Six months after departing Abeona, Tim Miller, Ph.D., is on his next chapter: Forge Biologics, a gene therapy biotech that’s providing manufacturing and development services to other companies while working on its own pipeline. To fuel this dual mission, Forge picked up $40 million from the Perceptive Xontogeny Venture Fund and Drive Capital.
Forge is the brainchild of Miller and a “confluence of gene therapy experts” who saw a widening gap between the growth of the industry’s gene therapy pipeline and its capacity to actually manufacture the treatments, Miller told Fierce Biotech.
Those experts include Jaysson Eicholtz, who previously oversaw gene therapy manufacturing for Nationwide Children’s Hospital; Erandi De Silva, Ph.D., who led program management at Myonexus Therapeutics; and Maria Escolar, M.D., the University of Pittsburgh professor who developed Forge’s lead program. The trio are Forge’s chief operating officer, chief strategy officer and chief medical officer, respectively.
“We came together with the idea that if we did this hybrid play, what you end up with is a development engine that helps secure the needs of additional clients, serving as an end-to-end solution provider, but also being able to do that for ourselves and build out our pipeline through that engine capacity,” Miller said.
Like other companies with a hub-and-spoke model, Forge comprises a “core” that offers vector design know-how, adeno-associated virus (AAV) vector manufacturing and an experienced management team, and will set up “subsidiary entities” around therapeutics, Miller said.
Forge’s 175,000-square-foot manufacturing site is already up and running, but the series A funding will boost its capacity and enable cGMP manufacturing by the middle of 2021.
The cash will also propel Forge’s lead program, FBX-101, which combines an AAV gene therapy with an umbilical cord bone marrow transplant. Forge is developing it for infants with Krabbe disease, a neurodegenerative disorder that destroys myelin, the fatty substance that coats and protects nerve fibers in the brain and nervous system. The combination approach could address a challenge in using AAVs to deliver gene therapies: Some people have developed a natural immunity to this type of virus, rendering an AAV gene therapy ineffective.
“The transplant … is solving for two problems at the same time,” Miller said. “You’re not going to see an immune response against the protein expressed from the gene therapy approach. And you’re able to remove a lot of the immune response to AAV capsids overall.”