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About Krabbe Disease

Krabbe disease is a devastating neurodegenerative disease affecting about 1 -2.5 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the protective cells surrounding the nerves in the brain and throughout the body (peripheral nervous system), eventually leading to the death of these cells. The progressive neuronal cell death manifests over the course of the disease: initially presenting as mental and physical delays in development, muscle weakness and irritability and advancing rapidly to vision and hearing loss and difficulty swallowing and breathing. Infantile Krabbe disease usually results in death by age 2, while Late Infantile Krabbe disease has a more variable course of progressive decline1. There is currently no cure for either form of Krabbe disease. 

The current standard of care for early infantile Krabbe disease, hematopoietic stem cell transplant (HSCT), was pioneered by Dr. Maria Escolar². After HSCT, symptoms of disease have been shown to stabilize by neurological assessment, and survival has improved through correction of the GALC deficiency in the brain. However, HSCT does not effectively correct the GALC deficiency in the peripheral nervous system and as pediatric patients grow, they will progressively lose motor and sensory skills eventually resulting in death³.

About FBX-101

The RESKUE Trial - a Phase 1/2 Clinical Trial for Patients with Krabbe Disease: Screening is open for the Phase 1/2 RESKUE trial. FBX-101 utilizes adeno-associated viral (AAV) gene therapy after hematopoietic stem cell transplant (HSCT) to deliver a functioning copy of the GALC gene, which encodes an enzyme needed to prevent the buildup of psychosine in myelinated cells of both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral myelination deficits, significantly improve the behavioral impairments associated with Krabbe disease in animal models, and drastically improve the lifespan of treated animal models of the disease. The use of transplant and intravenous AAV gene therapy infusion has the potential to overcome some of the immunological safety challenges of traditional AAV gene therapies.

FBX-101, for the treatment of patients with Krabbe disease, is currently in the early phase of clinical development, and the focus of our initial RESKUE Phase 1/2 clinical trial will be an evaluation for safety of FBX-101. These clinical trials are designed and implemented to gain an understanding of the safety and efficacy of FBX-101. Participation in these clinical trials, accepted by the U.S. Food and Drug Administration (FDA), or other regulatory authorities, is the best way for patients to access investigational therapies for their diseases.

More information on the ongoing “RESKUE” clinical trial can be found online at https://www.clinicaltrials.gov/ct2/show/NCT04693598.