Maria Escolar, MD, on Treating Krabbe Disease With Investigational Gene Therapy FBX-101


The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.

“I've been working on this disease for over 20 years and it is extremely exciting to see patients that are walking at this age. It is an amazing experience to be able to go from all the preclinical work to now seeing the effects in patients. I'm very excited and hopefully results continue to be good in the future.”

Infantile Krabbe disease (IKD) is a rare genetic disorder that leads to deficiency in the galactocerebrosidase (GALC) enzyme and build up of its substrate psychosine. If left untreated, patients typically do not survive past 2 years of age because of demyelination caused by psychosine accumulation. The current standard of care for IKD is umbilical cord blood transplantation (UCBT). UCBT can help address the disease’s effect on the central nervous system, but even after UCBT patients typically experience progressive peripheral neuropathy.

Forge Biologics’ FBX-101, an investigational adeno-associated virus (AAV) vector-based gene therapy, is intended to address unmet needs for patients with IKD by delivering a functional copy of the GALC gene. It is currently being assessed in the phase 1/2 RESKUE clinical trial (NCT04693598), in which it is administered after patients receive standard treatment with UCBT. This treatment approach, which is unusual for a gene therapy clinical trial, is being carried out with the expectation that prior UCBT could help prevent immune responses to the AAV vector that could interfere with efficacy. Maria Escolar, MD, the chief medical officer of Forge Biologics, recently presented updated data from RESKUE at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16 to 20, in Los Angeles, California.

In an interview with CGTLive™, Escolar discussed the rationale behind the study and gave an overview of the key results. She noted that in the 2 patients who have been treated with FBX-101 in RESKUE so far, antibodies to the AAV vector have not increased beyond baseline. Escolar also highlighted that upon follow-up the treated patients showed motor function in the normal range and were observed walking independently.

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1. Escolar ML, Vander Lugt M, Poe MD, et al. First in human RESKUE phase 1/2 clinical trial of intravenous FBX-101 (AAVrh10. hGALC) administered after immune and myeloablation for unrelated umbilical cord blood transplantation prevented immune responses, increased GALC activity, restored normal brain development, and normalized motor function in patients with infantile Krabbe disease. Presented at: American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting. May 16-20, 2023; Los Angeles, CA. Abstract #359
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