“We are very pleased that the FDA granted Orphan Drug Designation to GBC0905 as an accomplishment for our team and an important milestone in our journey toward bringing our lead product candidate to the clinic. We anticipate GBC0905 to be the first targeted therapy for patients suffering from FSHD, a relentless and debilitating disease,” said Dr. Uli Schmidt, President of Genea Biocells. “The Orphan Drug Designation also validates our skeletal muscle technology which is a highly useful platform for modeling any number of other myogenic indications as we expand our pipeline.”
Genea Biocells’ lead scientist for the FSHD program, Amanda Rickard, added, “Our targeted therapeutic candidate GBC0905 potently suppresses DUX4 function, prevents the activation of DUX4 target genes, and protects affected skeletal muscle cells in a dose-dependent fashion and, importantly, without affecting normal myogenesis. Aberrant DUX4 expression is the underlying cause of the disease and results in muscle fiber death. The FSHD field generally believes that blocking DUX4 will be curative. We are looking forward to thorough and expeditious development of this much-needed therapy.”
Currently, Genea Biocells is seeking capital to advance GBC0905 to clinical trials, complete preclinical work on their Spinal Muscular Atrophy (SMA) program, and expand the pipeline to other orphan muscle diseases. Under the U.S. Orphan Drug Act, the FDA grants Orphan Drug Designation status to novel therapeutics for rare diseases, defined as those affecting fewer than 200,000 people in the U.S. All of Genea Biocells’ product candidates are ultimately expected to benefit from Orphan Drug Designation. Advantages include tax credits for qualified clinical trials costs and seven years of market exclusivity upon regulatory product approval.
About FSHD
Facioscapulohumeral Dystrophy (FSHD) is an inheritable muscle disease affecting up to 1:8,000 people. There is no cure or treatment strategy for patients with FSHD. This debilitating disease slowly consumes skeletal muscle, robbing people of the active, healthy, and independent years of their lives. It is caused by the sporadic de-repression of a toxic gene, DUX4, in the muscles of FSHD patients.
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Contacts
Genea Biocells US Inc.
Debra Bressaw, 858 281 4840
