Pipeline
Primary Ciliary Dyskinesia (PCD)
RCT1100 is an inhaled mRNA-based investigational therapy for PCD caused by mutations in the DNAI1 gene. PCD is a rare and progressive genetic disorder that leads to chronic respiratory infections and loss of lung function.Cystic Fibrosis (CF)
RCT2100 is an inhaled mRNA-based investigational therapy for CF that focuses specifically on the 10-13% of patients with nonsense mutations that do not respond to approved CFTR modulators.Opportunities in a Wide Array of Genetic Diseases
With the versatility of genetic cargo and flexibility in administration, ReCode’s SORT LNP technology has potential applications across many genetically defined conditions, including muscular, CNS, liver, infectious disease and oncology indications.dyskinesia
(PCD) mRNA DNAI1 Inhaled
Cystic fibrosis
(CF) mRNA CFTR Inhaled
Alpha-1 antitrypsin Gene correction SERPINA1 Inhaled
cns Various Multiple Undisclosed Intrathecal
Indication
Primary ciliary dyskinesia (PCD)
Modality
mRNA
Target
DNAI1
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Modality
mRNA
Target
PCD gene 2
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Modality
mRNA
Target
PCD gene 3
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Cystic Fibrosis (CF)
Modality
mRNA
Target
CFTR
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Modality
Gene correction
Target
CFTR
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Alpha-1 antitrypsin
Modality
Gene correction
Target
SERPINA1
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Various
Modality
Multiple
Target
Undisclosed
Delivery
Intrathecal
DISCOVERY
PRECLINICAL
PHASE 1/2