This partnership aims to accelerate gene therapy

A public-private partnership, led by the Foundation for the National Institutes of Health (NIH), announced eight rare diseases for a clinical trial portfolio that will help accelerate gene therapies. Plus, the World Organization for Animal Health’s 90th general session opens with a focus on avian flu. (678 words, 3 minutes, 23 seconds)

This public-private partnership aims to accelerate gene therapy

A public-private partnership led by the Foundation for the National Institutes of Health (FNIH) has selected rare diseases for a groundbreaking clinical trial portfolio to help streamline and accelerate the development of gene therapies.

What: FNIH announced the selection of eight rare diseases for inclusion in the Accelerating Medicine Partnership Bespoke Gene Therapy Consortium (AMP BGTC) clinical trial portfolio, reports Bio.News.

Who: AMP BGTC is a public-private partnership between NIH, the U.S. Food and Drug Administration (FDA), biopharmaceutical companies, regulators, and patient advocacy groups.

How: The consortium aims to “standardize and streamline the process of going from an idea for gene therapy to an actual clinical trial," creating a “development playbook” and “repeatable pathway” for future clinical trials.

What they’re saying: “Over 175 people have come together across the entire consortium effort bringing a wide array of subject matter expertise and great discussion to the table with the aim to bring diversity across the portfolio,” FNIH’s Dr. Courtney Silverthorn told Bio.News. “It is also important to recognize that even organizations representing diseases not reflected within the clinical trial portfolio continue to support the consortium knowing that its work will benefit their disease communities, as well.”

Why it matters: “The efforts of the BGTC not only help to bring families with rare diseases together but take the responsibility and burden away so we can redirect our focus on being parents,” said Jocelyn Duff, whose daughter was diagnosed with Charcot-Marie-Tooth disease type 4J (CMT4J), one of the eight diseases included in the portfolio. Learn more about work to find cures for CMT and other rare diseases in this episode of the I am BIO podcast.

Read more about the announcement and what’s next for the clinical trial portfolio.

More Health News:

Bloom Science: Bloom Science names Dr. Grace E. Colón Board Chair
“A distinguished scientist and leading executive in biotechnology, Dr. Colón will lend her business, scientific, clinical and operational expertise towards helping Bloom Science in an exciting stage of research and development of new diagnostics and transformative medicines for patients with complex neurological disorders.” (Dr. Colón is a BIO Board member.)

Wall Street Journal (Opinion): Antitrust gone wild against Amgen
“The two drugs currently don’t have competition, and they may never get any. The Food and Drug Administration designated both ‘orphan drugs’ because they treat rare diseases,” says the WSJ Editorial Board.

Is it time for avian flu vaccination?

As avian flu ravages the world’s bird population, it’s time to consider a vaccination campaign, says the head of the World Organization for Animal Health (WOAH). Human infections are rare—though mutations that enable the spread between humans could bring about a pandemic.

What’s happening: WOAH’s 90th general session, which runs through Thursday in Paris, focuses on Highly Pathogenic Avian Influenza (HPAI), which has killed hundreds of millions of birds and infected mammals worldwide.

Bird vaccine hesitancy: Vaccinated birds test positive for HPAI, which is the main reason why only 25% of WOAH member states say they would accept imported vaccinated poultry, and why the U.S. relies on biosecurity instead of vaccination.

What they’re saying: “Since almost every country that does international trade has now been infected, maybe it’s time to discuss vaccination,” WOAH Director General Monique Eloit told Reuters. Because of COVID, “every country realized the hypothesis of a pandemic was real.”

The USDA still prefers biosecurity—but says it will consider a vaccine, Reuters reports. U.S. HPAI vaccine trials are already underway.

What’s next: The “Animal Health Forum on Avian Influenza” at the WOAH General Session kicks off today; bird flu and other global animal health concerns are expected to be discussed all week.

President Biden’s Monday: Resuming debt limit talks with House Speaker Kevin McCarthy this afternoon. Speaker McCarthy, who agreed to the talks yesterday, is more optimistic that they’ll come to a resolution than before, says The New York Times.

What’s Happening on Capitol Hill: Quiet today. Tomorrow, the House Oversight Committee holds a hearing on “The Role of Pharmacy Benefit Managers in Prescription Drug Markets Part I: Self-Interest or Health Care?” Announcing the hearing, Chair James Comer (R-KY) said: “Pharmacy Benefit Managers’ anticompetitive tactics are driving up health care costs for Americans and harming patient care. Greater transparency in the PBM industry is vital.”