FBX-101 and the RESKUE Trial: Exploring AAV-Based Gene Therapies 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

REFERENCES

1. Forge Biologics announces regulatory updates from FDA and EMA, accelerating manufacturing and clinical trial momentum. News release. Forge Biologics. September 20, 2021. https://www.businesswire.com/news/home/20210920005324/en/