Gene Therapy Resources
Gene Therapy Documents
Understanding the Importance of Gene Therapy for Rare Diseases
Read out Gene Therapy information sheet which provides answers to some of the most frequently asked questions around the process.
Gene Therapy Videos
“Gene Therapy: Your Questions Answered” (2018)
This animated video addresses some of the most asked questions about gene therapy from patients and caregivers including: Why is gene therapy getting so much attention? and Is gene therapy a cure?
“Genome Editing: Q and A for Patients and Caregivers” Video Series (2020)
This series of four animated videos address questions about genome editing shared with NORD by our members and social media followers.
- What is genome editing?
- What are the safety, ethical and cost concerns?
- How do I participate and what is the role of patient organizations?
- The approval process
Podcasts
“Ungeeking The Speak: Dr Rachel Bailey Talks “Gene Therapy 101” (2021)
NORDpodTM podcast features host Matthew Zachary talking with Dr. Rachel Bailey, an authority on developing gene therapies to treat neurodegenerative disorders (2021).
NORD Rare Breakthrough Summit Session Recordings
Can We Make Gene Therapy More Accessible?
This session recording from NORD’s 2024 Rare Breakthrough Summit explores the promise of gene and cell therapies that has brought great hope to the rare disease community, and how accessing these therapies remains a huge challenge. Panelists discuss how we can work together to make manufacturing more efficient and ensure equitable access to these life-saving treatments.
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Moderator:
- P.J. Brooks, PhD – Deputy Director, Division of Rare Diseases Research Innovation, NCATS, NIH
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Speakers:
- David Barrett, JD – CEO, American Society for Gene and Cell Therapy
- Nicole Gaudelli – Entrepreneur in Residence, GV
- Peter Marks, MD, PhD – Director, Center for Biologics Evaluation & Research (CBER), FDA
The New Frontier of Gene Editing: Can We Potentially Cure Genetic Diseases?
This session recording from NORD’s 2023 Rare Breakthrough Summit discusses genome editing as an emerging, promising treatment approach for rare diseases. A panel of experts discuss the state of the science of these technologies and the regulatory framework of the approval process in this newer therapeutic area.
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Moderator:
- Edward Neilan, MD, PhD – Chief Medical and Scientific Officer, NORD®
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Speakers:
- PJ Brooks, PhD – Acting Director, Division of Rare Diseases Research Innovation, NCATS, NIH
- David Liu, PhD – Professor, Broad Institute Harvard University
- Peter Marks, MD, PhD – Director, Center for Biologics Evaluation & Research (CBER), US Food & Drug Administration (FDA)
- Amy Simon, MD – Chief Medical Officer, Beam Therapeutics
Can We Overcome the Challenges Facing Cell and Gene Therapies?
This session recording from NORD’s 2023 Rare Breakthrough Summit explores how though initially perceived as transformational treatments, cell and gene therapies are now viewed by some through a less enthusiastic lens because of issues related to cost, access and duration of benefit. Panelists discuss whether the original scenario is still possible.
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Moderator:
- Erica Cischke, MPH – Vice President, Government Affairs, Alliance for Regenerative Medicine
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Speakers:
- Diana Castro, MD – Founder and Director, Neurology Rare Disease Center
- Leslie Meltzer, PhD – Chief Medical Officer, Orchard Therapeutics
- Kinnari Patel, PharmD, MBA – President and Chief Operating Officer, Rocket Pharmaceuticals
- Nicole Verdun, MD – Director, Office of Therapeutic Products, CBER, FDA
Gene Therapy and Gene Editing: Current Status and Potential Impact on the Rare Disease Community
This session recording from NORD’s 2021 Rare Breakthrough Summit highlights the current landscape, future capabilities, and potential barriers to expansion of gene therapy and gene editing therapeutics.
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Moderator:
- Jens Wendland, MD – Global Program Lear Rare Genetics and Hematology Therapeutic Area, Takeda Pharmaceuticals
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Speakers:
- Ashanthi De Silva, MPA – Membership Manager, NORD
- Larissa Lapteva, MD, MHS, MBA – Associate Director, Division of Clinical Evaluation, Pharmacology and Toxicology, Offices of Tissues and Advanced Therapies, FDA Center for Biologics Evaluation and Research (CBER)
- Michael Maitland, MD, PhD – Senior Director of Clinical Development, Intellia
Gene Therapy Webinars
NORD and our partner, the American Society for Gene and Cell Therapy (ASGCT) hosted a five-part webinar series on gene therapy that is available to view on-demand (2019).
Gene Therapy: Yesterday, Today, and Tomorrow
This session is directed towards patients and the public and offers clear and simple explanations of the field’s history, the benefits and challenges of gene therapy, along with future trends. Presenters include Phillip Tai, PhD, and Cenk Sumen, PhD, Assistant Professor, Horae Gene Therapy Center, UMass Chan Medical School and Cenk Sumen, PhD, chief technology officer of Stemson Therapeutics and adjunct professor at NYU’s Tandon School of Engineering.
The Science Behind Gene Therapy
This session explores how gene therapies are developed, the ways in which evidence-based science guides manufacturers of gene therapies, how vectors deliver gene therapies to cells, and what happens inside the body after gene therapy is delivered. Speakers include Sven Kili, MD, who has a long history developing gene therapies for pharmaceutical companies and now provides strategic consulting for regenerative medicine companies and Tiffany Lucas, PhD, an FDA product reviewer in the division of gene therapy in the FDA Center for Biologics Evaluation and Research (CBER).
The FDA’s Role in Gene Therapy
This session explores regulatory pathways for gene therapies, how gene therapies are deemed safe and more. Speakers include Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research (CBER) and Julie Tierney, JD, senior policy advisor for strategic planning and legislation at CBER.
Understanding the Gene Therapy Process and Aftercare
This session covers how patients are identified as candidates for gene therapy, who may be on a gene therapy treatment team, how long the procedure takes, and what to expect during recovery and longer term follow up. Participants hear from Jerry Mendell, MD, who has focused his clinical work on neuromuscular diseases for the past five decades and Barry Byrne, MD, PhD, a pediatric cardiologist who has focused on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function.
Life After Gene Therapy
This session features Charles Hough, who was diagnosed with sickle cell disease when he was two years old, and Nicole Almeida, who found out her son had spinal muscular atrophy (SMA) when she was five months pregnant. Charles received gene therapy through a clinical trial at NIH in 2017. Nicole’s son, Matteo, received gene therapy when he was just 27 days old. Charles and Nicole talk about symptoms before gene therapy treatments, how they got involved in clinical trials, what the procedures were like and what life is like now for them and their families.
Continuing Medical Education (CME) Series for Healthcare Professionals
ASGCT Core Curriculum Series (2024)
NORD, ASGCT and Medlive partnered to produce a eight-part CME program to explain the potential of gene‐based treatment approaches to change the disease course for patients with genetic conditions or cancers, explain the role of FDA in providing regulatory oversight, discuss ethical issues surrounding the use of these therapies, and identify the role of clinicians in counseling patients on the resources that can help them access breakthrough cell and gene therapies.
Module 1: Genetics of Human Disorders
Module 2: Gene Therapy Overview
Module 3: Methods of Correcting Genetic Diseases (non-oncology )
Module 4: Use of Gene and Cell Therapies in Cancer Treatment
Module 5: Ethical Issues in Genetic Alteration
Module 6: Current Status of Clinical Gene Therapy
Module 7: Regulatory Aspects for Gene Therapy Development
Module 8: Role of Patient Advocacy Groups and International Partnerships
Resources from the American Society for Gene & Cell Therapy (ASGCT)
Gene Therapy 101
ASGCT Gene & Cell Therapy Education: Information for patients and the public about gene therapy, including a clinical trials finder database
Gene Therapy 101 Workshop (2022)
ASGCT collaborated with NORD on a gene therapy “Lunch and Learn” workshop for patients, caregivers and others presented by Phillip Tai, PhD, and Rachel Bailey, PhD, PhD, Assistant Professor, University of Texas Southwestern Medical Center on the basics of gene therapy including how gene therapy works, the different types, and the important role of viral vectors and protein production.