ASGCT24: Translational Research Powering Potentially Transformative Therapies

ASGCT translational research panel

Pictured: Panelists on stage at ASGCT (L to R: NHLBI’s Gary Gibbons, NCATS’ Joni Rutter, Amy Jenkins of Advanced Research Projects Agency for Health, NINDS’ Amir Tamiz and ASGCT President Jeffrey Chamberlain)/Photo by Greg Slabodkin

Advancing translational research of novel therapies is a priority for both the scientific community and the National Institutes of Health, according to stakeholders at the 2024 American Society of Gene & Cell Therapy (ASGCT) annual meeting.

In a Friday fireside chat with officials from the National Institute of Health (NIH) at ASGCT 2024, ASGCT President Jeffrey Chamberlain described translational research as the “critical nexus” between basic science and clinical investigation.

“This is an area where we’re trying to bridge the gap between fundamental new discoveries and their applications to improve human health,” Chamberlain said.

The session’s agenda highlighted some of the pivotal basic science and translational studies, as well as the researchers who are making breakthroughs when it comes to transformative genetic therapies.

Gary Gibbons, director of the National Heart, Lung, and Blood Institute (NHLBI) at NIH, touted his organization’s launch of the Cure Sickle Cell Initiative in 2018 as a collaborative research effort to accelerate the development of gene therapies to cure sickle cell disease (SCD).

Gibbons said that new FDA-approved curative gene therapies for SCD— Vertex Pharmaceuticals’ CRISPR-based Casgevy and bluebird bio’s gene therapy Lyfgenia—are a case study in the value of translational research for creating transformative treatments for patients.

“We had folks from Vertex and bluebird bio at the table from the beginning, as well as our colleagues at FDA,” Gibbons commented. “Because we knew at that stage we were defining a regulatory pathway toward that end.”   

Chamberlain called Casgevy and Lyfgenia “possibly the most exciting development ever” in the field of gene therapies.

Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS) at NIH, said that one of her organization’s primary missions is to re-engineer the translational research process so new treatments and cures for diseases can reach patients faster. “Our mission is essentially to understand how to traverse that pipeline better, faster, more effectively and efficiently,” Rutter said.

NCATS has taken credit for enabling a path to market for ReveraGen BioPharma’s Duchenne muscular dystrophy therapy, which was approved by the FDA in late 2023. Another NCATS-led program Rutter pointed to is the Platform Vector Gene Therapy (PaVe-GT) pilot project. Launched in 2019, the PaVe-GT pilot aims to test the hypothesis of whether a platform vector approach will increase efficiency in preclinical testing and clinical trial start-up.

Rutter also pointed to the NCATS-led Bespoke Gene Therapy Consortium (BGTC), a public-private collaboration involving NIH, FDA, industry and patient groups intended to help accelerate the delivery of AAV-based gene therapies for rare diseases. With BGTC, “we’re establishing how to optimize the biology and the studies behind developing the AAV vectors and then we’re also optimizing the manufacturing,” Rutter said.

Greg Slabodkin is the news editor at BioSpace. You can reach him at Follow him on LinkedIn.

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