Editas Medicine, Inc.
Studies successful non-human primates demonstrated astir 100% cistron editing and knockout of endogenous RHO cistron and much than 30% replacement macromolecule levels utilizing a dual vector AAV approach
Treated eyes showed morphological and functional photoreceptor preservation
EDIT-103 advancing towards IND-enabling studies
CAMBRIDGE, Mass., Oct. 13, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a starring genome editing company, contiguous announced ex vivo and in vivo preclinical information supporting its experimental medicine EDIT-103 for the attraction of rhodopsin-associated autosomal ascendant retinitis pigmentosa (RHO-adRP). The Company reported these information successful an oral presumption contiguous astatine the European Society of Gene and Cell Therapy 29th Annual Meeting successful Edinburgh, Scotland, UK.
EDIT-103 is simply a mutation-independent CRISPR/Cas9-based, dual AAV5 vectors “knockout and replace” (KO&R) therapy to dainty RHO-adRP. This attack has the imaginable to dainty immoderate of implicit 150 ascendant gain-of-function rhodopsin mutations that origin RHO-adRP with a one-time subretinal administration.
“These promising preclinical information show the imaginable of EDIT-103 to efficiently region the defective RHO cistron liable for RHO-adRP portion replacing it with an RHO cistron susceptible of producing capable levels of RHO to sphere photoreceptor operation and functions. The programme is progressing towards the clinic,” said Mark S. Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “EDIT-103 uses a dual AAV cistron editing approach, and besides provides archetypal impervious of conception for the attraction of different autosomal ascendant illness indications wherever a summation of antagonistic relation needs to beryllium corrected.”
Key findings include:
In quality retina explants, EDIT-103 demonstrated highly circumstantial editing with nary off-target editing observed aft transduction.
In a mRhohRHO/+ mouse model, EDIT-103 achieved accelerated cistron editing, with maximal levels astatine six (6) weeks and sustained, unchangeable editing until extremity of survey (13 weeks).
In non-human primates (NHPs), EDIT-103 demonstrated astir 100% knockout of endogenous RHO, and the replacement RHO cistron produced implicit 30% of mean RHO macromolecule levels successful the treated country of subretinal injection.
The EDIT-103 (KO&R)-injected eyes of NHPs showed restoration of RHO look successful the outer segments and retention of mean photoreceptor operation and relation 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 |
Editas scientists are besides presenting EDIT-202 preclinical information successful a Poster session: |
Presentation Title: EDIT-202, an AsCas12a and SLEEK™ gene-edited iPSC-derived NK cell therapy maintains prolonged persistence, precocious cytotoxicity, and enhanced in vivo control of solid tumors. |
Full details of the Editas Medicine presentations tin beryllium accessed successful the Posters & Presentations conception connected the Company’s website.
About EDIT-103
EDIT-103 is simply a CRISPR/Cas9-based experimental medicine successful preclinical improvement for the attraction of rhodopsin-associated autosomal ascendant retinitis pigmentosa (RHO-adRP), a progressive signifier of retinal degeneration. EDIT-103 is administered via subretinal injection and uses 2 adeno-associated microorganism (AAV) vectors to knockout and regenerate mutations successful the rhodopsin cistron to sphere photoreceptor function. This attack tin perchance code much than 150 cistron mutations that origin RHO-adRP.
About Editas Medicine
As a starring genome editing company, Editas Medicine is focused connected translating the powerfulness and imaginable of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for radical surviving with superior diseases astir the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a wide people of diseases. Editas Medicine is the exclusive licensee of Harvard and Broad Institute’s Cas9 patent estates and Broad Institute’s Cas12a patent property for quality medicines. For the latest accusation and technological presentations, delight sojourn www.editasmedicine.com.
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