Tim Miller, PhD, CEO, President, and Co-Founder of Forge Biologics, discusses the phase 1/2 RESKUE study which will evaluate FBX-101 for the treatment of Krabbe disease. This clinical trial is currently recruiting.
Krabbe disease is a neurodegenerative disorder caused by a deficiency of galactosylceramidase. Deficiency of this enzyme impairs the growth and maintenance of myelin. This disorder is also characterized by the abnormal presence of globoid cells. Currently, there is no approved treatment specifically for Krabbe disease. Bone marrow transplants, along with supportive and palliative care are commonly used to manage the disease and its symptoms.
As Dr. Miller explains, FBX-101 is an adeno-associated viral (AAV) gene therapy in development which will be given intravenously after bone marrow transplant. The company hopes this will prevent most of the complications gene therapies often face and lead to a safer, more effective treatment.
To learn more about this study and how to enroll, visit https://www.clinicaltrials.gov/ct2/show/NCT04693598
To learn more about Krabbe disease and other rare neurological disorders, visit checkrare.com/diseases/neurology