Accelerate the development of gene therapy FDA/NIH leads the establishment of a research and development alliance

A few days ago, the US FDA announced that the FDA, the National Institutes of Health (NIH), 10 biopharmaceutical companies and 5 non-profit organizations have reached a partnership to jointly accelerate the development of gene therapies and treat patients with rare diseases

The FDA pointed out that there are nearly 7,000 rare diseases in the world, but currently only two genetic diseases have received FDA-approved gene therapy

However, the development of gene therapy for rare diseases is a highly complex, time-consuming and labor-intensive process

One of the primary goals of BGTC is to deepen the understanding of the basic biology of adeno-associated virus (AAV) vectors

In order to improve and speed up the production process of gene therapy and vector, BGTC will develop a set of standardized analysis tests, which can be widely used in different production methods to improve the efficiency of gene therapy development

In terms of clinical development, BGTC will sponsor 4-6 clinical trials focusing on different rare diseases

NIH and private partners will provide approximately $76 million to support these projects

Biomedical and technology companies that have joined BGTC include: Bojian, Janssen, Novartis Biomedical Research Institute, Pfizer, REGENXBIO, Spark Therapeutics, Takeda, Taysha Gene Therapies, Thermo Fisher Scientific, and Ultragenyx Pharmaceutical

Reference materials:

[1] FDA, NIH, and 15 private organizations join forces to increase effective gene therapies for rare diseases.

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