Geoff MacKay, president and CEO of Avrobio, a gene therapy company focused on lysosomal diseases talks about the FDA’s guidances they have published to help industry and researchers develop gene therapies.

As MacKay explains in this video, the guidances have been very beneficial for all parties involved in developing and marketing gene therapies.

The guidances cover a number of issues including:

  • Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
  • Long Term Follow-up After Administration of Human Gene Therapy Products
  • Testing of Retroviral Vector-Based Human Gene Therapy Products
  • Human Gene Therapy for Hemophilia
  • Human Gene Therapy for Rare Diseases
  • Human Gene Therapy for Retinal Disorders


  • Sign up
Lost your password? Please enter your username or email address. You will receive a link to create a new password via email.
We do not share your personal details with anyone.