Other Helpful Resources

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/

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.

http://www.aabmonline.org

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

NYSCF 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.

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

https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm072987.htm

Guidance for Industry: Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and During Follow-up of Patients in Clinical Trials Using Retroviral Vectors

https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm072961.htm

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

http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM070342.pdf

Guidance for Industry: Gene Therapy Clinical Trials - Observing Subjects for Delayed Adverse Events

https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm072957.htm

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

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/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Xenotransplantation/ucm074131.htm

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)

https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm072587.htm

Draft Guidance for Industry: Assay Development for Immunogenicity Testing of Therapeutic Proteins

https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM192750.pdf

Guidance for Industry: Cellular Therapy for Cardiac Disease

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

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM243392.pdf

Clinical Considerations for Therapeutic Cancer Vaccines

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Vaccines/UCM278673.pdf

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

https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm376136.htm

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/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm401869.htm

Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM564952.pdf

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

Homologous Use of Human Cells, Tissue, and Cellular and Tissue-Based Products; Draft guidance for Industry and FDA Staff

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/UCM469751.pdf

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

https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM466625.pdf

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

OFFICE OF BIOTECHNOLOGY ACTIVITES

GeMCRIS - Genetic Modification Clinical Research Information System

GeMCRIS allows users to access an array of information about human gene transfer trials registered with the NIH, including medical conditions under study, institutions where trials are being conducted, investigators carrying out these trials, gene products being used, route of gene product delivery, and summaries of study protocols.

http://www.gemcris.od.nih.gov/Contents/GC_HOME.asp