Another rare disease startup is entering the market, this time backed by a group of executives who have been here before.
Mendra, a new biotech led by former BioMarin and Escient Pharmaceuticals executives, has raised $82 million in a Series A round led by OrbiMed, 8VC, and 5AM Ventures. The company says the capital will go toward acquiring drug assets and pushing them into development.
That part is fairly standard. Where Mendra is trying to differentiate is how it plans to run development.
AI as an Operational Tool, Not Just a Buzzword
Mendra says it will use artificial intelligence across the full drug lifecycle, not just in discovery. That includes figuring out which patients are most likely to respond, improving enrollment in clinical trials, and planning commercialization earlier than usual.
Rare disease programs often struggle with these steps. Patient populations are small, geographically scattered, and expensive to reach. Even drugs that work can stumble operationally. Mendra appears to be betting that better data and earlier planning can reduce some of that friction.
Whether AI actually delivers on that promise remains to be seen, but investors seem willing to give the team the benefit of the doubt.
Investors Back the Team as Much as the Technology
Rare disease drug development is notoriously difficult. Companies have to balance high R&D costs against limited patient populations, all while convincing payers the drugs are worth premium pricing.
BioMarin is one of the few companies that has managed to make that model work at scale. It has had missteps, but it also built a portfolio of eight approved therapies and set a template others still follow.
Mendra’s leadership draws heavily from that background.
Leadership Has Been Through the Cycle Before
CEO Joshua Grass spent 15 years at BioMarin between 2002 and 2017, helping build its pipeline. He later led Modis Therapeutics and Escient Pharmaceuticals, both of which were acquired.
Other senior leaders include Jeff Ajer, former chief commercial officer at BioMarin, Lalarukh Haris Shaikh, previously an executive vice president at Palantir Technologies, and Gregory Balani, who ran business development at Escient.
That combination suggests Mendra is positioning itself less as a science experiment and more as a company built to scale if the assets cooperate.
Regulatory Timing May Work in Its Favor
Mendra is launching at a moment when U.S. regulators are showing more flexibility around rare and ultra-rare diseases. In November, the FDA outlined a new pathway aimed at speeding development of highly targeted therapies.
That framework has already encouraged new company formation, including Aurora Therapeutics earlier this year. It would not be surprising to see more startups follow a similar path.
For Mendra, the pitch is clear: experienced operators, AI used pragmatically rather than theoretically, and a regulatory environment that may shorten timelines. The hard part, as always in biotech, will be whether the assets justify the confidence.
