The Food and Drug Administration (FDA) has announced a draft guidance titled "IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations." This draft is aimed at helping sponsors who are developing personalized antisense oligonucleotide (ASO) drugs for severe or life-threatening genetic diseases, especially for those patients without other treatment options. It provides instructions on interacting with the FDA, making regulatory submissions, and ensuring ethical considerations like informed consent. The FDA encourages feedback on this draft to aid in the creation of the final guidance, which will assist in the swift start of drug investigations.
Simple Explanation
The FDA is making a new rule to help doctors make special medicine called ASOs for people with really bad genetic diseases that don't have other treatments. This new rule is supposed to guide doctors on how to work with the FDA when making these medicines.