. Forward-looking statements in this release should be evaluated together with the many uncertainties that affect AxoGen's business and its market, particularly those discussed in the risk factors and cautionary statements in AxoGen's filings with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and actual results may differ materially from those projected. The forward-looking statements are representative only as of the date they are made and, except as required by law, AxoGen assumes no responsibility to update any forward-looking statements, whether as a result of new information, future events, or otherwise.

Contact:
Annette Ruzicka
AxoGen Corporate & Investor Communications
aruzicka@AxoGenInc.com
(763) 458-4193