FDA Guidance Updates

ASGCT offers regular updates and analysis on FDA guidance documents related to gene therapy that are currently open for comment.

Six New FDA Draft Guidance Documents Cover Hemophilia, Retinal Disorders, Rare Diseases, Manufacturing, Safety, Long-Term Follow-up

Updated July 19, 2018

“Once just a theory, gene therapies are now a therapeutic reality for some patients,” FDA Commissioner Scott Gottlieb, MD, said in a July 11 statement following the release of the FDA's much-anticipated six guidance documents on gene therapy. Reflecting the rapid advancement in the field, three of them are the first for disease-specific applications of gene therapy—for hemophilia, retinal disorders, and other rare disease. The information is intended to assist sponsors in addressing the challenges of rare disease clinical development, such as limited study population size. In addition, the draft guidance on gene therapy for treatment of hemophilia provides recommendations regarding surrogate endpoints that could be used by sponsors pursuing accelerated approval of such products.

Human Gene Therapy for Hemophilia

Human Gene Therapy for Retinal Disorders

Human Gene Therapy for Rare Diseases

The other three guidance documents, which provide sponsors with manufacturing recommendations, are updates on previous guidance documents in need of revision due to the rapid advances in the field since their original release. These documents offer guidelines to sponsors regarding the provision of sufficient chemistry, manufacturing and control (CMC) information to assure safety, identity, quality, purity and strength/potency of gene therapy products; the proper testing for replication competent retrovirus during the manufacture of retroviral vector-based gene therapy products; and the design of long-term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a gene therapy product.

Chemistry, Manufacturing, and
Control (CMC) Information for
Human Gene Therapy Investigational
New Drug Applications (INDs)

Long Term Follow-Up After
Administration of Human
Gene Therapy Products

Testing of Retroviral Vector-Based
Human Gene Therapy Products for
Replication Competent Retrovirus
During Product Manufacture and
Patient Follow-up

Comments are due on all guidance documents by October 10, 2018. ASGCT will be evaluating the specific content of the guidance documents over the course of the comment period and leading up to its liaison meeting with the FDA on September 13, and will provide Society recommendations related to the details of these guidance documents. ASGCT appreciates the level of attention that FDA is providing to gene therapy at this important turning point in its development.

FDA Draft Guidance on Epidermolysis Bullosa Includes Gene Therapy Trial Requirements

Updated June 20, 2018

The FDA on June 18 issued a draft guidance document, Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations. The guidance, which has a 60-day comment period, indicates that studies involving drug products intended for gene therapy should include assessments related to lentivirus vector-based risks and long-term follow-up. It also references prior guidance documents on the design of early-phase clinical trials of cellular and gene therapy products and observing subjects for delayed adverse events in gene therapy clinical trials.

Epidermolysis Bullosa:
Developing Drugs for Treatment
of Cutaneous Manifestations

Pillars of Advocacy

The ASGCT Advocacy Program is built on three pillars: defining value, supporting research, and promoting awareness.

Strategic Alliances

To strengthen ASGCT’s advocacy efforts and its ability to significantly impact policymakers on a larger scale, the society engages in activities with likeminded groups. Collaboration is a cornerstones of ASGCT’s strategy to effectively and efficiently meet its government affairs-related goals. 


ASGCT 24th Annual Meeting

May 11-14, 2021