One Year In, Where Does the Bespoke Gene Therapy Consortium Stand?

In October 2021, ASGCT joined the Bespoke Gene Therapy Consortium (BGTC), a public-private collaboration that aims to accelerate the delivery of gene therapies for patients with rare diseases—particularly diseases that are so rare they challenge the typical commercial development model.

More than a year into this six-year initiative, the partnership has expanded, and efforts to select a handful of diseases for clinical trials are moving forward. In year two, according to organizers, manufacturing and preclinical testing will begin.

The bulk of the consortium’s work is to develop repeatable, optimized processes that will allow people to get into a first-in-human phase 1 gene therapy clinical trial, said Courtney Silverthorn, PhD, associate vice president of research partnerships at the Foundation for the National Institutes of Health (FNIH), which manages the program.

Dr. Silverthorn explained that in a normal pharmaceutical model, it takes a very large patient population and monetary investment to bring a treatment all the way to FDA approval. The return on investment allows for large financial commitments.

“Conversely, over 40% of rare diseases and disorders have fewer than 50 patients,” Dr. Silverthorn said. As such, there are no effective business models to return the investments needed to bring gene therapies to market for diseases that are so rare.

That’s where the BGTC comes in. The program launched to overcome development obstacles and create a gene therapy “playbook” that researchers can use to make the process more efficient. With the help of the FDA, 11 NIH partners, and 23 private sector partners, including ASGCT, the program is now the largest of all the projects from the Accelerating Medicines Partnership®.

In July, the FNIH announced the final 14 disease candidates eligible for clinical trial proposals, which Dr. Silverthorn said they’d narrow down to five or six candidates to include in clinical trials. Subject matter experts are working to create a consensus for manufacturing and preclinical requirements. They’ll eventually develop a blueprint of AAV research and clinical and manufacturing protocols that rare disease communities can use. They’ll also work with the FDA to create a publicly available standard regulatory submission and potentially create reference materials so people can point to FDA submissions that have already been completed instead of having to repeat them.

“Really, what we’re hoping to do here, aside from successfully treating 50 or 60 patients through our clinical trials, is to pressure test a process that every clinical trial that comes behind us for a gene therapy in a rare disease could use in order to get therapies to patients,” Dr. Silverthorn said.