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Pharmaceutical industry

Genetic Therapy, Inc. Initiates First Clinical Trial for Gene Therapy Treatment of Gaucher Disease

Strategic Insights

— 1 min read

Genetic Therapy, Inc. (Nasdaq: GTII) announced the initiation of the first clinical trial of a human gene therapy treatment for Gaucher disease, a genetic disorder that affects approximately 20,000 patients in the U.S. alone, including as many as 5,000 severe cases. The trial involves a total of 24 patients and is being conducted at the National Institutes of Health (NIH) and the Childrens Hospital Los Angeles. The goal of the trial is to incorporate a normal GC gene into precursor blood cells that have been removed from the patient's bone marrow or directly from peripheral blood, with the hope that the modified cells will produce GC in the blood.

Key Takeaways:

  • The clinical trial is focused on developing a gene therapy treatment for Gaucher disease, a genetic disorder that affects approximately 20,000 patients in the U.S. alone, including as many as 5,000 severe cases.
  • The trial involves a total of 24 patients and is being conducted at the National Institutes of Health (NIH) and the Childrens Hospital Los Angeles.
  • Genetic Therapy, Inc. is collaborating with a team of clinicians, including Principle Investigators Stefan Karlsson, M.D., Ph.D., Cynthia Dunbar, M.D., and Donald B. Kohn, M.D.
  • The Company is using a vector delivery system produced by Genetic Therapy, Inc. to modify the blood cells with a normal GC gene.
  • The trial is a Phase I/II clinical trial, indicating that the primary goal is to assess the safety and efficacy of the treatment.

Statistics:

  • 20,000: The estimated number of patients in the U.S. affected by Gaucher disease.
  • 5,000: The estimated number of severe cases of Gaucher disease in the U.S.
  • 24: The total number of patients involved in the clinical trial.
  • 2: The number of institutions conducting the trial (National Institutes of Health and Childrens Hospital Los Angeles).

Sources:

  • (Health Wire)--May 19, 1995
  • Genetic Therapy, Inc. (Nasdaq: GTII)
  • National Institutes of Health (NIH)
  • Childrens Hospital Los Angeles

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