Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting

Rhea-AI Summary

Editas Medicine announced the acceptance of two scientific abstracts for the ARVO Annual Meeting, showcasing preclinical data on treatments for Usher Syndrome 2A and retinitis pigmentosa 4 (RP4). The findings indicate successful gene editing for USH2A, restoring protein expression and improving photoreceptor morphology in retinal organoids. Additionally, a dual AAV CRISPR-Cas9 system demonstrated clinically relevant editing levels as a potential RP4 therapy. Editas aims to advance these programs towards clinical application, furthering their mission to develop transformative genomic medicines.

Positive

• Successful gene editing for Usher Syndrome 2A improves protein complex expression.
• Preclinical data shows promise for RP4 treatment using dual AAV CRISPR-Cas9 system.
• Presentation at ARVO highlights significant progress in ocular disease treatments.

Negative

• None.

Preclinical data support novel approaches for the treatment of Usher Syndrome 2A and retinitis pigmentosa 4

CAMBRIDGE, Mass., May 01, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that two scientific abstracts have been accepted for presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting being held virtually May 1 – 7, 2021. The Company is presenting preclinical data on its Usher Syndrome 2A (USH2A) and retinitis pigmentosa 4 (RP4) programs.

Editas Medicine’s presentations at ARVO will include preclinical data demonstrating:

• Gene edited USH2A exon 13 deletion restored USH2 protein complex expression and rescued deficits in photoreceptor morphology in human retinal organoids; and
• Clinically relevant levels of editing using a dual AAV CRISPR-Cas9 system as a therapeutic strategy for the treatment of RP4.

“Editas Medicine is making significant advancements in our efforts to treat ocular diseases, and we look forward to sharing recent progress and preclinical data on our USH2A and RP4 programs this week at the ARVO Annual Meeting,” said Kate Zhang, Vice President, Biological Development, Editas Medicine. “This data further supports our advancing these programs towards the clinic and our belief in the potential for gene editing medicines to transform the lives of those living with inherited retinal diseases.”

The complete list of Editas Medicine presentations is below. Abstracts can be accessed on the ARVO website at https://arvo2021.arvo.org/abstracts.

Oral Presentation:
CRISPR Gene Editing Rescues Deficits in Human USH2A Mutant Retinal Organoids
Date/Time: Tuesday, May 4, 2021, 2:15 p.m. – 3:45 p.m. EDT
Session: Gene Therapy in Ocular Diseases

Poster Presentation:
Advances Towards a Dual AAV CRISPR-Cas9-based ‘Knockout and Replace’ Strategy to Treat Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa
Date/Time: Monday, May 3, 2021, 11:15 a.m. – 1:00 p.m. EDT
Session: Gene Editing and Ocular Therapies

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 (also known as Cpf1) 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. 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, 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 key findings presented by Editas Medicine for Usher Syndrome 2A and RP4?

Editas Medicine presented preclinical data showing gene editing success for USH2A, which restored protein expression and improved retinal morphology. They also demonstrated effective editing for RP4 using a dual AAV CRISPR-Cas9 system.

When is Editas Medicine presenting its findings at ARVO?

Editas Medicine will present at the ARVO Annual Meeting on May 4, 2021, for an oral presentation and on May 3, 2021, for a poster presentation.

What is the significance of Editas Medicine's research for patients with inherited retinal diseases?

The research supports the potential of gene editing therapies to transform treatment options for patients suffering from inherited retinal diseases like Usher Syndrome 2A and RP4.

How does Editas Medicine's dual AAV CRISPR-Cas9 system work for treating RP4?

The dual AAV CRISPR-Cas9 system allows for clinically relevant levels of gene editing, which can potentially correct mutations associated with RP4, paving the way for effective therapeutic strategies.