---
title: "Resources - external links"
slug: "resources-external-links"
updated: 2026-07-20T20:05:23Z
published: 2026-08-04T17:22:02Z
canonical: "bgtcplaybook.document360.io/resources-external-links"
---

> ## Documentation Index
> Fetch the complete documentation index at: https://bgtcplaybook.document360.io/llms.txt
> Use this file to discover all available pages before exploring further.

# Resources - external links

#### Resources: Patient engagement

For further information on engaging with patients review the following resources.

- [National Organization for Rare Disorders (NORD) Resource Page](https://rarediseases.org/clinicians-researchers/)
  - Provides a centralized resource hub to support clinicians and researchers advancing research and treatment for rare diseases
- [Why Timelines are Tough: Communicating the Path to Gene Therapy, PJ Brooks, PhD at Empowering Patients 2026: A Cell and Gene Therapies Summit](https://www.asgct.org/education-career-resources/media-library/why-timelines-are-tough-communicating-the-path-to-gene-therapy)
  - Accelerating Medicines Partnership® (AMP®) Program Bespoke Gene Therapy Consortium (AMP BGTC) explores why defining timelines is so challenging and the many moving parts that shape development timelines for gene therapies
- [FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making](https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical)
  - Recognizing the importance of the patient perspective at various stages of pharmaceutical development, the FDA has dedicated a series of Guidance Documents advising how Sponsors can collect and incorporate patient input
  - A note on the above linked documents: documents 1-3 are final versions; document 4 is draft
- [Global Genes – Allies in Rare Disease](https://globalgenes.org/)
  - Global Genes is a nonprofit organization supporting patient advocacy for rare and genetic diseases
  - Includes resources for patients (e.g., disease education and patient services) and for researchers (e.g., patient data registries)

#### Resources: Platform based approaches

If you are interested in learning more from the experiences of gene therapy platform programs – such as PaVe-GT and Somatic Cell Genome Editing Consortium – refer to the resources below

- [PaVE-GT Resources](https://pave-gt.ncats.nih.gov/pave-gt-resources/)
  - PaVe-GT team has gained widely applicable first-hand experience in gene therapy development and regulatory interactions and is committed to publicly sharing scientific and regulatory lessons learned in developing four rare disease gene therapies
  - This page provides translatable resources to promote the development of other gene therapies
- [Somatic Cell Genome Editing (SCGE) Consortium Toolkit](https://scge.mcw.edu/toolkit/data/initiatives)
  - The Somatic Cell Genome Editing (SCGE) Toolkit team collects data and metadata from funded consortium laboratories, reviews, curates and standardizes data for loading into the Toolkit, works with the submitting laboratory and NIH program staff to certify data loading, and then makes data publicly accessible
- [National Institute for Innovation in Manufacturing Biopharmaceuticals (NIMBL](https://www.niimbl.org/projects-programs/viral-vectors/))
  - Program dedicated to the mission of creation and availability of a robust, economically viable, shared-access platform for the technical development, manufacturing, and characterization of AAV-based gene therapy vectors

#### Resources: Clinical trials

Refer to the following resources for additional information about considerations for cell and gene therapy clinical trials

- [ICH guidelines](https://www.ich.org/page/ich-guidelines)
  - Establishes the global standards for designing, conducting, and reporting clinical trials
- [FDA guidelines on cell and gene therapies](https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances)
  - Repository of draft and published FDA guidance on cell and gene therapy

#### Resources: Clinical / translational research

Refer to the following resources for additional information about considerations for clinical or translational research

- [NIH All of Us research program](https://allofus.nih.gov/)
  - All of Us Research Program is a historic effort to collect and study data from one million or more people living in the United States
  - Includes a health dataset from diverse participant cohort for researchers to leverage

#### Resources: Preclinical research

Check out the following resources if you are interested in more information on preclinical research related to cell and gene therapies

- [Therapeutics for Rare and Neglected Diseases (TRND)](https://ncats.nih.gov/research/research-activities/TRND)
  - TRND stimulates therapeutic development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses
- [NCATS BrIDGs program](https://ncats.nih.gov/research/research-activities/bridgs?utm_source=LinkedIn&utm_medium=Social&utm_campaign=BrIDGs)
  - This program allows research partnerships to advance candidate therapeutics for both common and rare diseases into clinical testing; selected researchers partner with NCATS experts to generate preclinical data and clinical-grade material through government contracts for use in Investigational New Drug (IND)
- [ASGCT Community Quick Takes: Biomarker Breakdown](https://patienteducation.asgct.org/research-clinical-trials/community-quick-takes/biomarker-breakdown)
  - Provides insights on how biomarkers can help move cell and gene therapies forward, and emphasizing the critical role of patient advocacy groups in shaping outcome measures
- [FNIH Biomarker Consortium](https://fnih.org/our-programs/biomarkers-consortium/)
  - Biomarkers Consortium is a pre-competitive public-private biomedical research partnership managed by the Foundation for the National Institutes of Health (FNIH) that endeavors to discover, develop, and seek regulatory acceptance of drug development tools/solutions (e.g. disease biomarkers and surrogate endpoints) to support new drug development, preventive medicine, and medical diagnostics

#### Resources: Regulatory

Check out the below resources for more information on and examples of regulatory submissions

- [Somatic Cell Genome Editing (SCGE) Consortium Shared Regulatory Documents](https://scge.mcw.edu/platform/public/documents/regulatory)
  - Repository of documents generated through regulatory interactions between the U.S. Food and Drug Administration (FDA) and consortium projects to help the rare disease communities and the providers and researchers that serve them

#### Resources: Manufacturing

Take a look at the following resources if you are interested in learning more about manufacturing considerations for cell and gene therapies

- [ASGCT Community Quick Takes: Manufacturing Matters](https://patienteducation.asgct.org/research-clinical-trials/community-quick-takes/manufacturing-matters)
  - Drs. Angelica Meyer, P.J. Brooks, and Steven Gray explore why gene therapy manufacturing matters, why quality checks are an essential part of the development timeline, and the challenges of large-scale production