Bespoke Gene Therapy Consortium (BGTC)

Bespoke Gene Therapy Consortium (BGTC)

Project overall strategic goals

The AMP Bespoke Gene Therapy Consortium aims to improve our understanding of therapeutically relevant biological pathways and validate information that could be relevant for the development of multiple therapeutics.

Short description of the main outputs of the project/coalition

The program focuses on generating a standard operational playbook for developing such gene therapies. This playbook, which will be established and piloted using up to eight clinical trial test cases, will include streamlined templates, master regulatory files, and uniform manufacturing processes to create a pathway toward the commercial viability and sustainability of gene therapies for very rare diseases by:

  • Making adeno-associated virus (AAV) technology more accessible to a broader range of diseases.
  • Accelerating the potential to streamline preclinical and product testing.
  • Facilitating scientific and regulatory advances that will ultimately benefit the entire field.
  • Bringing gene therapies to all individuals in need sooner.

How does the project/coalition address the specificities of the white spots?

“As thousands of the white spots are most likely genetic disease, the strategy of the Bespoke Gene Therapy Consortium is to make the process of developing gene therapies for rare diseaess much more efficient, and therefore applies to the white spots by the development of platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases”

How is the project/coalition a public-private partnership? 

The Accelerating Medicines Partnership program is a public-private partnership between the NIH, the FDA and multiple public and private organizations. Managed through the Foundation for the NIH.

How being a PPP helps to address the white spots?

Across the Consortium we have assembled the subject matter expertise of over 200 individuals from federal scientific research and regulatory agencies, industry representation from companies who have successfully brought AAV gene therapies to market, academics and clinicians who have worked with rare disease populations, non-profits who amplify the voice of the patient and advocate for the gene therapy and rare disease fields, and experts in manufacturing, pre-clinical testing, and regulatory science. This type of large, broad-scale PPP is necessary to undertake the complex work of accelerating AAV gene therapies to patients, particularly those in the white spots that lack commercial viability in the current landscape.