AMP® Symposium Day 2: Bespoke Gene Therapy Consortium

The Accelerating Medicines Partnership® (AMP®) Program Bespoke Gene Therapy Consortium (AMP BGTC) brings together partners from the public, private, and non-profit sectors to foster development of gene therapies intended to treat rare genetic diseases, which affect populations too small for viable commercial development.

Building on the successful AMP model, this 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. This approach could have substantial positive impacts on the larger gene therapy field, especially as the field moves into the era of genome editing.

The Accelerating Medicines Partnership® (AMP®) Program was launched in February 2014 to bring together the resources and expertise of both the National Institutes of Health (NIH) and the private sector to solve pressing challenges in various diseases and disease areas. The AMP program, managed by the FNIH, is comprised of projects with a common goal of accelerating the process of bringing new and effective therapies to patients and operates under the broad principle of delivering pre-competitive advancements to the research and medical fields to enable broad access and use of AMP research discoveries. Originally focused on three specific diseases, the program has since grown to 10 individual projects representing the contributions and participation of 16 NIH institutes and over 70 private sector partners.

This inaugural scientific symposium celebrated the 10th anniversary of the program, highlighted successful innovation and collaboration in each of the individual AMP project areas, and provided a forum for discussions and cross-collaboration in the broader AMP research community.

https://fnih.org/our-programs/accelerating-medicines-partnership-amp/bespoke-gene-therapy-consortium-bgtc/

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