Studies in non-human primates have demonstrated nearly 100% gene editing and inactivation of the endogenous RHO gene and over 30% replacement protein levels using a dual-vector AAV approach
The treated eyes showed morphological and functional preservation of the photoreceptors
EDIT-103 is progressing towards studies enabling IND
CAMBRIDGE, Mass., Oct. 13, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced ex-vivo and live preclinical data supporting its investigational drug EDIT-103 for the treatment of autosomal dominant rhodopsin-associated retinitis pigmentosa (RHO-adRP). The Company reported these data in an oral presentation today at the European Society for Gene and Cell Therapy 29e Annual meeting in Edinburgh, Scotland, UK.
EDIT-103 is a CRISPR/Cas9 independent AAV5 dual vector knock-out and replace (KO&R) therapy for treating RHO-adRP. This approach has the potential to treat any of over 150 dominant rhodopsin gain-of-function mutations that cause RHO-adRP with a single subretinal delivery.
“These promising preclinical data demonstrate the potential of EDIT-103 to effectively eliminate 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 function. . The program is moving into the clinic,” said Mark S. Shearman, Ph.D., executive vice president and chief scientific officer, Editas Medicine. “EDIT-103 uses a dual AAV gene editing approach and also provides initial proof of concept for the treatment of other autosomal dominant disease indications where a negative gain of function needs to be corrected.”
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 peak levels at six (6) weeks and sustained and stable editing through the end of the study (13 weeks).
In non-human primates (NHPs), EDIT-103 demonstrated nearly 100% removal of endogenous RHO, and the replacement RHO gene produced over 30% of normal levels of RHO protein in the treated area of the subretinal injection.
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.
Pitch session information:
Presentation title: A mutation-independent CRISPR/Cas9-based knockout and replacement strategy to treat rhodopsin-associated autosomal dominant retinitis pigmentosa
Editas scientists also present the preclinical data of EDIT-202 in a Poster session:
Presentation title: EDIT-202, an iPSC-derived NK cell therapy modified with AsCas12a and SLEEK™ maintains prolonged persistence, high cytotoxicity and improved in vivo control of solid tumors.
All the details of the Editas Medicine presentations are accessible in the Posters and presentations section on the Company’s website.
EDIT-103 is an investigational CRISPR/Cas9-based drug in preclinical development for the treatment of autosomal dominant rhodopsin-associated retinitis pigmentosa (RHO-adRP), a progressive form of retinal degeneration. EDIT-103 is administered by subretinal injection and uses two adeno-associated virus (AAV) vectors to eliminate and replace mutations in the rhodopsin gene to preserve photoreceptor function. This approach can potentially treat more than 150 genetic mutations that cause RHO-adRP.
About Edited by Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people with serious illnesses all over the world. Editas Medicine aims to discover, develop, manufacture and commercialize transformative, sustainable and precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of the Harvard and Broad Institute Cas9 patent domains and the Broad Institute Cas12a patent domain for human medicines. For the latest scientific information and presentations, please visit www.editasmedicine.com.
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