In the pipeline: Gene therapies

There are thousands of genetic diseases caused by mutations in single genes. Patients with these diseases are not yet well served by therapies.

There are treatments for a few diseases, but they may alleviate symptoms temporarily or require organ, bone marrow, or stem cell transplants. These are costly, time-consuming, and logistically challenging—not to mention incredibly demanding on the patient. 

We aspire to develop our technology to edit the specifically targeted gene to cure such diseases by simple injections of mRNA/LNP formulations consisting of the gene and molecular machinery for its integration into the genome.

Our sickle cell disease gene therapy candidate

Sickle cell disease affects about 100,000 people in just the United States and is prevalent in people of African and Middle Eastern descent. There is no cure for sickle cell disease, and current treatments focus on managing the pain crises and other effects such as anemia. 

Current treatment regimens—including blood transfusions and bone marrow transplants—are costly, invasive, and impractical for treating large segments of affected patient populations, especially in low-income countries. Gene therapies currently in development for sickle cell disease are cell therapies, which require facilities close to the patient that can edit the cells outside of the body, posing an additional challenge for populations in remote areas or without adequate facilities to perform the editing.

Current approaches to gene therapy have challenges to overcome. Therapies that use adeno-associated viruses (AAVs) as vectors can encapsulate and deliver genetic material of up to 5,000 base pairs only, which limits the diseases it can be applied to.

Our RNA-based gene therapy candidate is designed to deliver a healthy copy of the gene to stem cells. Our concept of simple injections of mRNA/lipid nanoparticle formulations is a treatment method we are actively researching to treat sickle cell anemia, and we have achieved a Bill & Melinda Gates Foundation milestone, allowing for the next phase of grant work.

The main attributes of our gene therapy concept are expected to be:

• Accessible: Based on our cost-competitive RNA platform and with an in vivo administration, our therapy is intended to bypass the need for facilities required to edit the cells ex vivo.
• Targeted: The delivery technology targets specific cells in tissue.
• One dose and done: Our strategy is to target precursor stem cells to provide long-lasting expression.
• Versatile: Our therapy has the potential to encode for full-length genes and address genetic indications that require therapy in nondividing cells.