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

Rhea-AI Summary

Editas Medicine reported promising preclinical results for its experimental medicine EDIT-103, aimed at treating rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). The study, presented at the European Society of Gene and Cell Therapy meeting, showcased nearly 100% gene editing in non-human primates with more than 30% of replacement protein levels achieved. This CRISPR/Cas9-based therapy demonstrated effective knockout and replacement of the defective RHO gene with indications of preserved photoreceptor function, advancing towards IND-enabling studies.

Positive

• Nearly 100% knockout of endogenous RHO gene in non-human primates.
• More than 30% replacement protein levels achieved.
• Demonstrated morphological and functional preservation of photoreceptors.

Negative

• None.

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

CAMBRIDGE, Mass., Oct. 13, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced ex vivo and in vivo preclinical data supporting 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 European Society of Gene and Cell Therapy 29^th Annual Meeting in Edinburgh, Scotland, UK.

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 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 gain of negative 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 mRho^hRHO/+ 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: October 13, 2022, from 8:30 a.m. to 10:45 a.m. BST (3:30 – 5:45 a.m. EDT) Presenter: Mariacarmela Allocca, Director, In Vivo Pharmacology &Toxicology, In Vivo Gene Editing, Editas Medicine Location: Edinburgh International Conference Centre, Sidlaw Auditorium

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: October 13, 2022, from 5:30 – 7:15 p.m. BST (12:30 – 2:15 p.m. EDT) Presenter: Samia Khan, Senior Scientist II, Ex Vivo Pharmacology, Stem Cell Therapies, Editas Medicine Location: Edinburgh International Conference Centre

Full details of the Editas Medicine presentations can be accessed in the Posters & Presentations section on the Company’s website.

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, 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 living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Harvard and Broad Institute’s Cas9 patent estates and Broad Institute’s Cas12a patent estate for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.

Forward-Looking Statements
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

FAQ

What are the results of the EDIT-103 study presented by Editas Medicine?

The EDIT-103 study reported nearly 100% gene editing and more than 30% replacement protein levels in non-human primates, indicating effective treatment potential for RHO-adRP.

What is the purpose of EDIT-103?

EDIT-103 is designed to treat rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP) by using a CRISPR/Cas9-based approach to knockout and replace defective RHO genes.

What are the next steps for EDIT-103?

EDIT-103 is advancing towards IND-enabling studies following successful preclinical data.

When was the EDIT-103 data presented?

The data for EDIT-103 was presented on October 13, 2022, at the European Society of Gene and Cell Therapy Annual Meeting.

What is the significance of the study's findings for Editas Medicine?

The findings suggest that EDIT-103 has potential for treating over 150 mutations causing RHO-adRP, with implications for future gene therapies.