Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the Association for Research in Vison and Ophthalmology Conference
Studies in non-human primates demonstrated nearly 100% gene editing knockout of endogenous RHO gene and more than 30% replacement protein levels
Treated eyes showed morphological and functional photoreceptor preservation
EDIT-103 advancing towards IND-enabling studies
Excerpt from the Press Release:
CAMBRIDGE, Mass., May 04, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced ex vivo and in vivo preclinical data for its experimental medicine EDIT-103 for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). The Company reported these data in an oral presentation today at the Association for Research in Vison and Ophthalmology (ARVO) Annual Meeting in Denver.
EDIT-103 is a mutation-independent CRISPR/Cas9-based, dual AAV5 vectors “knockout and replace” (KO&R) therapy to treat RHO-adRP. This approach has the potential to treat any of over 150 dominant gain-of-function rhodopsin mutations that cause RHO-adRP with a one-time subretinal administration.
“These promising data in non-human primates and mice demonstrate the potential of EDIT-103 to efficiently remove the defective RHO gene responsible for RHO-adRP, and, critically, replace it with a sufficient level of RHO to preserve photoreceptor structure and functions,” said Mark S. Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “The program is progressing toward the clinic, and we expect to start IND-enabling studies by the end of 2022. EDIT-103, which uses a dual AAV gene editing approach, also provides initial proof of concept for the treatment of other autosomal dominant disease indications where a gain of negative function needs to be corrected, and we are evaluating the development of additional programs.”
Key findings include:
- In human retina explants, EDIT-103 demonstrated highly specific editing with no off-target editing observed after transduction.
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