Erandi De Silva, PhD, co-founder and senior vice president of product development at Forge Biologics, discusses the dual approach of her company in manufacturing and also developing adeno-associated viral (AAV) -based gene therapies, including FBX-101 for Krabbe disease.
"The Krabbe disease program at Forge is a really unique intervention. This is coupling a hematopoietic stem cell transplant with an AAV-based gene therapy...The preclinical data has been strong."
A key challenge in the development of gene therapies is often manufacturing and scalability. Forge Biologics takes a dual approach, combining both a therapeutics division and a manufacturing arm in the hopes of overcoming that obstacle.
Joining CTGLive is Erandi De Silva, PhD, co-founder and senior vice president of product development at Forge, to discuss her company's approach and also the adeno-associated viral- (AAV) based gene therapies in development, focusing in particular on its open trial for Krabbe disease.
Forge's RESKUE trial is a phase 1/2 clinical trial for patients with Krabbe, a rare neurodegenerative disorder causing progressive demyelination of nerves in the body. The company's agent, FBX-101, couples hematopoietic stem cell transplant with an AAV-based gene therapy to deliver a functioning copy of the GALC gene.
The EMA granted orphan drug designation to FBX-101 in October.
“FBX-101 is the culmination of over 20 years of scientific research to understand the challenges faced by patients with progressive disease after hematopoietic stem cell transplantation, the current standard of care,” said Maria Escolar, MD, chief medical officer, Forge Biologics, in the statement.1
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.