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# New, Updated FDA Gene Therapy Guidances
- URL: https://www.fdaweb.com/new-updated-fda-gene-therapy-guidances/
- Published: 2018-07-11T12:00:00.000Z
- Updated: 2026-09-15T00:18:42.000Z
- Author: David McFarland
- Tags: Drugs, Devices, #legacy-id-D5141839

FDA has published three new draft guidances on development of gene therapy products for specific diseases. The agency also has released new and comprehensive updates to three existing guidances that address manufacturing issues related to gene therapy. A [statement](https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613026.htm?ref=fdaweb.com) from FDA commissioner **Scott Gottlieb** (includes links to all six guidances) says the agency’s new commitment to develop disease-specific guidance documents “reflects the increasing activity in this field, and its growing importance to advancing public health.”

The new disease-specific draft guidances are for: 

- Hemophilia Treatment – The guidance gives recommendations on clinical trial design and preclinical considerations to support development of gene therapy products being developed as single-dose treatments that may enable long-term production of the missing or abnormal coagulation factor in patients. The guidance provides recommendations regarding surrogate endpoints that could be used by sponsors pursuing accelerated approval of gene therapy products that are intended for treatment of hemophilia.
- Retinal Disorders – The guidance will assist those developing gene therapy products for a wide variety of retinal disorders affecting both adult and pediatric patients. Gene therapy products currently in clinical trials in the U.S. for retinal disorders are commonly delivered by intravitreal injections or by subretinal injections. In some cases, the products are encapsulated in a device to be implanted within the eye. The guidance has recommendations related to product development, preclinical testing, and clinical trial design for such products.
- Rare Diseases – The guidance provides recommendations on preclinical, manufacturing, and clinical trial design for all phases of the clinical development program for these types of gene therapies. The information is intended to assist sponsors in the design of clinical development programs where there may be limited study population size, potential feasibility, and safety issues, as well as issues relating to the interpretation of effectiveness.

The updates to existing manufacturing issue guidances reflect input from many stakeholders, Gottlieb says. The guidances are: 

- Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy INDs – The guidance gives recommendations on how to provide sufficient CMC information to assure safety, identity, quality, purity, and strength/potency of investigational gene therapy products. It applies to human gene therapies and to combination products that contain a human gene therapy in combination with a drug or device.
- Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up – The guidance has additional recommendations on the proper testing for RCR during the manufacture of retroviral vector-based gene therapy products, as well as during the follow-up monitoring of patients who have received retroviral vector-based gene therapy products. Specifically, the draft recommends the identification and amount of material to be tested.
- Long Term Follow-Up After Administration of Human Gene Therapy Products – The guidance has recommendations on the design of long-term follow-up observational studies for the collection of data on delayed adverse events following administration of a gene therapy product.

“Our goal is to help promote safe and effective product development in this field,” Gottlieb writes. “We’ll continue to work with the product sponsors to help make the development and approval of these innovative gene therapies more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We’ll also make full use of our expedited programs such as breakthrough therapy designation and regenerative medicine advance therapy designation whenever possible.”