Two companies working on treatments for rare genetic lung diseases have combined efforts and raised $80 million to bring into human testing next year two investigational treatments that use types of RNA to tweak mutated proteins.
Dallas company ReCode Therapeutics and Menlo Park, CA-based TranscripTx have merged under the ReCode name and leadership of CEO David Lockhart to advance treatments for progressive pulmonary diseases caused by genetic mutations. On Thursday the company announced it would put proceeds from the Series A financing toward continued preclinical work on its lead programs, investigational therapies for two genetic pulmonary diseases: primary ciliary dyskinesia and cystic fibrosis.
Primary ciliary dyskinesia is a rare disorder characterized by chronic respiratory tract infections, and in some patients abnormally positioned internal organs and infertility. The disease, caused by mutations in genes that result in cilia that move abnormally or are unable to move, is believed to occur in 1 out of every 16,000 people. ReCode is developing a messenger RNA drug to treat this disease.
Its other lead program is a treatment being developed for a subset of people with cystic fibrosis—a progressive genetic disease that causes frequent lung infections—whose disease is caused by what are known as nonsense mutations. Those patients aren’t able to benefit from new medicines, called modulators, like those developed by Vertex Pharmaceuticals (NASDAQ: VRTX) in recent years that have changed the outlook for many cystic fibrosis patients. The experimental ReCode drug uses transfer RNA to encourage production of the protein that in CF patients is missing or produced at abnormally low levels.
For both programs, ReCode aims to ask the FDA for the OK to start human testing in 2021.
The company is also working on a delivery method for RNA therapies and gene editing components that uses non-viral lipid nanoparticles to target specific organs.
OrbiMed Advisors and Colt Ventures led the round. Investors MPM Capital, Vida Ventures, Hunt Technology Ventures, and Osage University Partners also participated.
The discoveries that underpin the company’s research were made by Daniel Siegwart and Philip Thomas, professors at the University of Texas Southwestern Medical Center, and Texas A&M University professor emeritus Arthur Johnson.
ReCode, which has 15 employees, will maintain offices in Menlo Park and Dallas, both of which it plans to expand. Lockhart is based in Menlo Park.
As part of the deal, ReCode appointed a new board of directors, which includes OrbiMed partner Peter Thompson, MPM Capital managing director Ed Hurwitz, Vida Ventures managing director Helen Kim, and ReCode founder Michael Torres, the combined company’s new vice president of research and development.