Allergan (AGN) and Editas Medicine (EDIT) announced initial data from the ongoing natural history study to evaluate patients with Leber congenital amaurosis 10, or LCA10, a rare form of blindness caused by mutations in the CEP290 gene. The companies reported these data in an oral presentation at the Retinal Cell and Gene Therapy, or RCGT. The companies initiated the natural history study to prospectively evaluate and confirm the course of disease in patients and determine the reproducibility and stability of ophthalmic assessments over time. This knowledge has informed the planned Phase 1/2 interventional clinical trial design for AGN-151587, or EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of LCA10. Massachusetts Eye and Ear, an international center for treatment and research and a teaching hospital of Harvard Medical School, is one of seven sites enrolling patients in this natural history study.
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