ReCode Therapeutics
Clinical-stage genetic medicines company developing mRNA and gene-correction therapeutics with a focus on selective delivery to target organs and cell types beyond the liver. The company engineers lipid-nanoparticle delivery systems for multiple nucleic acid cargo types, conducts preclinical and multi-site clinical trials for respiratory genetic diseases (including cystic fibrosis and primary ciliary dyskinesia), and pursues collaborations for gene editing and antisense oligonucleotide delivery.
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
RCT1100
Investigational inhaled mRNA candidate developed to restore ciliary protein function in primary ciliary dyskinesia caused by pathogenic mutations in DNAI1 and related targets; evaluated in Phase 1/1b clinical trials with reported airway protein expression and functional readouts.
RCT2100
Investigational inhaled CFTR mRNA candidate for cystic fibrosis patients with mutations not responsive to approved CFTR modulators; under Phase 1b/Phase 2 clinical development and granted orphan drug designation by the U.S. FDA.
Inhaled DNAI1 mRNA therapy
Inhaled mRNA therapy developed for the treatment of primary ciliary dyskinesia using precision-engineered SORT LNPs.
SORT LNPs
Selective organ targeting lipid nanoparticle platform engineered with a biochemically distinct fifth lipid to direct genetic medicines to targeted organs such as the lung and spleen.
RCT1100
Investigational inhaled mRNA candidate developed to restore ciliary protein function in primary ciliary dyskinesia caused by pathogenic mutations in DNAI1 and related targets; evaluated in Phase 1/1b clinical trials with reported airway protein expression and functional readouts.
RCT2100
Investigational inhaled CFTR mRNA candidate for cystic fibrosis patients with mutations not responsive to approved CFTR modulators; under Phase 1b/Phase 2 clinical development and granted orphan drug designation by the U.S. FDA.
Inhaled DNAI1 mRNA therapy
Inhaled mRNA therapy developed for the treatment of primary ciliary dyskinesia using precision-engineered SORT LNPs.
SORT LNPs
Selective organ targeting lipid nanoparticle platform engineered with a biochemically distinct fifth lipid to direct genetic medicines to targeted organs such as the lung and spleen.
Expertise Areas
- Lipid nanoparticle delivery and formulation
- Nucleic acid therapeutic design (mRNA, siRNA, DNA, ASO)
- In vivo gene editing and DNA-writing approaches
- Preclinical respiratory disease models
Key Technologies
- Lipid nanoparticle delivery
- mRNA therapeutics
- siRNA therapeutics
- DNA payloads and gene-correction methods
News & Updates
Presentation of Phase 1b clinical data demonstrating inhaled mRNA delivery, protein expression and restoration of ciliary activity in airways of PCD patients; 57% of patients showed improvement in mucociliary clearance at 12 weeks; reported safety and tolerability.
U.S. FDA granted Orphan Drug Designation for an inhaled CFTR mRNA candidate intended to treat cystic fibrosis patients who do not respond to approved modulators.
Series B financing extension and subsequent funding rounds and foundation support to advance clinical and gene-correction programs.
Funding commitments from a disease foundation and a research collaboration with a gene-editing company to develop gene-correction therapies for cystic fibrosis, with subsequent increases in foundation support.
First patient dosed in a multi-dose Phase 1b study evaluating safety and tolerability of an inhaled CFTR mRNA therapeutic; study authorized across multiple regions.
Regulatory clearance to initiate a Phase 2 trial evaluating inhaled CFTR mRNA therapy in combination with an approved CFTR potentiator; U.S. sites opened for enrollment.
Presentation of Phase 1b clinical data demonstrating inhaled mRNA delivery, protein expression and restoration of ciliary activity in airways of PCD patients; 57% of patients showed improvement in mucociliary clearance at 12 weeks; reported safety and tolerability.
U.S. FDA granted Orphan Drug Designation for an inhaled CFTR mRNA candidate intended to treat cystic fibrosis patients who do not respond to approved modulators.
Series B financing extension and subsequent funding rounds and foundation support to advance clinical and gene-correction programs.
Funding commitments from a disease foundation and a research collaboration with a gene-editing company to develop gene-correction therapies for cystic fibrosis, with subsequent increases in foundation support.
First patient dosed in a multi-dose Phase 1b study evaluating safety and tolerability of an inhaled CFTR mRNA therapeutic; study authorized across multiple regions.
Regulatory clearance to initiate a Phase 2 trial evaluating inhaled CFTR mRNA therapy in combination with an approved CFTR potentiator; U.S. sites opened for enrollment.