12/06/2021
**Bespoke Gene Therapy Consortium: Request for Proposals and a Disease Nomination Form**
In order to support the newly-launched Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (BGTC), the Foundation for the National Institutes of Health (FNIH) has released multiple opportunities for researchers, clinicians, and patient advocates to contribute to the work of the consortium.
The BGTC is seeking high-throughput screens or other promising developments to optimize individual steps of the AAV vector generation and human gene expression pathways. To read the AAV Vector Generation RFP, click here. To read the AAV Gene Expression RFP, click here: http://ow.ly/aF9X50H4c74.
The BGTC Steering Committee also seeks information on rare diseases and disorders that could be candidates for AAV gene therapy trials conducted by the BGTC. A Disease Nomination form is has been made available to obtain information about the rare disease and the patient population, prior R&D work, and high-level factors that would be considered in designing a clinical trial. Patient groups, clinicians, and researchers are strongly encouraged to collaborate on preparing a disease or disorder nomination. Selected nominations will be invited to submit a full RFP at a later date. To access the Disease Nomination form, click here.
The FNIH plans to hold informational webinars about each of these opportunities, with details to follow on the BGTC website. RFP submissions and completed Disease Nomination forms are due by February 18, 2022 to [email protected].
NIH Press Release: http://ow.ly/UuWT50H4c70
NIH BGTC webpage: http://ow.ly/Xx7m50H4c73
FNIH Press Release: http://ow.ly/uwZb50H4c72
FNIH BGTC webpage: http://ow.ly/cBtC50H4c71
Breadcrumb Home Bespoke Gene Therapy Consortium (BGTC) Bespoke Gene Therapy Consortium (BGTC) Skip This Section The Problem With the current commercial drug development model, companies cannot recover the costs required to develop gene therapies to treat rare and ultra-rare genetic diseases, because...