ReCode Therapeutics
Clinical-stage genetic medicines company developing selective organ-targeted lipid nanoparticle delivery and nucleic acid therapeutics (mRNA and gene correction). The company advances inhaled and systemic nucleic acid programs, conducts early-phase clinical trials, performs CMC and analytical development for clinical manufacturing, and collaborates with external partners and scientific advisors to expand delivery and gene editing capabilities.
Industries
Nr. of Employees
small (1-50)
Patents
Polynucleotide compositions, related formulations, and methods of use thereof
US-12257318-B2
View Details
Polynucleotide compositions, related formulations, and methods of use thereof
US-12257318-B2
View DetailsProducts
Inhaled mRNA therapeutic candidate for cystic fibrosis (clinical-stage)
Inhaled mRNA therapy designed to deliver CFTR mRNA to lung target cells to restore CFTR protein function for patients with CF mutations not addressed by current modulators.
Inhaled mRNA therapeutic candidate for primary ciliary dyskinesia (PCD)
Inhaled mRNA therapy delivering DNAI1 mRNA to restore ciliary function in the lungs for PCD caused by DNAI1 mutations.
Gene correction programs for cystic fibrosis (preclinical/early clinical collaboration)
Development of gene correction approaches using CRISPR-based tools and organ-targeted delivery to precisely edit CFTR mutations in airway cells.
Inhaled mRNA therapeutic candidate for cystic fibrosis (clinical-stage)
Inhaled mRNA therapy designed to deliver CFTR mRNA to lung target cells to restore CFTR protein function for patients with CF mutations not addressed by current modulators.
Inhaled mRNA therapeutic candidate for primary ciliary dyskinesia (PCD)
Inhaled mRNA therapy delivering DNAI1 mRNA to restore ciliary function in the lungs for PCD caused by DNAI1 mutations.
Gene correction programs for cystic fibrosis (preclinical/early clinical collaboration)
Development of gene correction approaches using CRISPR-based tools and organ-targeted delivery to precisely edit CFTR mutations in airway cells.
Services
Collaborative research and development relationships to apply selective organ-targeted lipid nanoparticle delivery to partner payloads and indications.
Clinical study execution support and opportunities for patient enrollment in Phase 1/1b studies for inhaled and systemic nucleic acid therapeutics.
Collaborative research and development relationships to apply selective organ-targeted lipid nanoparticle delivery to partner payloads and indications.
Clinical study execution support and opportunities for patient enrollment in Phase 1/1b studies for inhaled and systemic nucleic acid therapeutics.
Expertise Areas
- Targeted lipid nanoparticle delivery
- mRNA therapeutic design and modified mRNA chemistry
- In vivo gene correction and CRISPR-based editing
- LNP formulation and lipid/material chemistry
Key Technologies
- Lipid nanoparticle delivery
- mRNA therapeutics (modified nucleoside chemistry)
- Non-viral CRISPR/Cas delivery
- siRNA and DNA payloads
News & Updates
Announcement of Phase 2 clinical trial enrollment of an inhaled mRNA candidate in combination with a modulator for cystic fibrosis and additional funding/collaborations.
Company CEO participated in a virtual biotech symposium.
Company participation and presenter roster at an industry conference.
Press release announcing U.S. FDA Orphan Drug Designation for the inhaled mRNA therapeutic candidate for CF.
Peer-reviewed article on inhaled DNAI1 mRNA therapy for PCD published in PNAS.
Publication describing in vivo gene editing of lung stem cells, supporting durable gene correction (Science).
Announcement of Phase 2 clinical trial enrollment of an inhaled mRNA candidate in combination with a modulator for cystic fibrosis and additional funding/collaborations.
Company CEO participated in a virtual biotech symposium.
Company participation and presenter roster at an industry conference.
Press release announcing U.S. FDA Orphan Drug Designation for the inhaled mRNA therapeutic candidate for CF.
Peer-reviewed article on inhaled DNAI1 mRNA therapy for PCD published in PNAS.
Publication describing in vivo gene editing of lung stem cells, supporting durable gene correction (Science).