Based in Connecticut, Apriligen, Inc. was founded in 2020 to commercialize a proven and scalable lentiviral gene therapy platform developed in Germany and Sweden to cure ultra rare, monogenic illnesses. 

Apriligen's lead therapy begins its pivotal clinical trial in 2H2025 to cure a bone marrow failure syndrome called Diamond-Blackfan Anemia (“DBA”).   With approximately 5,000 – 10,000 patients world-wide, DBA is an ultra-rare, terminal, pediatric bone marrow failure syndrome that renders the patient unable to produce red blood cells. There are over 20 variants of DBA, each of which is caused by the failure of a unique gene.

The Company’s first therapeutic candidate, APR-2020, is an autologous, ex-vivo gene-altered hematopoietic stem cell product targeting the RPS19 genetic variant (representing approximately 35% of the total global DBA population).  APR-2020 works by transfecting (inserting) a corrected RPS19 gene into the patient’s own stem cells, ex-vivo, and then reintroducing the corrected cells back into the patient to repopulate the bone marrow.  The ex-vivo correction is accomplished using Apriligen’s proprietary lentiviral vector platform and process. Once engrafted into the marrow, the “fixed” cells express sufficient amounts of RPS19 to allow normal red blood cell production.  These cells self-renew and sustain normal red blood cell production throughout the remainder of the patient’s life.  APR-2020 is expected to require a single treatment and offer a life-long, curative solution.  

As a curative gene therapy, APR-2020 has been shown to restore normal function to the patient's own hematopoietic cells.  Therefore, APR-2020 offers a paradigm-changing improvement in patient care over the current standard by eliminating the need for transfusion therapy, a bone marrow donor, the risks associated with transplanting foreign cells (such as Graft vs. Host Disease, long term immunosuppressants, etc.), reducing exposure to chemotherapy and vastly improving recovery and quality of life measures.