Vertex Pharmaceuticals announced Tuesday that it plans to acquire an ambitious startup for $950 million, betting the company’s early-stage science could lead to a functional cure for type 1 diabetes.
The Cambridge, Mass., company is buying Semma Therapeutics, a nearby firm turning stem cells into insulin factories. Based on the work of Harvard University stem cell scientist Douglas Melton, Semma’s approach involves turning moldable stem cells into beta cells, the insulin-producing machinery that is mistakenly attacked by the immune system in type 1 diabetes.
If it works — if Semma’s lab-grown beta cells can replace those lost to the disease — the company may have invented a permanent solution to a disease that affects more than 1 million people in the US. Semma’s cells would be transplanted directly into the liver or meted out by an implant, freeing patients forever from their insulin injections.
But it’s early days for Semma’s work, which is yet to be tested in a clinical trial. And development has been a longer process than Semma anticipated. In 2015, the company raised $44 million from investors and said that money would fund its work through human trials. As of today, Semma has raised more than $100 million more and has only tested its technology on primates and pigs.
For Vertex, which has built a multibillion-dollar business on treatments for the rare cystic fibrosis, the Semma deal is a nod toward the company’s scientific future. With its fourth — and likely final — oral treatment for CF expected to win approval soon, Vertex has turned to potentially curative technologies like CRISPR genome editing and synthetic messenger RNA for its next generation of products. The company’s stock edged down modestly in trading Tueday, closing at $178.70 a share.
In April, longtime chief executive Jeffrey Leiden will step down, to be succeeded by current Chief Medical Officer Reshma Kewalramani.
Damian Garde can be reached at firstname.lastname@example.org. Follow him on Twitter @damiangarde. Follow Stat on Twitter: @statnews.