Through innovative technologies and collaboration with leading academic institutions, we aim to harness the natural power of the human immune system for the treatment of cancer.

Despite major advances in the prevention, diagnosis and treatment of cancer, around 14.1 million patients are diagnosed and 8.2 million die each year worldwide. These statistics powerfully illustrate the need for new breakthroughs in the fight against cancer.

Engineered NKT Cell Therapies for Cancer

Cancers are usually characterized by rapid cell growth. In the early years of cancer research, this rapid growth was used as a marker to recognize cancer cells. Drugs, developed to target and kill all fast-growing cells (chemotherapy), often lead to undesired side effects, such as gastrointestinal toxicity, hematological toxicity and other problems, such as hair loss.  In some cases, toxicity from conventional chemotherapy drugs can cause serious harm or even be fatal.

Thanks to major advances in the understanding of the molecular biology of cancers specific cellular features have been discovered that are associated with certain cancers. These discoveries have allowed for the development of targeted cancer therapies, for example engineered T cell products. Targeted cancer therapies generally have fewer side effects compared to older chemotherapies.

We take this tactic even further by using engineered immune cells  (NKT cells) to target specific cancer molecules. This approach may bring two advantages:

  1. The engineered NKT cells have a prolonged life and can replicate in the patient, providing long term protection against newly developing cancer cells.
  2. The engineered NKT cells become part of the patient’s own immune system, making this a natural approach to the treatment of cancer.

Cancer cells express molecules either inside the cell or on the surface of the cancer cells that may be used as targets by engineered NKT cells.  The challenge for us is to train the engineered NKT cells to recognize and exclusively target the cancer cells and not recognize and attack normal tissues in the patient.

We basically use two different approaches to generate engineered NKT  products that can recognize and specifically targeting cancer cells.

  1. For cancer target molecules that are localized on the outside of the cancer cell, chimeric antigen receptor (CAR) technology is used. CAR technology involves combining different elements of normal immune system molecules in a way that allows immune cells to recognize specific cancer molecules on the outside of a cell.  Our CAR technology is unique in that we use a special type of immune cell, called a NKT lymphocyte to deliver the CAR to the tumor.  We have data to indicate that NKT lymphocytes are very efficient at getting to tumors in the body.
  2. To reach cancer molecules inside the cancer cell, we modify the T cell receptor (TCR) of immune cells called T lymphocytes.  The TCR technology is necessary for cancer molecules inside the cell because TCRs have evolved to recognize small parts of internal molecules that are presented on the surface of the cell by special immune molecules that recognize the TCR.  We have engineered TCRs to increase their numbers on the surface of lymphocytes, which should make them more active against cancer.  We call this technology “dominant TCR”.


Neuroblastoma is a cancer of the sympathetic nervous system which can occur in the chest, neck, abdomen and adrenal glands, and can metastasize to the bone marrow and other organs. Children with low or intermediate risk neuroblastoma can be cured through surgical intervention and/or chemotherapy, however, at least half of all children with neuroblastoma have high risk disease, which often requires combined surgical, radio-, immuno-, and chemotherapy, in addition to autologous stem cell transplantation.

GD2 is a molecule expressed on almost all neuroblastomas, and a substantial fraction of small cell lung cancer and melanoma, with restricted expression on normal tissues, making it a good target for CAR-NKT cell therapy.

We are developing CMD-501 for the treatment of high risk, relapsed, refractory neuroblastoma in children. CMD-501 is an autologous cell therapy targeting GD2, which utilizes our natural killer T (NKT) cell platform technology in combination with genetically engineered chimeric antigen receptors (CARs). A clinical trial is currently open to recruitment at Baylor College of Medicine and Texas Children’s Hospital, Houston, Texas.

More information about the Neuroblastoma Trial can be found here

See our press release regarding the first dosed patient here.