ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company developing mRNA and gene correction therapeutics using its proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform. The company's technology enables precise delivery of genetic medicines to targeted organs and cells beyond the liver, potentially improving efficacy and potency. ReCode's lead programs focus on primary ciliary dyskinesia (PCD) and cystic fibrosis (CF).

RCT1100, ReCode's lead candidate for PCD caused by DNAI1 gene mutations, is an inhaled mRNA therapy designed to restore proper ciliary function. In January 2024, ReCode dosed the first patient in a Phase 1 clinical trial of RCT1100. The company is also developing RCT2100 for CF patients with Class I CFTR mutations who do not respond to current modulators, representing 10-13% of CF patients. Both therapies utilize ReCode's SORT LNP platform for targeted delivery.

preclinical data presented in 2022 showed that ReCode's CFTR mRNA encapsulated in SORT LNPs could be nebulized, delivered as an aerosol, and rescue CFTR function in cells from CF patients to levels comparable with approved modulators. In June 2022, ReCode published research in Science demonstrating its SORT LNPs could facilitate high levels of persistent gene editing in lung stem cells when given intravenously, achieving over 70% editing efficiency sustained for up to 660 days in mice.

Key customers and partnerships

In January 2023, ReCode announced a strategic investment of up to USD 15 million from the Cystic Fibrosis Foundation to support development of its inhaled mRNA-based CF therapeutic. The investment includes USD 10 million upfront to fund preclinical studies and early clinical development, with an additional USD 5 million upon meeting milestones.

ReCode entered a multi-year research collaboration with Asklepios BioPharmaceutical (AskBio) in January 2023 to develop a novel platform for full gene insertion using single vector delivery of gene editing and DNA cargoes. The partnership aims to combine AskBio's synthetic DNA and gene editing expertise with ReCode's SORT LNP technology to enable precision delivery of both gene editing tools and DNA in a single LNP to desired targets.