Forge is working on a gene therapy treatment invented at UPMC but has covered its bases by targeting viral vector production for third-party clients.

Forge Biologics, a US-based gene therapy developer and manufacturer leveraging University of Pittsburgh Medical Center (UPMC) research, has completed a $40m series A round led by Perceptive Xontogeny Venture Fund with backing from Drive Capital.
Founded earlier in 2020, Forge Biologics produces viral vector-driven drugs from a 50-litre capacity manufacturing plant in Ohio on behalf of pharmaceutical sector clients, in addition to progressing its own pipeline for rare indications including the neurological disorder Krabbe disease.
Krabbe disease develops in babies and typically causes mortality at just two years old. Forge’s drug candidate, FBX-101, deposits its payload via an adeno-associated viral (AAV) vector to counter mutations in the GALC gene, in conjunction with umbilical cord blood transfusions administered to the infant.
The series A cash will go to building out Forge’s virus manufacturing plant to 500 litres of capacity by 2021, initially to serve third-party outsourcing but later extending into regulatory-standard production of its own drug candidates.
FBX-101 builds on work undertaken in the lab of Maria Escolar, director for the study of neurodevelopment in rare disorders at the paediatric neurology unit of UPMC Children’s Hospital of Pittsburgh.
Timothy Miller, president and co-founder of Forge Biologics, said: “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.
“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.”