The funding also will be used to support the development of RNA therapies for primary ciliary dyskinesia (PCD), a hereditary condition that impairs the function of lung cilia — the fingerlike projections in the airways — leading to chronic respiratory infections.
“Our preclinical studies demonstrate that our targeted RNA therapies have great potential for the treatment of life-threatening pulmonary diseases,” David Lockhart, PhD, president and CEO of ReCode Therapeutics, said in a press release.
The RNA therapy being developed for CF is called tRNA NanoCorrector. It uses an RNA molecule called transfer RNA (tRNA), which plays a role in the process of protein production.
Specifically, the tRNA NanoCorrector was designed to recognize mutations that introduce a premature stop sequence into the making of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. (These mutations are the cause of CF.) This premature stop sign results in the production of a shortened version of the CFTR protein, which is then degraded by the cell.
The NanoCorrector substitutes the stop sequence for a “go” sequence, so that the full CFTR protein is produced.
The new financing also will support the development of ReCode’s non-viral lipid nanoparticle delivery platform (LNP), which uses non-viral fat (lipid) particles to deliver RNA therapies and other types of gene-editing tools with precision to targeted organs.
“I’m especially grateful to ReCode’s founders, professors Daniel Siegwart, PhD, and Philip Thomas, PhD, at the University of Texas Southwestern Medical Center, and Professor Emeritus Arthur Johnson from Texas A&M University whose foundational research played an instrumental role in cultivating both the LNP delivery platform and the CF therapeutic program,” Lockhart said.
“We are grateful to the Cystic Fibrosis Foundation, whose financial support has allowed us to play a pivotal role in understanding the pathogenesis of CF and led to the development of a novel therapeutic approach for correcting nonsense mutations for this severe disease,” Lockhart added.
ReCode hopes to advance its RNA targeted therapies into clinical trials for CF and PCD. The company plans to file an investigational new drug application (IND) for both clinical programs in 2021.
“We are pleased to close this financing round with world-class investors who believe in the bold vision of our new company. With these additional resources, we’re focused on advancing our preclinical programs into the clinic over the next two years,” Lockhart said.
The investment in Series A financing was co-led by OrbiMed Advisors and Colt Ventures, and had the support of multiple investors, including MPM Capital, Vida Ventures, Hunt Technology Ventures, and Osage University Partners.
“We believe that ReCode has an exceptional opportunity to advance its targeted RNA therapies and LNP delivery platform to address the critical needs of patients living with devastating genetic respiratory diseases,” said Peter Thompson, MD, partner at OrbiMed.
“The board looks forward to supporting ReCode’s evolution as it moves these compelling therapies into the clinic,” Thompson said.
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