Prime Medicine, a biotech company that set out to use its more precise approach to gene editing to develop new one-time treatments for rare diseases, is expanding its scope to more prevalent therapeutic areas under a new partnership with Bristol Myers Squibb.
The pharmaceutical giant struck a deal for an exclusive global license to Prime Medicine’s technology, which will be used to develop next-generation ex vivo T cell therapies for oncology and immunology. The deal terms announced Monday call for BMS to pay $110 million up front, a sum that’s split equally between cash and an equity investment in Prime Medicine. The targets for the partnered cell therapies remain undisclosed, but they will be selected by BMS.
Cambridge, Massachusetts-based Prime Medicine focuses on therapies based on prime editing, an approach it says enables the company to edit, correct, insert, or delete DNA sequences in any target tissue. The company’s technology, Prime Assisted Site-Specific Integrase Gene Editing, or PASSIGE, does not employ engineered viruses for delivery like some genetic medicines. Furthermore, it can work with pieces of DNA too large for other gene-editing technologies. Prime editing’s more precise edits are intended to reduce the risk of complications that can result from other editing technologies.
Reducing Clinical and Staff Burnout with AI Automation
As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers.
The BMS partnership comes at a key time for Prime Medicine, which said in its most recent financial report that its cash would last only through the second quarter of 2025. When the company went public in 2022, its pipeline spanned 18 rare-disease programs — all of them preclinical. The company’s most advanced program, named PM359, recently began a Phase 1/2 study in chronic granulomatous disease (CGD), a rare, inherited blood disorder that makes patients susceptible to infections. Preliminary data are expected in 2025.
Along with the new BMS alliance, Prime Medicine on Monday also announced the refocusing of its pipeline on certain strategic programs, each of which addresses a disease with well-understood biology that offers a clearly defined clinical development and regulatory path. Furthermore, the company said that the prioritized program in each therapeutic area will serve as a beachhead from which it can expand.
Prime Medicine’s liver disease R&D is focused on a prime editor for Wilson’s disease, an inherited disorder that leads to a buildup of copper in the organs. Activities to support an investigational new drug application for this program are on track to begin in the fourth quarter of this year. The company said delivery of this therapy will leverage a universal lipid nanoparticle, which it expects will be used across other liver disease programs. Meanwhile, Prime Medicine’s lung research is focusing on adeno-associated virus prime editors to address multiple mutations that drive cystic fibrosis. This research has funding from the Cystic Fibrosis Foundation.
PM359 in CGD leads the biotech’s efforts in hematology, immunology, and oncology. As a follow-on to that program, Prime Medicine is also using its technology to develop a treatment for X-linked CGD leveraging elements from the PM359 program. The BMS partnership is part of this therapeutic area of focus. Under the terms of the agreement announced Monday, BMS is responsible for development, manufacturing, and commercialization of cell therapies stemming from the collaboration. Prime Medicine could receive more than $3.5 billion in development and commercialization milestone payments, plus royalties from sales of any commercialized products.
Market Trends to Watch for Health Systems and Their Specialty Pharmacies
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
“We are excited to collaborate with Bristol Myers Squibb, a global leader in cell therapy for hematology, immunology, and oncology,” Prime Medicine President and CEO Keith Gottesdiener said in a prepared statement. “Through this effort, we will apply our Prime Editing technology beyond the rare genetic diseases in our internal pipeline, potentially unlocking opportunities in areas of high unmet needs in immunological diseases and cancer.”
To maintain its focus on the prioritized programs, Prime Medicine will look for partners for its other programs. This research spans neurological diseases, cell therapy, ocular diseases, and hearing loss. With the savings from the pipeline reprioritization and the cash infusion from BMS, Prime Medicine now expects its capital will support the company into the first half of 2026.
Image: Getty Images