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GENE AND CELL THERAPY RESOURCES

Gene Therapy Resource Program

The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical interventions.
http://www.gtrp.org

PACT

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.
https://www.pactgroup.net/

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:
http://www.ggact.org/for-investigators/services-available/

ClinicalTrials.Gov

ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world.
https://www.clinicaltrials.gov

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.
http://www.acgtfoundation.org

CIRM California Institute for Regenerative Medicine

https://www.cirm.ca.gov/about-cirm

The New York Stem Cell Foundation

http://www.nyscf.org

REGULATORY RESOURCES

US FDA

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 www.fda.gov and search for the Cellular & Gene Therapy Guidances homepage.

1998 Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/guidance-human-somatic-cell-therapy-and-gene-therapy

2004 Guidance for Industry: Sterile Drug Products Produced by Aseptic Processing - Current Good Manufacturing Practice
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM070342.pdf

2007 Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products; Guidance for Industry
https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/UCM091345.pdf

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)
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/content-and-review-chemistry-manufacturing-and-control-cmc-information-human-somatic-cell-therapy

2011 Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products
https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM243392.pdf

2011 Clinical Considerations for Therapeutic Cancer Vaccines
https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Vaccines/UCM278673.pdf

2013 Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/preclinical-assessment-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
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/determining-need-and-content-environmental-assessments-gene-therapies-vectored-vaccines-and-related

2015 Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products; Guidance for Industry
https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM404087.pdf

2016 Recommendations for Microbial Vectors Used for Gene Therapy; Guidance for Industry
https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM466625.pdf

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
https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM574889.pdf

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
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/regulatory-considerations-human-cells-tissues-and-cellular-and-tissue-based-products-minimal

2020 Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Draft Guidance for Industry
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/interpreting-sameness-gene-therapy-products-under-orphan-drug-regulations

2020 Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Guidance for Industry
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/chemistry-manufacturing-and-control-cmc-information-human-gene-therapy-investigational-new-drug

2020 Long Term Follow-up After Administration of Human Gene Therapy Products; Guidance for Industry
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/long-term-follow-after-administration-human-gene-therapy-products

2020 Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/testing-retroviral-vector-based-human-gene-therapy-products-replication-competent-retrovirus-during

2020 Human Gene Therapy for Hemophilia; Guidance for Industry
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-hemophilia

2020 Human Gene Therapy for Rare Diseases; Guidance for Industry https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-rare-diseases

2020 Human Gene Therapy for Retinal Disorders; Guidance for Industry https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-retinal-disorders


OFFICE OF SCIENCE POLICY

Research involving recombinant or synthetic nucleic acid molecules that is conducted at or sponsored by an institution that receives any support for such research from the National Institutes of Health (NIH) requires compliance with the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines). The NIH Guidelines span work done at the bench, in cell lines, in animals, and in humans. In April 2019, NIH revised the NIH Guidelines to streamline oversight of human gene transfer (HGT) research. Under the amended NIH Guidelines, the NIH Office of Science Policy (OSP) does not:

  • accept new HGT protocols for the protocol registration process under the NIH Guidelines
  • accept annual reports, safety reports, amendments or other documentation for any previously registered HGT protocols under the NIH Guidelines.

Robust oversight continues under the FDA, and NIH-funded HGT research remains subject to oversight by local authorities such as Institutional Review Boards and Institutional Biosafety Committees.

Because of the changing landscape, in 2019, the NIH refocused the Recombinant DNA Advisory Committee (RAC) into a role closer to its original mandate, which was to follow and provide advice to the NIH Director on scientific, safety and ethical issues associated with emerging biotechnologies. The RAC was renamed the Novel and Exceptional Technology and Research Advisory Committee (NExTRAC), which continues to provide a public forum for discourse, and the advice the NIH receives from the NExTRAC will be essential to the agency in appropriate stewardship of its investment in cutting-edge science. The RAC documents, meeting materials, and webcasts have been archived and remain available on the NIH Office of Science Policy website.w 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. https://osp.od.nih.gov/biotechnology/main-nextrac/

GENE THERAPY NEWSLETTERS/JOURNALS

Biomedicines
https://www.mdpi.com/journal/biomedicines

Cancer Gene Therapy
http://www.nature.com/cgt/

Gene Therapy
www.nature.com/gt/

Gene Therapy Net.com
Online Newsletter
http://www.genetherapynet.com

Gene Therapy Review
http://www.genetherapyreview.com

Human Gene Therapy Journals:
Human Gene Therapy https://www.liebertpub.com/doi/10.1089/hum.2019.317
Human Gene Therapy Methods https://www.liebertpub.com/loi/hgtb
Human Gene Therapy Clinical Development https://www.liebertpub.com/loi/humc

Journal of Gene Medicine
http://onlinelibrary.wiley.com/journal/10.1002/%28ISSN%291521-2254

Molecular Family of Journal:
Molecular Therapy
Molecular Therapy Oncolytics
Molecular Therapy Methods and Clinical Development
Molecular Therapy Nucleic Acids
https://www.cell.com/molecular-therapy-family/home

The CRISPR Journal
https://home.liebertpub.com/publications/the-crispr-journal/642

GENE THERAPY SOCIETIES

Australasian Gene Therapy Society (AGTS)
https://agcts.org.au/

British Society of Gene Therapy (BSGT)
https://www.bsgct.org/

Netherlands Society of Gene and Cell Therapy (NVGT)
https://www.nvgct.nl/

European Society of Gene and Cell Therapy (ESGCT)
https://www.esgct.eu/

German Gene Therapy Society (DGGT)
https://dg-gt.de/

International Society for Cancer Gene Therapy (ISGCT)
https://isctglobal.org/

Japan Society for Gene Therapy (JSGT)
http://www.jsgt.jp

Korean Society for Gene and Cell Therapy (KSGT)
http://www.ksgct.org/

Societe Francophone de Therapie Cellulaire et Genique (SFTCG)
https://www.sftcg.fr/

Spanish Society of Gene and Cell Therapy (SETGyC)
https://www.setgyc.es/

Swedish Society of Gene and Cell Therapy (SSGCT)
http://www.ssgct.org/main/