Maria Escolar, MD, on Addressing Unmet Needs in Krabbe Disease With Gene Therapy


The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.

“I think the biggest advantage is that we will probably be addressing the peripheral nerves and the spinal cord, which is something we know that the standard of care doesn't address. [In] preclinical studies … we were able to correct the neuropathy, both pathologically and functionally as measured by nerve conduction velocities in the dogs. So, we're hoping that that we will see the same thing in patients over time.”

While the current standard of care for Krabbe disease, which includes umbilical cord blood transplant, is able to address symptoms in the brain, it does not address the peripheral neuropathy and the demyelination of the spinal cord caused by the disease. As such, there remains significant unmet need for the patient population.

At the 29th Congress of the European Society of Gene & Cell Therapy (ESGCT), held October 11-14, 2022, in Edinburgh, Scotland, Maria Escolar, MD, chief medical officer, Forge Biologics, presented data from the phase 1/2 RESKUE clinical trial (NCT04693598) of FBX-101, an investigational gene therapy intended to address unmet needs for patients with Krabbe disease.

In an interview with CGTLive, Escolar briefly discussed the limitations of the current standards of care for Krabbe disease and the remaining unmet needs for the patient community. She then gave a topline overview of the data from the 2 patients treated so far in the RESKUE clinical trial that were presented at ESGCT. The data included several promising signs of efficacy, such as an 80-fold increase in the galactocerebrosidase (GALC) enzyme measured in the plasma, and improvements in white matter integrity. In terms of safety, no serious adverse events related to treatment were reported. Escolar concluded by discussing the potential advantages of FBX-101 over the current standard of care.

Forge Biologics announces updated positive clinical data in RESKUE, a novel phase 1/2 gene therapy trial for patients with Krabbe disease. News release. Forge Biologics. October 11, 2022. 
Related Videos
David Suhy, PhD, the cofounder and chief scientific officer of Earli
Deepak L. Bhatt, MD, MPH, MBA, on Incorporating AI into Genetic Research for Cardiovascular Disease
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
Jonathan W. Weinsaft, MD, on Integrating Genetic Research into Cardiovascular Medicine
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
Vanee Pho, PhD, the senior director of product management, cell and gene therapy, at Mission Bio
Michael Wang, MD, a professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center
Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology
Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations
Rawan Faramand, MD, an assistant professor at Moffit Cancer Center
© 2024 MJH Life Sciences

All rights reserved.