Hope from a Familiar Place
The APR-2020 treatment process involves collecting stem cells from a patient, inserting a functioning/healthy replacement RPS19 gene into those stem cells, ex vivo, and then reinfusing the ‘updated’ patient’s cells back into their body. The insertion of the healthy gene is accomplished by using a proprietary lentiviral vector as the delivery mechanism.
Once the stem cells containing the corrected gene have been re-infused intravenously into the patient, the cells integrate into the patient’s bone marrow cells. From there the cells repopulate the bone marrow and regenerate naturally, thereby restoring normal functionality and ‘curing’ the patient’s inability to produce red blood cells.
Since APR-2020 is autologous (uses the patient’s own cells), no-transplant or bone marrow donor is required. This gives APR-2020 a potential advantage because the patient’s immune system doesn’t have to be suppressed and there is no risk of graft vs. host disease. In addition, a reduced intensity protocol can be used with APR-2020 to ‘make room’ in the marrow for the new cells, rather than the myeloablative chemotherapy regiment required for an allogenic (donor) transplant.
This makes our method potentially far less toxic and beneficial to the patient in comparison to a bone marrow transplant or other therapies that require high doses of chemotherapy.
The IND for APR-2020 was filed with the FDA on August 4th 2025 and on September 3rd, 2025, the FDA issued Apriligen a Safe to Proceed letter for APR-2020. Phased FDA human clinical trials of APR-2020 are expected to begin in 1H2026. Trial sites have been selected in the United States. European trial sites are expected to open towards the end of 2H2027.
In addition to receiving Orphan Drug and Rare Pediatric Disease Designations from the FDA and EMA, in 1Q2026, APR-2020 was awarded Fast Track and RDEP designations, both of which provide an accelerated regulatory approval pathway.
Fast Track offers early and frequent communications with the FDA to rapidly resolve issues and questions, including a rolling review process, to expedite drug approval.
RDEP allows a program to receive approval through a single, well-controlled clinical trial.
Both programs were implemented and designed to support the development of medicines for rare diseases. APR-2020 is currently an investigational therapy.
Our Process
Red blood cell-generating stem and progenitor cells are withdrawn from a patient with RPS19-deficient DBA and combined with a delivery mechanism (the “Vector”) in a laboratory setting that contains a healthy copy of the RPS19 gene.
The Vector binds with the patient’s cells and introduces the corrected gene that in turn binds in the target location within the patient cells.
Because we are reintroducing the patient’s own cells back into the patient, the body and system may consider these cells native and a perfect match.
To ensure there is sufficient room in the bone marrow for the corrected stem and progenitor cells to grow naturally and to overtake defective cells, a reduced dose of bone marrow conditioning is administered prior to reinsertion.
Expanded access policy
Apriligen does not currently offer an expanded access program. While Apriligen is committed to making APR-2020 broadly accessible to the Diamond-Blackfan Anemia (DBA) patient population, our first priority is safety.
We believe this critical objective is best achieved through clinical trials that are well regulated and monitored. Subsequently, access to APR-2020 is currently possible only through participation in our clinical trial.
Pre-clinical testing of APR-2020 has shown positive outcomes in both research models and patient cells (ex. vivo).