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% of people with CF with 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.(CF) mRNA CFTR Inhaled
Primary ciliary
dyskinesia
(PCD) mRNA DNAI1 Inhaled
Other lung indications Multiple Undisclosed Inhaled IV
Other mRNA Undisclosed Inhaled IV
liver Various Multiple Undisclosed IV
cns Various Multiple Undisclosed Intrathecal
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
Primary ciliary dyskinesia (PCD)
Modality
mRNA
Target
DNAI1
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Modality
mRNA
Target
CCDC39/40
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Modality
mRNA
Target
DNAH5
Delivery
Inhaled
DISCOVERY
PRECLINICAL
PHASE 1/2
Other lung indications
Modality
Multiple
Target
Undisclosed
Delivery
Inhaled IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Other
Modality
mRNA
Target
Undisclosed
Delivery
Inhaled IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Modality
Multiple
Target
Undisclosed
Delivery
Inhaled IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Various
Modality
Multiple
Target
Undisclosed
Delivery
IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Various
Modality
Multiple
Target
Undisclosed
Delivery
Intrathecal
DISCOVERY
PRECLINICAL
PHASE 1/2