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
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
Key findings include:
- In human retina explants, EDIT-103 demonstrated highly specific editing with no off-target editing observed after transduction.
- In a mRhohRHO/+ mouse model, EDIT-103 achieved rapid gene editing, with maximal levels at six (6) weeks and sustained, stable editing until end of study (13 weeks).
- In non-human primates (NHPs), EDIT-103 demonstrated nearly 100% knockout of endogenous RHO, and the replacement RHO gene produced over 30% of normal RHO protein levels in the treated area of subretinal injection.
- The EDIT-103 (KO&R)-injected eyes of NHPs showed restoration of RHO expression in the outer segments and retention of normal photoreceptor structure and function compared to the KO-injected eye.
Presentation Session Information: | |
Presentation Title: A Mutation-Independent CRISPR/Cas9-based ‘Knockout and Replace’ Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa | |
Session Title: Gene therapy and other novel therapeutics in ophthalmic diseases 2 | |
Session Date and Time: |
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Presentation Time: |
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Presenter: Zoe (Chi-Hsiu) |
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Location: Denver |
Full details of the
About EDIT-103
EDIT-103 is a CRISPR/Cas9-based experimental medicine in preclinical development for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive form of retinal degeneration. EDIT-103 is administered via subretinal injection and uses two adeno-associated virus (AAV) vectors to knockout and replace mutations in the rhodopsin gene to preserve photoreceptor function. This approach can potentially address more than 150 gene mutations that cause RHO-adRP.
About Editas Medicine
As a leading genome editing company,
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Contacts: MediaCristi Barnett (617) 401-0113 cristi.barnett@editasmed.com InvestorsRon Moldaver (617) 401-9052 ir@editasmed.com
Source: Editas Medicine, Inc.