The Cystic Fibrosis Foundation will invest significantly in 2 investigational gene therapies for patients with cystic fibrosis (CF). The therapies aim to use novel nanoparticle technologies to deliver correct copies of CF transmembrane conductance regulator (CFTR) messenger RNA (mRNA) to lung cells so they can produce functional CFTR proteins.

“Messenger RNA therapy has the potential to help all people with CF, including those who don’t respond to CFTR modulator treatment,” Steven Rowe, MD, chief scientific officer at the foundation, said. “ReCode [Therapeutics] is testing whether they can optimize their RNA therapy to reach the correct cells in the lungs using its novel lipid nanoparticles, restoring CFTR activity.”

The therapies are being developed by Gensaic and ReCode Therapeutics. Gensaic plans to utilize its proprietary phage-derived particle (PDP) platform to deliver the genes to lung tissue.

PDPs represent a new class of genetically engineered nanoparticles capable of delivering over 20 kb of DNA. Two phases of development are planned, with the second phase dependent on achieving specific preclinical milestones in the first phase. If successful, Gensaic’s new therapy would represent a redosable, targetable, and scalable gene therapy for individuals with CF.

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ReCode is developing an inhaled mRNA therapy for the delivery of CFTR mRNA to lung tissue. The company is also employing nanoparticle technology, specifically a unique, selective organ-targeting lipid nanoparticle designed to enter lung cells more easily than current therapies.

Preclinical testing of ReCode’s therapeutic approach demonstrated that the CFTR mRNA could be delivered to human lung cells via an aerosol. CFTR protein function was shown to improve in lung cells with 1 or 2 F508del mutations and 1 G542X mutation. The Cystic Fibrosis Foundation is one of several institutional and strategic investors in the ReCode project.

References

Gensaic receives funding from the CF Foundation to develop a gene therapy for people with cystic fibrosis. News release. Gensaic; January 9, 2023.

CF Foundation invests up to $15M in ReCode Therapeutics for development of mRNA therapy. News release. Cystic Fibrosis Foundation; January 10, 2023.