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R&D Pipeline · RECORNA
Accelerating
Novel Therapies
for Unmet Needs
Development Progress
CNS-Targeted Therapies
Population
Indication
Mechanism
Discovery
Preclinical
IIT
Clinical
Dravet Syndrome / SCN1A
mRNA upregulation
Discovery
Preclinical
IIT
Clinical
Neurodegenerative Diseases
Introduce a protective mutation
Discovery
Preclinical
IIT
Clinical
Liver-Targeted Therapies
Population
Indication
Mechanism
Discovery
Preclinical
IIT
Clinical
Rare Metabolic Diseases
repair a pathogenic vatiant
Discovery
Preclinical
IIT
Clinical
Metabolic Disorders
repair a pathogenic vatiant
Discovery
Preclinical
IIT
Clinical
Cardiovascular Diseases
mRNA Upregulation
Discovery
Preclinical
IIT
Clinical
Anti-Aging Research
Introduce a beneficial mutation
Discovery
Preclinical
IIT
Clinical