The BGTC is Working To Streamline The Development of Gene Therapies for Rare Diseases

The FDA, the NIH, 5 nonprofits, and 10 pharmaceutical companies, have formed an official partnership to accelerate gene therapy development for rare diseases. Out of 7,000 known rare diseases, just 2 have an FDA approved gene therapy.

This new partnership is titled Bespoke Gene Therapy Consortium (BGTC). It is a part of the NIH Accelerating Medicines Partnership program (AMP) and it is managed by the Foundation for the NIH (FNIH). The primary goal is to streamline the development of gene therapies and get new treatments to patient populations with an unmet need as quickly as possible.  

This is the very first AMP project which is specifically focused on rare diseases.

Gene Therapy & Rare Disease

The vast majority of rare diseases are caused by one singular defective gene. That means there is a huge potential for treatment using gene therapy.

Gene therapy is a treatment method where the defective gene is targeted and replaced with the corrected gene.

Although each rare disease only affects a few people, together they affect millions in the United States alone. Unfortunately, because each therapy will only assist one patient population, they are expensive and time consuming. This means that many companies avoid researching them.

Other challenges include the fact that there aren’t standards across companies, and working on one disease at a time limits the amount of progress that can be made for all patients.  Standardizing a gene therapy development process which includes one common delivery technology, could lead to much faster development for rare disease treatments. It would also save money.


BGTC hopes to streamline the development of rare disease gene therapies, incentivize companies to work in this area, and make it less costly for companies to focus on rare diseases.

They hope to use a common delivery platform called adeno-associated virus (AAV). Using one common platform, they will be able to standardize the process more effectively. They plan to standardize the analytic tests for gene therapy development so that they are applicable to many different methods of manufacturing.

BGTC will ultimately fund 4-6 clinical trials. Each trial will be for its own rare disease. Different AAV vectors will be used in these trails. The team hopes to shorten the time period of research so that its possible to move from animal models to human trials faster.

Additionally, this collaboration will work to make the FDA regulatory process more streamlined for rare disease gene therapies. For instance, they will standardize toxicology studies and other preclinical studies.

Over 76 million dollars from the NIH as well as their partners, will go into this partnership over the course of 5 years. Additionally, NCATS plans to provide over 8 million dollars.

You can read more about this new collaboration here.

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