Pipeline

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A Pipeline Built for Scale – and for the Individual Patient.

Therna is advancing programmable RNA medicines across rare genetic disorders, immunological disorders, and cardiovascular disease, alongside single-patient programs for ultra-rare conditions.

Therapeutic Pipeline

Immunology, Metabolism and Cardiovascular Target & Biology Selection Candidate Design & Optimization Experimental Validation Preclinical Development Readiness
Immunological Diseases
Metabolic Diseases
Cardiovascular Diseases

Single-Patient Programs

Therna is selectively pursuing single-patient programs for ultra-rare conditions where conventional clinical development is not feasible. These efforts apply the RNA-Logix™ capabilities while generating biological insights that strengthen model performance across the broader pipeline.

Immunology, Metabolism and Cardiovascular Preclinical Development IND-Enabling Studies Investigational New Drug (IND) Clearance Dosing Single Patient
Pulmonary Fibrosis
Lamb Shaffer Syndrome
(Neurodevelopment Disorder)