Genome editing can be delivered inside the body – by injection. This is called in vivo therapy…
or the therapy can be applied to a sample of a patient’s cells, which are then re-infused. This is called ex vivo therapy.
Focus in these disease areas requires the team to eschew assumptions and overcome obstacles that stand in the way of effective therapies.
In vivo and ex vivo programs require different competencies in research, manufacturing and commercialization.
eXtellia focuses on the research and development of ex vivo therapeutic applications of CRISPR/Cas9. With eXtellia, our vision is to create the world’s premier engineered cell therapy company using the potential of CRISPR/Cas9 to deliver cellular medicines. Engineered cells promise to transform clinical medicine.
eXtellia is focused on diseases with high unmet medical need where genome edited cell therapy offers a powerful and differentiated therapeutic modality.
Modulating the immune system to eliminate cancerous tumors or to arrest auto-immune diseases
In oncology, we aim to discover and unlock the capabilities of the body’s immune system – selecting, preparing and directing these cells to destroy cancerous tumors. In autoimmunity, we strive to achieve the opposite – modulating the patient’s immune cells to prevent them from destroying self-tissues.
Engineered cells promise to transform clinical medicine
eXtellia’s ex vivo programs in immuno-oncology and autoimmune diseases will help broaden the application of CRISPR/Cas9. Longer-term, we expect eXtellia to focus on other applications and indications, such as induced pluripotent stem cells, mesenchymal stem cells and muscle satellite stem cells for tissue-targeted treatments.
Improving the way ex vivo therapies are administered
We also are working to improve and simplify the way ex vivo therapies are administered.
Today, cell therapies are approved only for patients with hematological cancers. We envision a better way. There are several steps to generate the treatment for administration. At the first visit to the clinic, blood is withdrawn from the patient and separated. The relevant immune cells are isolated and genetically engineered to target the cancer. These engineered cells are allowed to divide and grow to increase their number before the patient returns to the clinic to have their own modified cells reinfused. The overall process is time-consuming and cannot be performed for all patients.
For example, patients may not have sufficient healthy cells that can be genetically engineered for re-infusion and, if healthy donor cells are used, there are chances of rejection by the patient’s body, which could lead to even further health complications and risks.
In the short term, eXtellia intends to use CRISPR/Cas9 to optimize the engineering of the selected immune cells, and to improve their performance by CRISPR/Cas9 genome editing. These improvements are aimed at yielding immune cells that will more effectively identify and attack cancerous cells upon re-infusion.
In our vision for the future, patients would need a single visit to the clinic for administration of an off-the-shelf (e.g., allogeneic) cellular medicine not derived from the cells of a particular patient. Applying CRISPR/Cas9 to human cells, we intend to pre-manufacture targeted cellular therapies that can be administered to patients based on their genetic needs. These therapies would address a broader range of both hematological and solid cancers even on individuals with depleted or low levels of healthy cells.