Mammoth Biosciences to Unveil Groundbreaking CRISPR Therapy Data at ASGCTMeeting

Mammoth Biosciences will present preclinical data on its novel ultracompact CRISPR systems at the ASGCT 27th Annual Meeting. The company's Chief Scientific Officer will discuss the system's potential to treat triglyceride metabolism disorders.

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Nitish Verma
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Mammoth Biosciences to Unveil Groundbreaking CRISPR Therapy Data at ASGCTMeeting

Mammoth Biosciences to Unveil Groundbreaking CRISPR Therapy Data at ASGCTMeeting

Mammoth Biosciences, a pioneeringbiotechnology company, is set to present groundbreaking preclinical data on its novel ultracompact CRISPR systems at the upcoming American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore, Maryland. The highly anticipated presentation, scheduled for May 7-11, 2024, will showcase the company's innovative advancements in developing in vivo therapies targeting the APOC3 gene, a key player in triglyceride metabolism.

Why this matters: This breakthrough in CRISPR technology has the potential to transform the treatment of genetic diseases, offering new hope to patients and families affected by triglyceride metabolism disorders. The successful development of in vivo therapies could also pave the way for treating a wide range of other The successful development of in vivo therapies could also pave the way for treating a wide range of othergenetic diseases, with far-reaching implications for the future of medicine.

The oral presentation, titled "Reduction in Triglycerides through a Novel Ultracompact CRISPR System: Efficacy in Mouse Models and NHP Studies," will be delivered by Lucas Harrington, Ph.D., Mammoth's co-founder and Chief Scientific Officer, on Tuesday, May 7, from 2:30 p.m. to 2:45 p.m. ET in Ballroom 1. Dr. Harrington will share the company's latest findings, which demonstrate the remarkable potential of their proprietary ultracompact CRISPR platform in targeting the APOC3 gene, a primary genetic driver of triglyceride metabolism, in both 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," stated Dr. Harrington. This groundbreaking research holds immense promise for the development of long-term curative therapies for patients suffering from life-threatening and debilitating diseases related to triglyceride metabolism disorders.

In addition to the highly anticipated presentation, Janice Chen, Ph.D., co-founder and Chief Technology Officer of Mammoth, will co-chair a scientific symposium titled "Advances in Genome Editing: In Vivo Small Edits and the Promise of Large Insertions" on Friday, May 10, from 8:00 a.m. to 9:45 a.m. ET in Ballroom 2. This symposium will explore the latest advancements in genome editing technologies and their potential applications in the development of novel therapies.

Mammoth Biosciences, founded by CRISPR pioneer and Nobel laureate Jennifer Doudna, along with Trevor Martin, Janice Chen, and Lucas Harrington, has been at the forefront of developing innovativeCRISPR-based technologies. The company's proprietary ultracompact CRISPR systems have attracted significant attention from the scientific community and industry experts for their potential to transform the field of gene editing and pave the way for groundbreaking therapies.

The ASGCT 27th Annual Meeting serves as a premier platform for showcasing the latest advancements in gene and cell therapy, bringing together leading researchers, clinicians, and industry professionals from around the world. Mammoth Biosciences' presentation at this prestigious event highlights the significance of their research and its potential impact on the future of medicine.

The scientific community eagerly awaits the unveiling of Mammoth Biosciences' groundbreaking findings, with the implications of their research extending far beyond triglyceride metabolism disorders. The successful optimization of their ultracompact CRISPR platform for in vivo therapies opens up a world of possibilities for treating a wide range of genetic diseases, offering hope to countless patients and their families.

Key Takeaways

  • Mammoth Biosciences to present novel CRISPR system at ASGCT 27th Annual Meeting.
  • System targets APOC3 gene, a key player in triglyceride metabolism disorders.
  • Breakthrough has potential to transform treatment of genetic diseases.
  • In vivo therapies could treat a wide range of genetic diseases.
  • Research holds promise for long-term curative therapies for triglyceride metabolism disorders.