ReCode to secure funds for cystic fibrosis gene correction treatments

The CF Foundation's investment will propel the development of up to three gene correction programmes.

gullapalli November 19 2024

ReCode Therapeutics is set to secure up to $15m in additional funding from the Cystic Fibrosis Foundation to enhance its gene correction research programme.

The initiative, conducted in collaboration with Intellia Therapeutics, aims to develop new treatments for cystic fibrosis (CF), including for patients with genotypes that are non-responsive or intolerant to existing cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

The programme utilises Intellia's clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing platform and deoxyribonucleic acid (DNA)-writing technology along with the company’s selective organ targeting (SORT) lipid nanoparticle (LNP) delivery platform aiming at gene correction.

The investment will propel the development of up to three gene correction programmes which are designed to address CF mutations not covered by current therapies.

It will also enhance ReCode's existing RCT2100 clinical programme, an investigational inhaled messenger ribonucleic acid (mRNA) therapy, currently in a Phase Ib multi-dose study for CF patients.

ReCode Therapeutics CEO Shehnaaz Suliman stated: “This funding will support our gene correction approach, enabling us to significantly accelerate research in collaboration with Intellia and to advance the development of a potentially transformative treatment for CF.

“Along with RCT2100, our inhaled mRNA programme, we are building a therapeutic franchise to expand disease-modifying treatment options for all people with CF.”

In February 2024, the company announced a strategic collaboration with Intellia Therapeutics to develop gene editing therapies for CF.

CF is a debilitating genetic disease leading to persistent lung infections and respiratory failure, stemming from mutations in the CFTR gene.

The company’s lead programmes, RCT2100 and RCT1100, target CFTR gene mutations unresponsive to approved modulators and primary ciliary dyskinesia, respectively. Both are inhaled mRNA-based therapies formulated with ReCode's SORT LNP delivery platform.

In January 2023, Asklepios BioPharmaceutical entered a research partnership with ReCode to explore a single-vector gene-editing platform.

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