Krabbe Disease Trial Update

Press Release from Forge Biologics

Forge Biologics Reports Positive FBX-101 Clinical Updates for Patients with Krabbe Disease

Press Release: Forge Biologics Press Release

“It is important to highlight that the early data in the RESKUE trial demonstrates that intravenous delivery of FBX-101 after HSCT has been safe and well tolerated 6 and 12 months post-administration. Notably, the data indicates an absence of antibody response against the systemically delivered AAV, and significantly increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid (CSF). All patients enrolled to date have shown improved motor activity and brain development, which would not be anticipated in the absence of systemic gene transfer of the GALC gene.

Forge has also launched and treated one patient in the REKLAIM trial for FBX-101 at the University of Michigan Medical Center. REKLAIM is a Phase 1b clinical trial currently enrolling patients to investigate the safety and efficacy of FBX-101 and is administered to patients who received HSCT at least 90 days previously and is asymptomatic with infantile Krabbe disease or early symptomatic with late infantile Krabbe disease. The purpose of REKLAIM is to offer this potential therapy to a larger population of children with Krabbe disease, targeting children that received HSCT as the standard of care, but are developing or are at high risk of developing progressive peripheral nerve disease.”

RESKUE Trial: ClinicalTrials Page

REKLAIM Trial: ClinicalTrials Page

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