Today, a brief rundown of news involving Tessera Therapeutics and Corvus Pharmaceuticals, as well as updates from Sarepta Therapeutics, Cara Therapeutics and Johnson & Johnson that you may have missed.
The Bill & Melinda Gates Foundation will invest as much as $50 million to support Tessera Therapeutics’ development of a curative genetic treatment for sickle cell disease that can work without the intensive preparatory regimens needed for drugs like Casgevy and Lyfgenia. Tessera, which specializes in technology it says can change single DNA letters as well as add whole genes, is developing its sickle cell therapy as an “in vivo” treatment, meaning it does its modification to cell genes directly inside the body. Casgevy and Lyfgenia are “ex vivo,” built from editing patient cells in a laboratory. Simpler in vivo therapies could be an important to bringing genetic medicine to parts of the world without advanced healthcare infrastructure. — Ned Pagliarulo
Shares in Corvus Pharmaceuticals collapsed Wednesday, shedding nearly half of their value following the company’s release of study data for its drug soquelitinib in people with eczema. Results came from the first portion of a Phase 1 study involving 12 participants given Corvus’ drug and four who received placebo. The data showed roughly similar skin clearance in both groups through 15 days, but greater clearance for soquelinitib-treated volunteers than those on placbeo through 28 days. — Ned Pagliarulo
Sarepta Therapeutics on Wednesday said it has finished enrolling a Phase 3 study of a gene therapy it’s developing for a type of limb-girdle muscular dystrophy. The company expects data from the trial in the first half of next year and, if positive, anticipates that they’d support an approval application to the Food and Drug Administration. Sarepta is also developing gene therapies for other forms of the muscle-weakening disease. — Ned Pagliarulo
Cara Therapeutics and private biotechnology firm Tvardi Therapeutics have agreed to merge, giving Tvardi an entry to public markets to help fund development of medicines for fibrotic diseases. Once the deal closes, the new company will operate under Tvardi’s name and a new ticker, “TVRD.” Its combined cash on hand will fund operations into the second half of 2026, before which Tvardi expects data from two Phase 2 studies of its experimental STAT3 blocker in idiopathic pulmonary fibrosis and hepatocellular carcinoma. Separately, Cara has reached a deal with Vifor Fresenius Medical Care Renal Pharma to sell rights to its approved drug Korsuva. — Delilah Alvarado
The Food and Drug Administration has rejected Johnson & Johnson’s application for a subcutaneous form of its lung cancer drug Rybrevant. The complete response letter cited findings from a pre-approval inspection of a manufacturing facility and did not flag concerns with the drug product, or its safety and efficacy, J&J said. The company plans to work “closely” with the agency to bring the under-the-skin shot to market. The approved intravenous formulation of Rybrevant is not affected by the complete response letter. — Delilah Alvarado
This article was originally published here.
