Forge Biologics is located in a 175,000 ft2 facility in Columbus, Ohio. This facility, referred to as The Hearth, is home to a custom-designed cGMP facility dedicated to AAV viral vector manufacturing, currently capable of up to 50L scale research and toxicology grade AAV manufacturing. By mid-2021, 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.
“Forge is a true gene therapy development engine, bringing together access to viral vector design expertise, quality AAV manufacturing, a management team with significant gene therapy experience, and an exciting pipeline of new therapies for patients,” said Timothy J. Miller, Ph.D., co-founder, president and CEO of Forge Biologics. “As gene therapy programs continue to demonstrate clinical success, the need for AAV manufacturing has never been greater. Our mission is to enable access to potentially life-saving therapies and help bring them from idea into reality. Furthermore, we are pleased to welcome Perceptive Advisors and their PXV Fund as lead investor in this financing and look forward to benefiting from their deep expertise in the gene therapy and rare disease space.”
In addition to its CDMO capabilities, Forge is developing its own pipeline of innovative gene therapies aimed at treating patients with genetic diseases. On July 21, Forge announced its lead program, a novel AAV and umbilical cord transplant combination approach to treat infantile Krabbe disease, a 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.
New Chief Medical Officer
The combination treatment was pioneered at the University of Pittsburgh in the lab of Maria Escolar, M.D., who joins Forge as chief medical officer. Escolar is currently a professor of Pediatrics and director, Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at University of Pittsburgh and brings a wealth of rare disease and clinical gene therapy experience to the team.
“It is a true privilege to join Forge as chief medical officer to advance FBX-101 as an important treatment for Krabbe patients. The development approach of FBX-101 combines an Umbilical Cord Bone marrow Transplant (UCBT) and AAV gene therapy to comprehensively address both central and peripheral nerve degeneration associated with this disease,” said Escolar, who also holds equity in Forge. “We believe our combination approach has the potential to address some of the immunological safety challenges recently observed in gene therapy clinical trials.”
Researchers recently presented data at the American Society for Gene and Cell Therapy annual meeting demonstrating that this combination approach significantly improved efficacy compared to a single AAV administration alone in canines affected with Krabbe disease.
In conjunction with the Series A financing, Chris Garabedian, manager of the PXV Fund for Perceptive Advisors and CEO of Xontogeny will be joining the Forge Board of Directors as chairman.
“We are excited to have Forge represent the PXV Fund’s first investment in the gene therapy space as we are confident that the Forge team’s previous gene therapy experience will make them a capable and trusted partner for gene therapy drug developers,” said Garabedian. “Given the overwhelming demand for a CDMO that can successfully take a gene therapy program from inception through commercialization, we believe Forge serves the industry in filling this important gap and has the team that can do this successfully.”