Gene-Edited Cell Therapy

Gene editing tools like Crisper-Cas9 has allowed the precise gene editing possible in the field of biology.

We plan to develop multiple products using this technology to treating cancers and genetic disorders. Gene-edited adult hematopoietic stem cells for treating Thalassemia and Sickle cell disease, gene edited allogenic CAR T cells for treating different cancers, and gene edited cells for treating monogenic genetic disorders are in our priority.

Is a progenitor cell engineered cell therapy for treating beta Thalassemia, which is a genetic disorder caused by errors in the genes for hemoglobin, a substance composed of a protein (globin) plus an iron molecule (heme) that is responsible for carrying oxygen within the red blood cell. Genetic mutation in globin gene leads to fewer healthy red blood cells and less hemoglobin than normal. People who have thalassemias can have mild or severe anemia. Normal hemoglobin, also called hemoglobin A, has four protein chains—two alpha globin and two beta globin. The two major types of thalassemia, alpha and beta, are named after defects in these protein chains. Four genes (two from each parent) are needed to make enough alpha globin protein chains. Alpha thalassemia trait occurs if one or two of the four genes are missing. If more than two genes are missing, moderate to severe anemia occurs. The most severe form of alpha thalassemia is called alpha thalassemia major or hydrops fetalis. Babies who have this disorder usually die before or shortly after birth. Two genes (one from each parent) are needed to make enough beta globin protein chains. Beta thalassemia occurs if one or both genes are altered. The severity of beta thalassemia depends on how much one or both genes are affected. If both genes are affected, the result is moderate to severe anemia. The severe form of beta thalassemia is known as thalassemia major or Cooley's anemia. ThalaGenX is a globin gene engineered progenitor cell therapy for treating patients with Beta thalassemia. Our goal to bring a durable cure by increasing the beta globin production and transfusion independence.

Is a novel gene-edited and CAR engineered allogenic T cells therapy for treating specific type of lymphoma and leukemia. It has several advantage as it eliminates the blood collection from each patient to develop CAR T cell therapy. It will be very useful for patients with advanced disease and collapsed veins following extensive chemotherapy. Multiple doses can be prepared and stored in suitable condition for ready to use for any patient. It will be an “off the shelf product’ that could be used on any patient at any time. This is very advanced technology which is made possible due to the availability of reliable gene-editing tools. At Biomac, the Team is working to bring this in to the clinic for treating cancer patients.