Ohio-based startup Forge Biologics closed a $40 million Series A financing round that will be used to expand manufacturing capabilities and support the clinical development of a gene therapy program for rare diseases, including the typically fatal Krabbe disease.

Forge Biologics is a viral vector contract development manufacturing organization (CDMO) and therapeutics developer. The company plans to use proceeds from the funding round to expand its adeno-associated virus (AAV) manufacturing capabilities, as well as its gene therapy pipeline. Its lead program is FBX-101, a novel AAV and umbilical cord transplant combination approach to treat infantile Krabbe disease, a devastating neurodegenerative disorder. Krabbe disease has a predicted incidence of about 1 in 12,000 people in the U.S. with symptoms developing in babies and rapidly progressing to death by age two. Forge anticipates FBX-101 will enter the clinic later this year.

FBX-101 was initially developed in the laboratory of Maria Escolar, a professor of pediatrics and director of the Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at the University of Pittsburgh. Escolar will serve as chief medical officer of Forge Biologics. The combination of AAV gene therapy and umbilical cord bone marrow transplant addresses both central and peripheral nerve degeneration associated with Krabbe disease, she said. Recent data showed the combination approach significantly improved efficacy compared to a single AAV administration alone in canines affected with Krabbe disease.

The companys manufacturing facility, known as The Hearth, will host end-to-end cGMP AAV manufacturing services at 500L scale, enabling biotech and pharma clients the ability to accelerate their gene therapy programs from pre-clinical development through clinical and commercial stage manufacturing.

Timothy J. Miller, president and chief executive officer of Forge Biologics, said the young company is a true gene therapy development engine. With AAV manufacturing, its pipeline and a strong leadership team, Miller said the company is ready to bring life-saving therapies for rare diseases into reality.

In addition to Miller, who previously served as CEO of Abeona Therapeutics, and Escolar, Forges leadership team includes Chief Operations Officer Jaysson Eicholtz and Chief Strategy Officer Erandi De Silva. Eicholtz was previously responsible for Nationwide Childrens Hospitals gene therapy manufacturing facility, and Silva was previously head of Program Management at Myonexus Therapeutics.

The Series A financing round was led by Perceptive Xontogeny Venture Fund with participation from Drive Capital. Chris Garabedian, CEO of Xontogeny, will join the Forge Board of Directors as its chairman.

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Forge Biologics Launches with $40 Million to Manufacture and Develop Gene Therapies - BioSpace