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Efficient Delivery of Target Genes for HSC and T Cell Based Therapies

Optimized Cocal vector and producer cell line for pseudotyping of therapeutic vectors.

  • Stage: Clinical
  • Type: Therapeutic, Cell Therapy, Gene Therapy
  • Categories: Nucleic Acid, Vectors

Technology Overview

Gene and cell therapy-based therapeutics to address orphan monogenic diseases, viral diseases, and CAR T and engineered TCR immunotherapy are dependent on efficient engineering of the target cell population. Lentiviral vectors (LVs) are the current standard for manipulation of hematopoietic stem cells (HSCs) and T cells. Dr. Kiem’s research group has demonstrated that Cocal pseudotyped lentiviral vectors exhibit higher titers, broader species and cell-type tropism, and improved serum stability and decreased serum neutralization in patient samples, compared to the current gold standard, VSV-G. These advantages allow for efficient manipulation in vivo or ex vivo of the target cell population resulting in higher engraftment in vivo. In recent studies, Dr. Kiem has demonstrated that cocal LVs transduce CD34+ and CD4+ T cells with greater efficiency than the commonly used pseudotype VSV-G lentivirus.

Applications

  • Efficient in vivo gene therapy
  • Engineering HSCs and T cells ex vivo for autologous cell therapy

Advantages

  • Cocal LVs transduce human, nonhuman primate, and canine HSCs allow for streamlined preclinical to clinical translation
  • Validated producer cell line that stably expresses Cocal envelope for scalable manufacturing
  • Optimized Cocal envelope outcompetes VSV-G in head to head comparisons

Patent Information

  • EP 2427577
  • WO 2016118775

Market Overview

Autologous stem cell and non-stem cell based therapies utilize patient cells which are cultured, modified, and then reintroduced into the donor patient’s body. The autologous cell therapy is expected to represent a $2.2 billion industry by 2017.

Investigator Overview

Hans-Peter Kiem, MD, PhD, Clinical Research Division
Tech ID: 09-012
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