Award highlights the potential of MB-111 as a first-in-class, single-dose in vivo ultracompact CRISPR gene editing therapy for persistent chylomicronemia
BRISBANE, Calif. – November 5, 2026 – Mammoth Biosciences, Inc., a biotechnology company harnessing its proprietary next-generation in vivo CRISPR gene editing platform to create potential one-time curative therapies, today announced that MB-111, its first clinical development candidate, has received the 2026 Biotech Breakthrough Award for Genomic Medicine of the Year.
MB-111 is a potential first-in-class, single-dose therapy for persistent chylomicronemia, a metabolic disorder characterized by extremely elevated triglyceride levels (>1000 mg/dL) and recurrent, potentially life-threatening episodes of acute pancreatitis. The therapy uses CasPhi, Mammoth’s proprietary ultracompact CRISPR nuclease, delivered within a lipid nanoparticle, to durably lower triglycerides by permanently disrupting expression of the APOC3 gene in the liver, reducing production of apoC-III protein, a key regulator of triglyceride metabolism.
“Even when medicines are available, staying on treatment over many years can be challenging,” said Trevor Martin, Ph.D., co-founder and chief executive officer of Mammoth Biosciences. “With MB-111, our goal is to achieve sustained triglyceride lowering through a single treatment, potentially reducing dependence on repeated dosing. As we prepare for clinical evaluation, we’re focused on understanding the benefits of this approach for patients who require long-term triglyceride control.”
Preclinical studies of MB-111 demonstrated substantial reductions in serum triglycerides in a mouse model of hypertriglyceridemia and sustained reductions in apoC-III protein in nonhuman primates. These findings support the continued development of MB-111 toward clinical evaluation, which is expected in 2027.
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 packageable to 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 Biosciences’ 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.
About BioTech Breakthrough
BioTech Breakthrough is a leading independent market intelligence organization focused on technology research and industry recognition. Its annual awards highlight advances in biotechnology and the life sciences, spanning areas such as genetic research, drug development, and bioinformatics.
For more information, please visit www.biotechbreakthroughawards.com.
Mammoth Biosciences Media Contact:
Mohana Ray
Email: Mammoth.PR@hdmz.com Phone: 312-506-5210