Maria Escolar, MD, on Combining HSCT With Gene Therapy in Krabbe Disease


The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.

“Our hypothesis was that if we treat the patients during this time of myeloablation, we will get less antibody response, and possibly even prolong the therapeutic effect of the gene therapy because we wouldn't have the challenge of the antibodies... And until now, we haven't seen any antibodies, which again, is really encouraging.”

Forge Biologics’ FBX-101 (AAVrh.10-GALC), an investigational gene therapy intended to address unmet needs for patients with Krabbe disease, is currently being evaluated in the phase 1/2 RESKUE clinical trial (NCT04693598). Participants in the trial receive a dose of the gene therapy after first being treated with hematopoietic stem cell transplantation (HSCT), the current standard of care for infants with Krabbe disease. 

Maria Escolar, MD, chief medical officer, Forge Biologics, presented data from the first 2 children treated in the trial at the 29th Congress of the European Society of Gene & Cell Therapy (ESGCT), held October 11-14, 2022, in Edinburgh, Scotland.

In an interview with CGTLive, Escolar discussed the rationale behind the combination therapy approach used in the trial, noting that due to the small window of ideal efficacy for the standard of care, it would not be practical to delay HSCT until after gene therapy administration. She also pointed out the potential benefits of myeloablation during HSCT for preventing an immune response during subsequent administration of the gene therapy. Furthermore, Escolar discussed a synergistic benefit to survival from the combination of HSCT and gene therapy seen in preclinical animal models which the clinical trial investigators hope to replicate for patients. She concluded by outlining the future plans for the clinical evaluation of FBX-101 and noted the importance of expanding newborn screening for Krabbe disease, which is currently only conducted in 10 states in the United States.

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. 
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