As if every child is our own

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 goal is to develop therapies for pediatric monogenic illnesses that have no cure.

Our first program, APR-2020, is an autologous solution for RPS19 deficient Diamond Blackfan Anemia Syndrome patients. We believe this platform can be applied to many DBA variants as well as other monogenic illnesses.

Once approved for RPS19, Apriligen’s long-term vision is to leverage its lentiviral platform and seek additional regulatory approvals for applications in rare diseases beyond DBA.

The Challenge

Treating rare, monogenic pediatric diseases like DBAS currently involves harsh side effects. We are dedicated to providing a safer, curative path for these rare patients.

Therapy

Our autologous gene therapy uses a patient’s own cells to restore function, avoiding the toxicity and risks of traditional transplants.

Our Vision

We strive for “More Cures, More Birthdays,” creating a scalable platform to finally make gene therapy accessible for rare diseases.

Our Progress

With FDA Fast Track status and clinical trials launching in 2026, we are rapidly advancing towards a life-saving solution.

Science

Backed by twenty years of research, we utilise proven lentiviral technology to precisely correct genetic defects at their source.

Pre-clinical testing has shown that APR-2020 works in both research models and patient cells (ex. vivo).

Clinical trial enrollment for APR-2020 has begun and the trial is ongoing. The goal of this trial is to prove that APR-2020 ‘cures’ the RPS19 variant of DBAS and results in permanent and normal bone marrow function without graft vs. host disease, the need for long-term immunosuppressants or high intensity chemotherapy as required for allogeneic bone marrow transplants.