ReCode Therapeutics Secures Funding, Partners with Gene Editing Firm to Fast-Track Cystic Fibrosis Gene Therapy with SORT LNP Platform

August 17, 2026

BioSpace (via Business Wire) USA

Overview

ReCode Therapeutics has announced new funding from the Cystic Fibrosis Foundation and a strategic partnership with a leading gene editing company. These initiatives will accelerate the development of their proprietary Selective Organ Targeting (SORT) Lipid Nanoparticle (LNP) platform, which precisely delivers genetic medicines to disease-relevant cells. With their lead program, RCT2100 for cystic fibrosis, having fully enrolled its FDA Fast Track-designated Phase 2a trial, ReCode is poised to advance innovative therapies for genetic diseases.

In Depth

Key Announcements

ReCode Therapeutics has announced significant new funding from the Cystic Fibrosis Foundation and a strategic partnership with a leading gene editing company. These developments are set to further advance the company’s groundbreaking Selective Organ Targeting (SORT) Lipid Nanoparticle (LNP) platform, accelerating the development of genetic medicines for inherited diseases, including cystic fibrosis (CF). Notably, the Phase 2a trial for RCT2100, ReCode’s lead program, has now fully enrolled, signifying steady progress in its clinical development.

Background and Industry Context

While gene editing and mRNA therapies hold immense promise for treating genetic diseases, a significant challenge remains: precisely controlling “where, how much, and when” these therapeutics are delivered. Lipid nanoparticles (LNPs) have proven their efficacy as a primary delivery system in mRNA vaccines; however, most systemically administered LNPs tend to accumulate in the liver. ReCode’s SORT LNP technology offers a breakthrough approach to overcome this limitation, enabling targeted delivery to specific organs beyond the liver, particularly the lungs. The funding from the Cystic Fibrosis Foundation and the partnership with a leading gene editing company clearly underscore the potential value of this technology and the substantial unmet need in cystic fibrosis treatment. These partnerships are crucial for accelerating technological development and expanding the scope of clinical applications.

The SORT LNP Platform: A Technical Overview

ReCode Therapeutics’ SORT LNP platform represents a unique technology designed for the highly precise delivery of genetic medicines—specifically mRNA, siRNA, and CRISPR gene editing tools—to organs, tissues, and cells specifically implicated in disease. This proprietary platform precisely modifies the lipid composition and surface characteristics of LNPs, enabling them to recognize specific cellular receptors and molecular markers. This dramatically enhances uptake efficiency into target organs, minimizing off-target effects of therapeutic agents while maximizing drug concentration at the disease site. The expected outcome is significantly improved therapeutic efficacy and safety.

Clinical Development: RCT2100 for Cystic Fibrosis

RCT2100, ReCode’s lead therapeutic candidate, is a treatment for cystic fibrosis (CF) and has already received Fast Track designation from the FDA. Cystic fibrosis is a genetic disorder caused by mutations in the CFTR gene, leading to severe symptoms primarily affecting the lungs and digestive system. RCT2100 leverages the SORT LNP platform to deliver functional CFTR mRNA to lung cells, aiming to address the root cause of the disease by complementing the defective protein. The ongoing Phase 2a trial has now fully enrolled patients, and data regarding safety, pharmacokinetics, and initial efficacy are anticipated to be announced in due course.

Future Outlook

The results from the RCT2100 Phase 2a trial will represent a crucial milestone, not only for ReCode Therapeutics but also for the broader SORT LNP platform technology. Should promising data emerge, it would significantly broaden the potential application of this technology to various genetic diseases beyond cystic fibrosis and to therapies targeting other organs. Through new funding and partnerships, ReCode aims to further strengthen its research and development pipeline and establish itself as a leader in the targeted delivery of genetic medicines. The long-term vision is to provide safer and more effective gene therapies to a greater number of patients.

Source: https://www.biospace.com/press-releases/recode-therapeutics-announces-new-funding-from-the-cystic-fibrosis-foundation-collaboration-with-a-leading-gene-editing-company-and-leadership-transition

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