8 rare diseases were selected for the portfolio of AMP BGTC gene therapy trials

The Foundation for the National Institutes of Health (FNIH) said the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its portfolio of clinical trials.

This portfolio pioneers new approaches to the development of gene therapies for rare diseases by demonstrating that manufacturing and testing standards can provide a simplified approval pathway for the first clinical trials in humans. The announcement was made on the first day of the American Society of Gene & Cell Therapy (ASGCT) 26th annual meeting in Los Angeles.

Administered by the FNIH, AMP BGTC is a public-private partnership between the National Institutes of Health (NIH), the US Food and Drug Administration (FDA), biopharmaceutical and life science companies, and non-profit and other organizations to help accelerate the development and delivery of effective gene therapies. customized, or “bespoke”, treatments that can treat millions of people affected by rare diseases.

BGTC aims to streamline the regulatory approval process by establishing minimum standards for manufacturing, product analytical testing, and pre-clinical testing. All of this information will be provided to the public by means of standard templates and an instructional “development manual” free of charge.

“BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, CEO of FNIH.

“The announcement of its portfolio of clinical trials is an important milestone, bringing people with rare genetic diseases one step closer to approved therapies.”

What rare diseases will the portfolio of AMP BGTC gene therapy trials be?

The eight rare diseases that will be part of the clinical trial portfolio are:

  • Charcot-Marie-Tooth disease type 4J
  • Congenital hereditary endothelial dystrophy
  • Morquio A syndrome
  • Multiple sulfatase deficiencies
  • NPHP5 retinal degeneration
  • Propionic acid (PCCB)
  • Retinitis pigmentosa 45
  • Spastic paraplegia 50

Eight were selected based on a scientific and technical review by a panel of gene therapy experts using criteria set by the BGTC. These criteria include, but are not limited to, sufficiency of the gene for inclusion into an adeno-associated virus (AAV) vector, sufficient proof of concept and natural history data, presence of an established disease model, lack of available treatment and overall readiness to enter clinical trials. .

Potential path

“As the parent of a child with a rare disease, it has been a long and arduous journey to research and test safe and effective treatments,” said Terry Pirovolakis, patient advocate, founder of CureSPG50 and CEO of Elpida Therapeutics.

“BGTC offers a path of potential and hope for children who have no other choice.”

More than 30 million people in the US are living with the devastating effects of rare diseases. There are more than 10,000 rare diseases, and more than 80% of them are caused by genetic defects. Rare disease patients and their families often suffer without hope of treatment, as many rare diseases are neglected due to small patient populations and limited commercial viability.

“With this portfolio of clinical trials, we are able to build a bridge that creates a standardized and publicly available roadmap for all AAV gene therapies to follow, with iterative solutions for templates, regulatory files, and manufacturing processes,” said Courtney Silverthorn, associate vice president, partnership science, at FNIH.

“A single rare disease can affect hundreds of thousands of people globally, or just a few dozen, and BGTC aspires to make gene therapy more accessible and sustainable, no matter the size of the patient population.”

BGTC has more than $97 million in financial and in-kind commitments from its partnerships with 33 member organizations, including 11 NIH institutes and centers, 12 life sciences companies, and 10 nonprofits.

The FNIH is also active in other areas, such as the fight against cancer.

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