Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene and Cell Therapy Annual Meeting
Studies in non-human primates demonstrated nearly 100% gene editing and knockout of endogenous RHO gene and more than 30% replacement protein levels using a dual vector AAV approach
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 preclinical data demonstrate the potential of EDIT-103 to efficiently remove the defective RHO gene responsible for RHO-adRP while replacing it with an RHO gene capable of producing sufficient levels of RHO to preserve photoreceptor structure and functions. The program is progressing towards the clinic,” 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 and epigenetic editing II
Session Date and Time:
Presenter: Mariacarmela Allocca, Director, In
Location: Edinburgh International Conference Centre,
|Editas scientists are also presenting EDIT-202 preclinical data in a Poster session:|
|Presentation Title: EDIT-202, an AsCas12a and SLEEK™ gene-edited iPSC-derived NK cell therapy maintains prolonged persistence, high cytotoxicity, and enhanced in vivo control of solid tumors.
Session Date and Time:
Location: Edinburgh International Conference Centre
Full details of the
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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Source: Editas Medicine, Inc.