The Cystic Fibrosis Foundation has invested $3 million in ReCode Therapeutics to provide additional support for the Phase 2 clinical trial of its messenger RNA (mRNA) therapy. This therapy has the potential to help all people with CF, including those with rare and nonsense mutations who do not benefit from modulators.
The Foundation previously invested $15 million in ReCode to support the development and early-stage clinical trials of this inhaled mRNA therapy, designed to provide a correct copy of the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA to lung cells to make a functional CFTR protein.
“Clinical trials for mRNA therapy may be small, but they represent a big opportunity, especially for those people with CF who are urgently waiting for a transformative treatment,” said Steven M. Rowe, MD, MSPH, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “This investment demonstrates our dedication to learning more about these treatments, so we can aggressively pursue options for everyone with this disease.”
To deliver the mRNA therapy into lung cells, ReCode is using a unique tissue and cell-targeting lipid nanoparticle — an alternative to other delivery systems such as engineered viruses, and an advancement over the conventional lipid nanoparticles used to deliver the mRNA COVID-19 vaccines. Similar to a pill capsule that protects medicines, lipid nanoparticles shield and direct genetic material to cells without damaging the material along the way. ReCode’s lipid nanoparticles may allow genetic therapies to enter lung cells more easily and, importantly, may allow safe redosing of the CFTR mRNA.
In the Phase 2 trial, a new cohort (or group) will enroll three people with CF who will receive multiple doses of mRNA therapy for four weeks. Additionally, six other participants will receive a lower dose level in a 12-week expansion group. The trials will test safety, tolerability, and the efficacy of the doses. The expansion group will test whether prolonged treatment time leads to better outcomes.
In total, the Foundation has agreed to invest up to $33 million in ReCode for its mRNA and gene editing research programs. Our collective investments with ReCode demonstrate the Foundation’s commitment to strategically advance the most promising science to deliver transformative therapies for all people with CF.
About mRNA Therapy
Genes contain the DNA instructions for making proteins. The CFTR gene contains the DNA blueprint for producing CFTR protein. To make the protein, the cell copies the genetic instructions into a similar molecule called mRNA. The mRNA then acts as a template, carrying the instructions from the gene to the protein-making machinery of the cell where the protein can be assembled.
In cystic fibrosis, mutations in the CFTR gene produce faulty mRNA instructions, resulting in missing or defective CFTR protein that cause cystic fibrosis. However, mRNA therapy delivers correct copies of CFTR mRNA to lung cells. The lung cells use the new, correct instructions to produce healthy CFTR protein.