BRISBANE, Calif.--(BUSINESS WIRE)--#CRISPR--Mammoth Biosciences, Inc., a biotechnology company harnessing its proprietary next-generation CRISPR gene editing platform to create potential one-time curative therapies, today announced that Renan Sper, DVM, Ph.D., principal scientist, preclinical therapeutics at Mammoth, will present new preclinical data at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, May 13-17, 2025.
The oral presentation will showcase Mammoth Biosciences’ latest results with NanoCas, the first efficient ultracompact extrahepatic gene editor. The data demonstrates that NanoCas efficiently edits skeletal muscle tissue in mice and non-human primates when delivered systemically using a single adeno-associated viral (AAV) vector. Mammoth Biosciences published a study describing the discovery, engineering and benchmarking of NanoCas on the preprint server bioRxiv in January.
“This study represents a major advancement for the gene editing field, which has long been limited by access to extrahepatic tissues,” said Janice Chen, Ph.D., cofounder and chief technology officer of Mammoth Biosciences, and chair of the ASGCT Gene Editing Committee. “NanoCas not only overcomes that barrier, but also offers a solution to unlock diverse gene modification approaches across multiple tissues. It’s an important step toward making targeted in vivo gene editing broadly accessible for a wide range of therapeutic applications.”
Presentation Details
Title: Non-Human Primate Muscle Gene Editing Via Single Systemic AAV Delivery of Ultra-Compact CRISPR Nuclease
Date and Time: May 15, 2025, 1:30 p.m. - 1:45 p.m.
Location: Room 288-290
More information about the oral presentation and conference may be found on the ASGCT meeting website.
About Mammoth Biosciences
Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact systems are designed to be more specific and enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, reverse transcriptase editing, and epigenetic editing. The company is building out its wholly owned pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology platform. Mammoth’s deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the promise of CRISPR technologies.
For more information, please visit www.mammoth.bio or follow Mammoth on LinkedIn or X.
Contacts
Media Contact:
Mohana Ray
Email: Mammoth.PR@hdmz.com
Phone: 312-506-5210
