APR-2020
RPS19-deficient Diamond Blackfan Anemia (DBA)
Phase 1Active
Key Facts
About Apriligen
Apriligen is a private, pre-revenue biotech founded in 2021 and based in San Diego, focused on developing curative gene therapies for rare pediatric diseases. Its lead candidate, APR-2020, has received multiple regulatory designations (Orphan Drug, Fast Track, Rare Pediatric Disease, RDEP) from the FDA and EMA, with an IND cleared and Phase 1 trials set to begin in Q1 2026. The company's autologous lentiviral vector platform offers a potentially less toxic alternative to bone marrow transplant for DBA and could be expanded to other monogenic illnesses.
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