Analysts: FDA’s Guidance on Gene Editing is Important, Albeit “Not Surprising”

March 30, 2022

While not surprised by the details of the FDA’s new guidance for gene editing in the biotech industry, analysts welcome the recommendations. Although largely in line with current industry practices, they argue that the guidelines provide a baseline for biotech firms and help alleviate uncertainty and unexpected drug development holdups.

According to Annalee Armstrong of Fierce Biotech, “The FDA suggested patient selection for phase 1 trials should focus on those who do not have any other treatment options. This could mean that gene editing studies will need to be conducted abroad when a disease has an approved medication. Intellia Therapeutics is doing just that for the phase 1 study for its lead asset, transthyretin (ATTR) amyloidosis CRISPR therapy NTLA-2001, with sites in the U.K., Sweden and New Zealand. AATR amyloidosis has approved treatments from Alnylam and Pfizer.”