> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Final Guide on Rare Disease Gene Therapies
- URL: https://www.fdaweb.com/final-guide-on-rare-disease-gene-therapies/
- Published: 2020-01-30T12:00:00.000Z
- Updated: 2026-09-14T16:00:55.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5145966

*Federal Register* notice: FDA has made available a final guidance entitled “Human Gene Therapy for Rare Diseases; Guidance for Industry.” The document provides information to assist sponsors in designing clinical development programs for such products, where there may be limited study population size and potential feasibility and safety issues as well as issues relating to the interpretability of bioactivity/efficacy outcomes. To view the notice, click [here](https://www.govinfo.gov/content/pkg/FR-2020-01-30/pdf/2020-01704.pdf?ref=fdaweb.com).