Gene Therapy Resources
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 Summit Session Recording
Gene Therapy and Gene Editing: Current Status and Potential Impact on the Rare Disease Community
This session recording from NORD’s 2021 Rare Summit highlights the current landscape, future capabilities, and potential barriers to expansion of gene therapy and gene editing therapeutics.
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
The Time Is Now: Understanding Gene Therapy, Applications, and Implications (2021)
NORD, Platform Q Health and ASGCT partnered to produce a two-part CME program to explain the potential of gene‐based treatment approaches to change the disease course for patients with genetic conditions.
Day 1: Gene Transfer, Editing, Silencing and RNA Based‐Approaches: What, When and How?
- Gene Therapy Day 1, Session 1 HCP Session – NORD (National Organization for Rare Disorders) (rarediseases.org)
- Gene Therapy Day 1, Session 2 HCP Session – NORD (National Organization for Rare Disorders) (rarediseases.org)
Day 2: Examples of Diseases that Utilize Gene‐Based Therapy; Post-Gene Therapy: Implications for Patients’ Management
- Gene Therapy Day 2, Session 1 HCP Session – NORD (National Organization for Rare Disorders) (rarediseases.org)
- Gene Therapy Day 2, Session 2 HCP Session – NORD (National Organization for Rare Disorders) (rarediseases.org)
Workshops
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.
