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CRISPR Therapeutics to Present Oral Presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting

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CRISPR Therapeutics is set to present an oral presentation at the American Society of Gene & Cell Therapy 2024 Annual Meeting, showcasing their lipid nanoparticle approach for ocular editing. The presentation will focus on the potential treatment for glaucoma by targeting the MYOC gene, with promising results of up to 95% editing and 85% protein knockdown in human cells.
CRISPR Therapeutics è pronta a presentare un intervento orale al Congresso annuale 2024 della American Society of Gene & Cell Therapy, esponendo il loro approccio con nanoparticelle lipidiche per l'editing oculare. La presentazione si concentrerà sulla possibile cura del glaucoma tramite il targeting del gene MYOC, mostrando risultati promettenti con fino al 95% di modifica genetica e l'85% di riduzione proteica nelle cellule umane.
CRISPR Therapeutics está programada para realizar una presentación oral en la Reunión Anual 2024 de la Sociedad Americana de Terapia Génica y Celular, donde mostrarán su enfoque con nanopartículas lipídicas para la edición ocular. La presentación se centrará en el tratamiento potencial del glaucoma mediante la manipulación del gen MYOC, con resultados prometedores de hasta un 95% de edición y un 85% de reducción de proteínas en células humanas.
CRISPR Therapeutics가 2024년 미국 유전 및 세포 치료학회 연례 회의에서 구두 발표를 할 예정이며, 이는 안구 편집을 위한 지질 나노입자 접근법을 선보일 것입니다. 이 발표는 MYOC 유전자를 목표로 하는 녹내장의 잠재적 치료에 초점을 맞추며, 인간 세포에서 최대 95%의 편집 및 85%의 단백질 저하라는 유망한 결과를 보여줄 예정입니다.
CRISPR Therapeutics est prête à présenter une exposé oral à la réunion annuelle 2024 de la Société Américaine de Thérapie Génique et Cellulaire, mettant en avant leur approche des nanoparticules lipidiques pour l'édition oculaire. La présentation se concentrera sur le traitement potentiel du glaucome en ciblant le gène MYOC, avec des résultats prometteurs atteignant jusqu'à 95% d'édition et 85% de réduction de protéines dans les cellules humaines.
CRISPR Therapeutics plant, eine mündliche Präsentation auf dem jährlichen Treffen 2024 der American Society of Gene & Cell Therapy zu halten, die ihren Ansatz mit Lipid-Nanopartikeln für die Augenbearbeitung vorstellt. Der Fokus der Präsentation wird auf der potenziellen Behandlung von Glaukom durch gezielte Bearbeitung des MYOC-Gens liegen, mit vielversprechenden Ergebnissen von bis zu 95% Gen-Editing und 85% Proteinabbau in menschlichen Zellen.
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ZUG, Switzerland and BOSTON, April 22, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced an oral presentation highlighting the Company's lipid nanoparticle (LNP) approach for ocular editing will be presented at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually.

The abstract describes our proprietary capabilities to deliver to and edit genes in the eye, opening a potential new focus area. Multiple LNPs as well as modified gRNAs and mRNAs were screened to achieve maximal editing in vivo. These optimized components have been applied to target myocilin (MYOC). Mutations of MYOC in trabecular meshwork cells have been linked to severe glaucomatous conditions. In human primary trabecular meshwork cells, up to 95% MYOC editing and 85% protein knockdown were seen. This novel approach aims to facilitate glaucoma treatment using transient expression of editing machinery targeting MYOC.

Title: Development of an In Vivo Non-Viral Ocular Editing Platform and Application to Potential Treatments for Glaucoma
Session Type: In-Person Oral Presentation
Session Title: Ophthalmic and Auditory: Delivery Innovations
Abstract Number: 87
Location: Room 318 – 323
Session Date and Time: Wednesday, May 8, 2024, 1:30 p.m. – 3:15 p.m. ET

The accepted abstract is available online on the ASGCT website. The data are embargoed until 6:00 a.m. ET on the presentation day, Wednesday May 8, 2024. A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.

About CRISPR Therapeutics 
Since its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company that recently celebrated the historic approval of the first-ever CRISPR-based therapy and has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY™ (exagamglogene autotemcel) was approved in some countries to treat eligible patients with either of those conditions. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. To learn more, visit www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement
This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) its ongoing and/or planned preclinical studies, clinical trials and pipeline products and programs, including, without limitation, the status of such studies and trials, potential expansion into new indications and expectations regarding data generally (including expected timing of data releases) as well as the data in the above-described abstract and any associated poster and the data that is being presented as described above; (ii) the safety, efficacy and clinical progress of its various clinical and preclinical programs including the program described in the oral presentation and poster; (iii) the data that will be generated by ongoing and planned preclinical studies and/or clinical trials, and the ability to use that data for the design and initiation of further preclinical studies and/or clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the efficacy and safety results from ongoing pre-clinical studies and/or clinical trials will not continue or be repeated in ongoing or planned pre-clinical studies and/or clinical trials or may not support regulatory submissions; pre-clinical study and/or clinical trial results may not be favorable or support further development; one or more of its product candidate programs will not proceed as planned for technical, scientific or commercial reasons; future competitive or other market factors may adversely affect the commercial potential for its product candidates; uncertainties inherent in the initiation and completion of preclinical studies for its product candidates and whether results from such studies will be predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for its technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics’ most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact: 
Susan Kim 
+1-617-307-7503 
susan.kim@crisprtx.com  

Media Contact: 
Rachel Eides 
 +1-617-315-4493 
rachel.eides@crisprtx.com 


FAQ

What is CRISPR Therapeutics presenting at the American Society of Gene & Cell Therapy 2024 Annual Meeting?

CRISPR Therapeutics will present an oral presentation highlighting their lipid nanoparticle approach for ocular editing, focusing on potential treatments for glaucoma.

What is the date and time of the presentation at the ASGCT 2024 Annual Meeting?

The presentation will take place on Wednesday, May 8, 2024, from 1:30 p.m. to 3:15 p.m. ET.

What is the abstract number of the presentation?

The abstract number of the presentation is 87.

What is the focus of the presentation regarding gene editing?

The presentation will focus on targeting the MYOC gene for potential treatments for glaucoma.

Where can the accepted abstract be found online?

The accepted abstract can be found on the ASGCT website.

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Biotechnology
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