26. ElevateBio

Founders: David Hallal (CEO), Vikas Sinha, Mitchell Finer
Launched: 2019
Headquarters: Waltham, Massachusetts
Funding: $1.2 billion
Valuation: N/A
Key technologies: Cloud computing
Industry: Biotech
Previous appearances on Disruptor 50 List: 1 (No. 10 in 2021)

Rare diseases are known to affect 25 to 30 million Americans, but oftentimes companies shy away from investing in research and technology for treatments because of high costs, uncertain results, and relatively small patient populations. 

The FDA has approved less than 30 cellular and gene therapy products to date, but with the breakthroughs in CRISPR gene editing, there are thousands of gene therapy companies and treatments in the pipeline or expected to be in the years ahead.

ElevateBio, based in the Boston-area biotech hub, has been described as the health R&D equivalent of a "tech stack," providing infrastructure for gene editing, stem cells, and protein, viral, and cellular engineering. Its BaseCamp, a 140,000-square-foot warehouse facility in Waltham, Massachusetts, was built to design gene therapy products and collaborate with new partners. 

Operating as both a biotech start-up and a holding entity, ElevateBio announced in February that its company Life Edit Therapeutics had signed a deal with Moderna to discover and develop in vivo mRNA gene editing therapies. ElevateBio believes mRNA may hold the key to delivering gene-editing technology, potentially developing treatments or even curing rare genetic diseases.

In 2022, ElevateBio formed a 30-year partnership with the University of Pittsburgh and the RK Mellon Foundation to build another BaseCamp facility in Pittsburgh, equipped with ElevateBio's technology and manufacturing capabilities. The $100 million grant was the largest in the foundation's 75-year history, and could set the stage for further expansion of biomanufacturing across the U.S. as it cultivates local partnerships and funding. Since 2019, it has had a 10-year deal for cell and gene therapy clinical trials with the Massachusetts General Hospital, which conducts the biggest hospital-based research effort in the U.S.

The company established 10 new partnerships in total within the last year, including one with Boston Children's Hospital and Harvard Medical School Dean George Daley to develop off-the-shelf cell therapies for cancer.