NEW YORK – Belief BioMed announced Wednesday that the US Food and Drug Administration has cleared its investigational new drug application enabling it to begin testing its gene therapy candidate for Duchenne muscular dystrophy (DMD).
Shanghai-based Belief will study the investigational gene therapy, dubbed BBM-D101, in an open-label, single-dose clinical trial. BBM-D101 is an adeno-associated virus-based gene therapy designed to deliver an optimized version of the dystrophin gene to muscles throughout the body. In DMD, an X-linked recessive disorder, mutations in the dystrophin gene lead to a loss of the ability to walk, cardiac and respiratory dysfunction, and early death.
In the trial, Belief will examine the safety and efficacy of one dose of BBM-D101 delivered intravenously in DMD patients between 4 and 9 years of age. An investigator-initiated trial of BBM-D101 began in July.
The FDA granted BBM-D101 orphan drug and rare pediatric disease designations in November.
Belief is also developing gene therapies for hemophilia A and hemophilia B. A registrational clinical trial of BBM-H803, its hemophilia A gene therapy, began in January 2024, and the company announced in July that China's National Medical Products Administration accepted its new drug application for BBM-H901 for hemophilia B.
In September, Belief entered into a collaboration with Bayer's Asklepios BioPharmaceutical to develop liver-targeted gene therapies.