ReCode Therapeutics Secures $29M Financing to Advance Inhaled mRNA Therapy for Cystic Fibrosis
核心洞察
ReCode Therapeutics (搜索) raised over $29 million in additional financing to advance its genetic medicines pipeline, including investigational therapies for cystic fibrosis (搜索).
The Cystic Fibrosis Foundation committed an additional $3 million to support the ongoing Phase 2 clinical trial of RCT2100, bringing total CF Foundation investment to up to $33 million.
RCT2100 is designed to deliver functional CFTR (搜索) protein via inhaled mRNA therapy, potentially benefiting all CF patients including those with rare mutations who don't respond to existing modulator therapies.
ReCode Therapeutics (搜索), a clinical-stage genetic medicines company, announced the completion of over $29 million in additional financing to advance its pipeline of genetic medicines, with particular focus on investigational therapies for cystic fibrosis (搜索) (CF). The funding comes alongside expanded support from the Cystic Fibrosis Foundation and a new research collaboration with Praxis Precision Medicines.
"With continued support from organizations like the CF Foundation and our collaboration with Praxis, we are building on our momentum to deliver on the promise of genetic medicines for people living with genetic diseases who currently have limited or no effective treatments," said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics (搜索).
Expanded CF Foundation Support Reaches $33 Million
The Cystic Fibrosis Foundation has committed an additional $3 million to support ReCode's ongoing Phase 2 clinical trial of RCT2100, an inhaled mRNA therapy for cystic fibrosis (搜索). This follows the Foundation's previous $15 million investment to support the development and early-stage clinical trials of the investigational treatment. In total, the CF Foundation has agreed to invest up to $33 million in ReCode's mRNA and gene editing research programs.
RCT2100 is designed to provide functional CFTR (搜索) protein by delivering a correct copy of CFTR mRNA to lung cells. The therapy offers potential benefits to all people with CF, including those with rare and nonsense mutations who do not benefit from existing modulator therapies. This addresses a significant unmet medical need, as approximately 10% of people with cystic fibrosis (搜索) have genetic mutations in the CFTR gene that do not respond to currently approved CFTR modulators or are intolerant of approved CFTR modulators.
Proprietary SORT Platform Enables Precision Delivery
ReCode's approach leverages its proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform, which enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease. This precision delivery system is designed to improve efficacy and potency of genetic medicines.
The company's lead programs include RCT2100 for cystic fibrosis (搜索) treatment and RCT1100 for the treatment of primary ciliary dyskinesia (搜索) caused by pathogenic mutations in the DNAI1 (搜索) gene. Both RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated with the SORT LNP delivery platform.
Strategic Collaboration with Praxis Precision Medicines
ReCode has entered into a research collaboration with Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance. The collaboration aims to identify a well-tolerated lipid nanoparticle formulation that enhances the delivery of antisense oligonucleotides (ASOs) to underexposed brain regions.
Strengthened Financial Position for Clinical Advancement
The over $29 million in new financing strengthens ReCode's financial foundation as it progresses its clinical and preclinical programs. The funding will support the advancement of the company's genetic medicines pipeline, with particular emphasis on continuing development of therapies for patients with genetic diseases who currently have limited or no effective treatments.
ReCode leadership plans to attend the European Respiratory Society (ERS) Congress in Amsterdam, Netherlands, from September 27 to October 1, and the North American Cystic Fibrosis (搜索) Conference (NACFC) in Seattle from October 22-25, providing opportunities to share updates on their clinical programs with the scientific community.