Company Overview

Helix Therapeutics is a seed stage company based on technology discovered in the laboratory of Peter Glazer MD/PhD and licensed on a world wide, exclusive basis from Yale University. Targeted DNA modifications can be designed to:

  • » correct mutations, leading to restoration of normal function of key gene products
  • » alter levels of gene expression, potentially turning genes on or off
  • » functionally disrupt molecules, such as the CCR5 receptor, which mediates HIV entry into T cells

Through the use of SBIR grants and seed funding, Helix will advance preclinical projects targeting the human globin gene for applications on sickle cell anemia and beta-thalassemia, the CCR5 receptor for the HIV/AIDS population, and genetic mutations responsible for the lysosomal storage disease, Gaucher’s.

Genetic alteration of stem cells has the potential to dramatically impact the efficacy of stem cell therapy for human disease. Due to the accessibility of HSC without bone marrow transplants, the Helix technology is currently focused on blood based genetic diseases. As advances in human stem cell biology are realized, however, it is possible to envision applications in a broad array of human genetic diseases. Moreover, because the DNA modifications introduced to stem cells are stable and heritable, this technology offers the hope of curing certain genetic diseases.

The Helix approach does not rely on viral vectors and corrects the patients’ own cells, eliminating potential immune reactions. This approach is classified as Cellular Therapy, which provides another level of safety to the patient.