Clinical Trials in Cell and Gene Therapy

clinical trial is a research study that tests how well new medical approaches work on people and determines if a treatment is safe and effective. The new cell and gene therapies (CGTs) that are advancing from the laboratory into early phase clinical trials has proven to be a complex task even for experienced investigators . As a result of wide variety of CGT products and their potential applications, a case by case assessment is warranted for the design of each clinical trial. Some of the latest and advanced clinical trials include safety and efficacy trial of AAV gene therapy in patients with CNGA3 Achromatopsia, A clinical trial for treatment of Aromatic L- Amino acid Decarboxylase (AADC) deficiency using AAV2-hAADC- An expansion and Glypican 3-specific Chimeric antigenic receptor expressed in T cells for patients with pediatric solid tumors. 

  • Cell based immunotherapy
  • Target tissues & routes of delivery
  • New cell therapies for allergies
  • Cell based immunosuppression in transplantation
  • Cancer immune cells

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