11.16.18
Source: 4D Molecular Therapeutics
Gene therapy company 4D Molecular Therapeutics (4DMT) announced that the FDA has granted orphan drug designation to 4D-110 for the treatment of choroideremia.
Choroideremia (CHM) is an X-linked monogenic recessive disease (caused by mutation of the REP-1 gene) starting with reduced night vision and progressive loss of peripheral vision and eventually leading to loss of visual acuity and blindness.
“Our product for CHM patients, 4D-110, will be administered by intravitreal injection, and is designed to result in widespread expression of the REP-1 protein within cells in the retina,” Peter Francis, SVP Clinical, Translational R&D Program Leader, Retina Therapeutic Area, said in a company news release. “In contrast to other AAV gene therapy agents for CHM patients,
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