Gene Therapy Guidance

This week the team had Eyes on Gene Editing – well some new draft guidance from the US FDA on Gene Therapy Products Incorporating Genome Editing.

1. FDA DRAFT Guidance – Human Gene Therapy Products Incorporating Human Genome Editing:

   The document is non-binding and sets-out to provide guidance to those who are developing human gene therapies incorporating genome editing of human somatic cells. The document can be found here. Included in the document are draft recommendations for details that should be included in an investigational new drug application such as information on product design, manufacturing, testing, and trial designs.

   The FDA is seeking comments on the draft – information on how to submit and when can be found here.

2. Commentary:

   Several analysts and consulting firms have provided their preliminary opinions on the draft guidance. BIOPHARMADIVE published an article which provides a quick briefing highlighting that the FDA reiterates their position that the potential risks of gene editing are unclear and that most of the Wall Street analysts view the guidance as straightforward and without and major surprises. They dive deeper into the gene editing area in the latter part of the article that you can find here.
   
   Fierce Biotech also noted the analysts are not worried about the guidance, but in fact may be happy to see the FDA taking a more active role in providing guidance to companies that are already moving down this development path and possibly avoid delays and clinical holds on their programs.

Tip of the Week

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