Brisbane, CA — Mammoth Biosciences has named its first clinical development candidate, MB-111, a potential first-in-class, one-time CRISPR-based treatment targeting severe lipid disorders. Alongside this milestone, the company has also appointed Dr. Bob D. Brown, a seasoned leader in genetic medicines, to its board of directors as it prepares to enter the next phase of therapeutic development.
MB-111 leverages Mammoth’s proprietary CasPhi system, an ultracompact in vivo CRISPR gene editing platform that is less than half the size of traditional Cas9-based systems. Delivered via lipid nanoparticles, MB-111 is designed to target the liver and permanently disrupt the APOC3 gene, reducing production of the ApoC-III protein—a key driver of dangerously high triglyceride levels in conditions such as familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG).
“These disorders are linked to recurrent acute pancreatitis and increased cardiovascular risk,” said Mammoth Biosciences CEO and co-founder Trevor Martin, Ph.D. “The nomination of MB-111 marks not just a milestone for the company, but a significant step forward for gene editing in treating chronic and rare metabolic conditions.”
The company is expected to begin investigational new drug (IND)-enabling studies for MB-111 later this year.
As part of its continued growth, Mammoth has also welcomed Dr. Bob D. Brown to its board. Dr. Brown brings more than three decades of expertise in nucleic acid-based therapeutics. He most recently served as Chief Scientific Officer and EVP of R&D at Dicerna Pharmaceuticals, an RNAi-focused company acquired by Novo Nordisk, where he led the development of multiple investigational drugs now advancing through major pharmaceutical pipelines.
“CRISPR is on the verge of reshaping how we treat disease,” said Dr. Brown. “Mammoth’s ultracompact gene editing technology holds remarkable promise, and I’m excited to support the team in translating that potential into transformative therapies, beginning with MB-111.”
Dr. Brown’s appointment reinforces Mammoth’s broader ambition to become a leader in the field of in vivo gene editing. With a growing pipeline and proprietary delivery systems, the company aims to expand access to next-generation genetic medicines targeting both rare and prevalent conditions.
