Apriligen’s mission is to cure rare, monogenic pediatric diseases. Our first clinical gene therapy program, APR-2020, is a purpose-built lentiviral vector to treat RPS19 deficient Diamond Blackfan Anemia (“DBA”).
Apriligen’s mission is to cure rare, monogenic pediatric diseases. Our first clinical gene therapy program, APR-2020, is a purpose-built lentiviral vector to treat RPS19 deficient Diamond Blackfan Anemia (“DBA”).