LifeMine Therapeutics Raises $263M to Advance Novel Calcineurin Inhibitor LIFE-001 for Organ Transplant Rejection
Key Insights
LifeMine Therapeutics (search) secured $263 million in Series D and E financing to advance LIFE-001, a novel direct calcineurin (search) inhibitor discovered through fungal genome mining.
LIFE-001 binds directly to calcineurin (search) without relying on immunophilin proteins, potentially avoiding the multiorgan toxicities associated with legacy drugs like tacrolimus and cyclosporine.
An ongoing Phase 1 study in over 120 adults has shown no clinically meaningful renal, metabolic, or cardiovascular safety signals to date.
LifeMine Therapeutics (search), a biotechnology company founded by serial entrepreneur and former Harvard chemical biologist Greg Verdine, announced Thursday it has raised $263 million across two venture financings to propel LIFE-001—a novel, long-acting calcineurin (search) inhibitor—into later-stage clinical testing for organ transplant rejection (search).
The financing comprises a $75 million Series D round completed in the fourth quarter of 2025 and an oversubscribed $188 million Series E round led by Milky Way Investments. New investors in the Series E include Jeff Bezos' investment firm Bezos Expeditions, Gates Frontier, and RA Capital Management, with continued support from GV, LoLa Capital Partners, GlaxoSmithKline, Invus, and ARCH Venture Partners. The company has now raised approximately $580 million in private financing since its founding in 2017.
A Billion-Year-Old Starting Point
LIFE-001 emerged from LifeMine's Top-Down Drug Discovery platform, which systematically mines the fungal genome—the same biological kingdom that gave rise to the first statin, penicillin, and the immunosuppressant cyclosporine. The platform integrates human genetics, genomics, bioinformatics, machine learning, and synthetic biology to identify potential medicines with new mechanisms of action.
"LIFE-001 is designed to overcome the limitations of legacy calcineurin (search) inhibitors through the direct inhibition of calcineurin activation without relying on intermediary immunophilin proteins," said Gregory Verdine, Ph.D., Co-Founder, President and CEO of LifeMine Therapeutics (search).
A Differentiated Mechanism
Existing calcineurin (search) inhibitors such as tacrolimus, cyclosporine, and voclosporin prevent T-cell activation and organ rejection by functioning as molecular glues—forcing together two proteins that would not otherwise interact. This indirect mechanism contributes to substantial toxicities, a critical concern given that transplant recipients typically remain on these therapies for life. Tacrolimus, commonly prescribed to kidney transplant patients, is associated with tremors, seizures, and post-transplant diabetes mellitus.
LIFE-001 takes a fundamentally different approach: it binds directly to calcineurin (search) at a previously unobserved site in the enzyme's structure, inhibiting T-cell activation without engaging immunophilin proteins. Importantly, the drug does not appear to penetrate the brain or other peripheral tissues, a property that could translate into an improved safety profile.
The drug is also formulated as a long-acting injectable. "This flattens out the pharmacokinetics, so you don't have this oscillation between too much and too little suppression," Verdine explained.
Early Clinical Data and Upcoming Trials
In an ongoing Phase 1 single- and multiple-ascending-dose study involving more than 120 adults, LIFE-001 has not produced clinically meaningful renal, metabolic, or cardiovascular safety signals to date, according to the company.
LifeMine now plans to advance LIFE-001 into a Phase 2 study in kidney transplant recipients and a Phase 1b trial in patients receiving islet cell transplants for conditions such as type 1 diabetes (search) or chronic pancreatitis (search). Data from the islet cell transplant study could arrive by the end of 2027, while results from the kidney transplant trial are expected in 2028.
Strategic Positioning
While LifeMine explored the possibility of developing LIFE-001 for autoimmune conditions such as Crohn's disease and lupus nephritis, the company opted to focus on transplantation. "Transplantation is a major opportunity that was hiding in plain sight that everyone just overlooked," Verdine said.
The company also announced the appointment of Yves Zinggeler as Chief Commercial Officer. Zinggeler previously served as Vice President and General Manager of Vertex's U.S. Cystic Fibrosis Business Unit and has participated in 14 product launches and more than a dozen label expansions.
LifeMine has not ruled out an initial public offering, though Verdine indicated the company would wait until accumulating data from its first two human studies. The biotech is also considering eventually commercializing LIFE-001 independently.
"A next-generation calcineurin (search) activation inhibitor capable of maintaining efficacy while significantly reducing multiorgan toxicity would represent one of the most meaningful advances in transplantation in decades," the company stated.