Prior to the COVID-19 pandemic, much research was devoted to the study of gene therapy and, in particular, to adeno-associated virus vector gene therapy. The research was carried out in oncology, hemophilia, and other serious and/or orphan diseases, however, approved FDA did not take place. Gene therapy appears to eventually become a reality.

We recommend great publications on that hide nothing:

Here you can find publications with attachments:

  • πŸ”₯Clinical Study Protocol;
  • πŸ”₯πŸ”₯ Statistical Analysis Plan;
  • πŸ”₯πŸ”₯πŸ”₯ TLF layouts for SAP;
  • πŸ”₯πŸ”₯πŸ”₯πŸ”₯ Full package of documentation for DMC.

Studying a design based on a published example, that eventually was done by the FDA with received a Priority Review, Orphan and Breakthrough Therapy designations is really helpful in practice.