All of our past Lunch & Learn events are available to watch on this page and on our YouTube channel.
Roadblocks to Developing a Treatment- This session explored some of the areas where treatments can get stopped while en route to an approval.
Recently Approved Therapies- Speakers focused on gene therapy approvals in 2022 including Skysona (for CALD), Zynteglo (for beta thalassemia), and Hemgenix (for hemophilia B).
Meet the Patients- Two mothers share their families' journeys participating in clinical trials for SMA and Duchenne muscular dystrophy.
Navigating the Patient-Provider Conversation- A clinician and caregiver give their perspectives and advice on navigating the diagnosis process to considering a treatment plan related to gene therapy.
Meet the Researchers- Three researchers discuss what they do in the lab, how they interact with stakeholders, and how the work relates to advancing gene and cell therapies for various patient populations.
The Pathway to an Approval- Speakers explore the clinical pathway for getting a therapy through a clinical trial and into the approval phase, and thoughts on cost and access once a therapy is approved.
Routes of Administration- Review the most common routes of gene therapy drug delivery, including the preclinical study model, and how these routes are currently used in certain approved therapies or clinical trials.
Diving into Preclinical Studies- Researchers break down the preclinical models for gene therapies and a patient advocate shares direct examples of how communities can gather this data to further expand the knowledge around their disease and possible treatments.
Annual Meeting Research Highlights- Patient Outreach Committee members share new research findings presented at ASGCT’s 25th Annual Meeting most relevant to patient groups.
What to Expect as a Participant in a Clinical Trial- Clinicians and advocates share what to expect before, during, and after a gene therapy clinical trial.
How AAV Vectors Are Made- Experts review the manufacturing process of how gene therapies are made.
Intro to Viral Vectors and Immune Responses- Researchers share how and why viral vectors are used as a common delivery approach for gene therapies, and concerns related to immunosuppression, immunity, and re-dosing.
Gene Therapy 101- Experts in the field review different gene therapy approaches, disease applications, and research processes, and describe the current state of the field.
Gene Therapy for SMA- Clinicians share insights on gene therapy options for SMA and a parent discusses having a child receive approved treatments.