FDA Provides Recommendations for Using Microbial Vectors in Gene Therapy

October 19, 2015

On Wednesday, October 13, 2015, FDA released a draft guidance entitled “Recommendations for Microbial Vectors Used for Gene Therapy.” The document provides information and recommendations regarding the submission of microbial vectors for gene therapy (MVGTs) in early-phase clinical trials, and is intended to assist sponsors in the process of developing or submitting Investigational New Drug (IND) applications.

In a previous guidance on gene therapy clinical trials (from November 2006), FDA defined gene therapies as “[p]roducts that mediate their effects by transcription and/or translation of transferred genetic material and/or by integrating into the host genome and that are administered as nucleic acids, viruses, or genetically engineered microorganisms. The products may be used to modify cells in vivo or transferred to cells ex vivo prior to administration to the recipient.” However, according to FDA’s statement in the recently released guidance, MVGTs meet the regulatory definition of a 'biological product’, which the agency defines as a medical product that is “made from a variety of natural sources (human, animal, or microorganism). Like drugs, some biologics are intended to treat diseases and medical conditions. Other biologics are used to prevent or diagnose diseases.”

The guidance is focused around chemistry, manufacturing, and controls (CMC) information and, when finalized, will serve as a supplement to the April 2008 guidance entitled “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs).” The document provides sponsors with information directly related to the submission of INDs for MVGTs, and provides “provides an overview of preclinical and clinical considerations for these products.”

The draft guidance provides sponsors with various related information, including:

Product Manufacturing & Characterization

Preclinical Studies

  • Sponsors should provide adequate information about pharmacological and toxicological studies of the MVGT on the basis of which they have concluded that it is reasonably safe to conduct the proposed clinical trial. This information must be submitted in the IND (FDA recommendations provided on the selection of appropriate animal species and animal models of disease, as well as the overall design of preclinical proof-of-concept and toxicology studies for investigational products, including MVGT products, in previously issued guidance here). The guidance contains information on the following considerations of preclinical study design for MVGTs include:

Clinical Studies

FDA has released the draft guidance for the sole purpose of receiving feedback and comments from the industry before beginning to compose the final version. Comments can be submitted either electronically (here) or in writing, and must be done by December 14, 2015, 60 days from the announcement in the Federal Register.

For more details and information, view the full guidance here.

Are you in the process of developing an IND application? We can help with you achieve a successful outcome with FDA. We work with our clients throughout the entire process, from the first step of developing a regulatory strategy all the way through final submission and approval, using a proprietary approach that has proven to be successful numerous times. To find out more about our services and how we can help, please contact us.


February 3, 2017

FDA Releases CDER’s 2017 Guidance Agenda

Last month, FDA’s Center for Drug Evaluation and Research (CDER) released its annual guidance agenda, announcing the new and revised draft guidances that the Center plans to publish during the 2017...

April 19, 2016

Minimizing Medication Errors Related to Product Design, Part One: FDA’s Recommendations for Conducting Proactive Risk Assessments

On September 27, 2007, the Prescription Drug User Fee Act (PDUFA IV) reauthorization and expansion was signed into law, significantly strengthening FDA’s drug safety program. As part of the...

Three piles of pills

November 1, 2018

FDA Encourages Development of Novel Drugs for Rare Diseases with Draft Guidance Regarding the Role of Pre-IND Meetings

On Monday, October 12th, FDA issued a draft guidance regarding the role of Pre-IND Meetings in the development of drugs to treat rare diseases. The document, entitled "Rare Diseases: Early Drug...