Company to present preclinical therapeutic data on novel ultracompact CRISPR systems

Mammoth Biosciences, a biotechnology company harnessing its proprietary next-generation CRISPR gene-editing platform to create potential one-time curative therapies, today announced that Lucas Harrington, Ph.D., Mammoth’s co-founder and Chief Scientific Officer, will present new preclinical data at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore, May 7 - 11, 2024.

The oral presentation will describe the optimization of Mammoth’s novel ultracompact CRISPR platform, and its performance in targeting the APOC3 gene, a primary genetic driver of triglyceride metabolism, in humanized mouse models and non-human primates.

“Our findings demonstrate the potential of Mammoth’s ultracompact CRISPR systems to deliver in vivo therapies in the liver, beginning with our lead program targeting APOC3, a key factor in familial chylomicronemia syndrome and severe hypertriglyceridemia,” Dr. Harrington said. “We look forward to sharing these data with the scientific community at ASGCT, and to providing future updates as we continue to develop our novel ultracompact nucleases and associated gene-editing technologies to better target tissues beyond the liver and to positively impact the lives of patients living with genetic disease.”

Presentation Details

Title: Reduction in Triglycerides through a Novel Ultracompact CRISPR System: Efficacy in Mouse Models and NHP Studies

Abstract #: 15

Date and Time: 2:30 p.m. - 2:45 p.m. ET, Tuesday, May 7

Location: Ballroom 1

Additionally, Janice Chen, Ph.D., co-founder and Chief Technology Officer of Mammoth, and incoming chair of the ASGCT Genome Editing Committee, will co-chair the following scientific symposium:

Scientific Symposium Details

Title: Advances in Genome Editing: In Vivo Small Edits and the Promise of Large Insertions

Date and Time: 8:00 a.m. - 9:45 a.m. ET, Friday, May 10

Location: Ballroom 2

More details about the oral presentation and symposium are available 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.

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