Cystic Fibrosis (CF)
ReCode’s CFTR mRNA is an investigational therapy for CF that focuses specifically on the 10 to 13 percent of CF patients who have nonsense mutations and do not respond to currently approved CFTR modulators
Primary Ciliary Dyskinesia (PCD)
ReCode is developing DNAI1 mRNA for the treatment of PCD caused by mutations in DNAI1, an essential gene for ciliary movement
Opportunities in a Wide Array of Genetic Diseases
With versatility of genetic cargo and flexibility in administration, ReCode’s SORT LNP technology has potential applications across a broad range of genetically defined rare diseases, including respiratory, CNS, liver, and cancer
dyskinesia
(PCD) mRNA DNAI1 Inhaled
IND 2H 2023 mRNA PCD gene 2 Inhaled
Cystic fibrosis
(CF) mRNA CFTR Inhaled
Surfactant deficiencies mRNA Undisclosed IV
spleen Vaccines mRNA Undisclosed IM
liver Alpha-1 antitrypsin Gene correction SERPINA1 IV
cns Various Multiple Undisclosed Intrathecal
Indication
Primary ciliary dyskinesia (PCD)
Modality
mRNA
Target
DNAI1
Delivery
Inhaled
DISCOVERY
PRECLINICAL
CTA Q4 2022, IND 2H 2023
PHASE 1/2
CTA Q4 2022, IND 2H 2023
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
IND 2H 2023
PHASE 1/2
Modality
Gene correction
Target
CFTR
Delivery
IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Surfactant deficiencies
Modality
mRNA
Target
Undisclosed
Delivery
IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Vaccines
Modality
mRNA
Target
Undisclosed
Delivery
IM
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Alpha-1 antitrypsin
Modality
Gene correction
Target
SERPINA1
Delivery
IV
DISCOVERY
PRECLINICAL
PHASE 1/2
Indication
Various
Modality
Multiple
Target
Undisclosed
Delivery
Intrathecal
DISCOVERY
PRECLINICAL
PHASE 1/2