Other Helpful Resources


Gene Therapy Resource Program

The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical interventions.


PACT is a NHLBI funded program that offers expertise in translational development and scale-up capability needed to produce cellular products under cGLP and cGMP/cGTP regulations. PACT's 5 cell processing facilities and coordinating center are charged with implementing the new PACT initiative.

The Growing Gene and Cell Therapy (GGACT)

A collaboration between Boston Children's Hospital, Cincinnati Children's Hospital Medical Center, and the University of California Los Angeles, was established in 2016 with funding from NIH NCATS. We aim to support investigators to rapidly translate complex gene and cell therapies to early phase, investigator-initiated clinical trials. For information and available services please visit our website:

Association of Academic Biologics Manufacturers (AABM)

A cooperative network of non-profit academic based biologics manufacturers a national resource to bring together clinical investigators with manufacturing capacity and expertise. Share facility operations and expertise.

ClinicalTrials.Gov is a database of privately and publicly funded clinical studies conducted around the world.

Alliance for Cancer Gene Therapy (ACGT)

Raises and distributes funds for scientific research into the causes, treatment and prevention of all types of cancer, utilizing cells and genes as medicines.

CIRM California Institute for Regenerative Medicine

The New York Stem Cell Foundation



Selected Guidances

The FDA provides Guidance for Industry for a wide variety of topics. Below are selected documents of interest to the gene and cell therapy community. The documents are arranged in chronologic order of issuance. For other documents, or if the link below has changed, please go to the and search for the Cellular & Gene Therapy Guidances homepage.

1998 Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy

2004 Guidance for Industry: Sterile Drug Products Produced by Aseptic Processing - Current Good Manufacturing Practice

2007 Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products; Guidance for Industry

2008 Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs)

2011 Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products

2011 Clinical Considerations for Therapeutic Cancer Vaccines

2013 Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products

2015 Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products; Guidance for Industry

2015 Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products; Guidance for Industry

2016 Recommendations for Microbial Vectors Used for Gene Therapy; Guidance for Industry

2017 Deviation Reporting for Human Cells, Tissues, and Cellular and Tissue-Based Products Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR Part 1271; Guidance for Industry

2017 Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use; Guidance for Industry and Food and Drug Administration Staff

2020 Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Draft Guidance for Industry

2020 Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Guidance for Industry

2020 Long Term Follow-up After Administration of Human Gene Therapy Products; Guidance for Industry

2020 Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry

2020 Human Gene Therapy for Hemophilia; Guidance for Industry

2020 Human Gene Therapy for Rare Diseases; Guidance for Industry

2020 Human Gene Therapy for Retinal Disorders; Guidance for Industry


Recombinant DNA work in the US requires compliance with the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines). The guidelines span work done at the bench, in cell lines, in animal models and in humans. Previously all human gene therapy required review at the federal level by the OBA Recombinant DNA Advisory Committee (RAC), at the local level by the respective Institutional Biosafety Committee (IBC), and by the US FDA. In 2019, the NIH determined that the scientific and ethical issues had progressed to the point where federal review was not necessary. Review is still required at the institutional level (IBC) and by the US FDA. OBA was restructured and the NIH Guidelines are now managed by the Office of Science Policy

A new committee has been created to provide a forum for the discussion of scientific, safety, and ethical issues in emerging biotechnologies. The Novel and Exceptional Technology and Research Advisory Committee (NExTRAC) is a federal advisory committee that provides recommendations to the NIH Director. While it does not review gene therapy protocols, it does serve the public forum role previously provided by the RAC.



Cancer Gene Therapy

Gene Therapy

Gene Therapy
Online Newsletter

Gene Therapy Review

Human Gene Therapy Journals:
Human Gene Therapy
Human Gene Therapy Methods
Human Gene Therapy Clinical Development

Journal of Gene Medicine

Molecular Family of Journal:
Molecular Therapy
Molecular Therapy Oncolytics
Molecular Therapy Methods and Clinical Development
Molecular Therapy Nucleic Acids

The CRISPR Journal


Australasian Gene Therapy Society (AGTS)

British Society of Gene Therapy (BSGT)

Netherlands Society of Gene and Cell Therapy (NVGT)

European Society of Gene and Cell Therapy (ESGCT)

German Gene Therapy Society (DGGT)

International Society for Cancer Gene Therapy (ISGCT)

Japan Society for Gene Therapy (JSGT)

Korean Society for Gene and Cell Therapy (KSGT)

Societe Francophone de Therapie Cellulaire et Genique (SFTCG)

Spanish Society of Gene and Cell Therapy (SETGyC)

Swedish Society of Gene and Cell Therapy (SSGCT)