Vertex Pharmaceuticals is committing further to Duchenne muscular dystrophy research, announcing on Tuesday a deal with biotechnology startup Tevard Biosciences to develop RNA-based therapies for the progressive genetic condition.
The two companies didn’t disclose deal terms. But they’ve begun a four-year research collaboration to develop medicines for a subset of Duchenne patients, with an option to broaden the alliance into other muscular dystrophies and a second, unspecified disease type.
Tevard is getting an upfront payment, and could receive more cash if the programs progress or Vertex exercises its option rights.
Best known for its cystic fibrosis drugs, Vertex has made a major effort in recent years to supplement that business with a pipeline of medicines for “specialty diseases” — typically rare conditions for which medicines can be sold at high prices and with a small sales force. The company has experimental drugs in development for sickle cell disease and beta thalassemia, as well as Type 1 diabetes and a type of genetic kidney disease.
Duchenne, a progressive and deadly disorder with no cure, has emerged as a focus as well. Vertex first expressed interest in the disease in 2019, when it acquired gene editing biotech Exonics Therapeutics and rights to CRISPR Therapeutics’ Duchenne research.
Yet the biotech is well behind Duchenne drugmakers like Sarepta Therapeutics, which has multiple marketed treatments and a gene therapy U.S. regulators are reviewing. Vertex, by comparison, expects to ask regulators to begin human testing of a gene editing treatment this year, according to a regulatory filing this month.
Nonetheless, Vertex is building a portfolio of prospective treatments, supplementing its gene editing work with Tevard’s research.
“Vertex is committed to transforming the treatment of [Duchenne] and we are pursuing various approaches across multiple therapeutic modalities to bring treatments to as many patients as possible,” said Mike Cooke, the senior vice president of Vertex’s cell and genetic therapies division, in a statement.
Tevard’s programs are focused on transfer RNA, or tRNA, which are carrier molecules for the amino acids cells use to make proteins. The startup is one of several that aim to harness tRNA to make medicines. It was co-founded by MIT cell biologist Harvey Lodish and is developing treatments for a type of epilepsy called Dravet syndrome and other rare conditions.
Tevard’s work with Vertex will focus on Duchenne caused by “nonsense mutations,” in which a change to a single letter of genetic code stops the body from producing the muscle-boosting protein dystrophin. These malfunctions are part of a group of genetic changes that account for 15% to 30% of Duchenne cases, according to advocacy group Parent Project Muscular Dystrophy. They’re also the target of a drug from PTC Therapeutics, called Translarna, that’s approved in Europe but not the U.S.
Tevard’s drugs are meant to restore dystrophin production in those patients. Vertex will fund the costs of Tevard’s research, as well as future development and marketing of any approved programs.