Forge Biologics Reports Positive FBX-101 Clinical Updates for Patients with Krabbe Disease at WORLDSymposium
Forge Biologics, a genetic medicines development and manufacturing organization, announced today that Maria Escolar, M.D., Chief Medical Officer, will present updated data from the RESKUE Phase 1/2 clinical trial for FBX-101, the Company’s novel AAV gene therapy for the treatment of patients with Krabbe disease, during the 19th Annual WORLDSymposium being held February 22-26, 2023, in Orlando, Florida. Forge has also dosed the first FBX-101 subject in the REKLAIM Phase 1b clinical trial at the University of Michigan Medical Center.
Patients with infantile Krabbe disease have mutations in the gene encoding the lysosomal enzyme galactocerebrosidase (GALC), which is essential for normal metabolism of myelin components. Absence of GALC results in the accumulation of psychosine, a toxic substrate in cells making myelin, which then causes inflammation, rapid nerve demyelination, and progressive deterioration of the central nervous system (CNS) and peripheral nervous system (PNS). This results in progressive motor disease and often early death of patients by two years of age.
“We are encouraged by the continued safety and efficacy observed in FBX-101 treated patients. Notably, some of the patients were identified by newborn screening, which enabled early disease intervention,” stated Dr. Escolar. “All patients to date have exhibited normal motor function and brain development six and 12 months post-FBX-101 administration, which would not be anticipated in the absence of systemic gene transfer of the GALC gene. We also observed up to 170-fold and 10-fold increases in plasma and cerebrospinal fluid (CSF) GALC levels, respectively, during the six and 12 months follow-up milestones, demonstrating sustained, high expression of the GALC transgene. Importantly, no treatment-related serious adverse events, liver enzymes elevations, nor development of anti-AAVrh10 antibodies have been observed after FBX-101 systemic administration. These results are very encouraging and provide hope for the families impacted by Krabbe.”
Forge has also dosed one subject in the REKLAIM Phase 1b clinical trial for FBX-101 at the University of Michigan Medical Center. The REKLAIM trial is evaluating the safety and efficacy of FBX-101 in asymptomatic infantile or symptomatic late infantile Krabbe disease patients that received HSCT at least 90 days before FBX-101 administration. Children assessed in REKLAIM have received the standard of care (HSCT) and are partially or fully immuno-competent, but are at risk of developing progressive peripheral nerve disease.
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