Session 2C

Symposium: “Rare Genetic Disorders” organized by Sudha Bhattacharya, JNU, New Delhi

Translational and Manufacturing of Viral Vectors for Gene Therapy to Correct Genetic Disorders

Rare genetic disorders was a neglected field due to lack of market potential.  Gene therapy has given some hope to correct some rare genetic disorders.   Safety and regulatory issues have hindered the progress of gene therapy during the last two decades.  Of late, more than twenty products have been licensed for  use as therapy for  genetic disorders.  Several  of the products use different viral vectors. The vectors have to be manufactured in cGMP manufacturing facility and strict quality control and quality assurance have to be established.  India needs to gear up in translational research and manufacturing of viral vectors.  Manufacturing, quality control, quality assurance and regulatory issues will be discussed briefly for viral vectors that are required for Gene therapy for correcting genetic disorders.