Ep 86: AAV Gene Therapy as a Potential Cure for Diabetes is No Longer Science Fiction
Gene therapy for diabetes. It sounds like pure science fiction, something that’s decades away. But what if it wasn’t? This concept isn’t just a far-off dream. In fact, AAV gene therapy is already FDA-approved and working in other medical fields. And for diabetes, its approval is right around the corner. We get that this is a confusing, complex topic—even for providers—which is exactly why we recorded this podcast.
Here’s what you’ll learn:
- What AAV gene therapy is (delivering a DNA “blueprint” for a protein like insulin).
- Why this is a “one and done” therapy that could last for years, or even a lifetime.
- How a harmless, engineered virus (called an AAV vector) is used as a “delivery truck.”
- How this new diabetes treatment could work for anyone, even if you’ve had diabetes for 100 years.
- Other FDA approved therapies that use gene therapy technology
- How this approach could allow your own muscle tissue to produce insulin.
- The realistic timeline: Why this is here now, with human trials on the horizon.
LISTEN TO THE EPISODE
AAV Gene Therapy: From Science Fiction to Real-World Science
Our goal is to help you cut through the medical jargon, understand what this new science really means for your health, and feel empowered by the future.
When you live with diabetes, talk of a “cure” often takes place in the medical office, e.g., in 1970, Dr. Edelman heard from his doctor that a cure is right around the corner, yet here we are in 2025, where numerous labs are working on developing different approaches to help people with diabetes. Today, we’re talking about a new class of therapy that is already FDA-approved for other conditions, such as blindness. Now, that same proven technology is on the cusp of being used for diabetes.
This is your moment of clarity. Dr. Edelman, Dr. Pettus, and special guest Dr. Fraser Wright translate the high-level science into something that anyone can understand. You’ll learn why this “one and done” approach could truly be the real deal.
Understanding the Science: What the Terms Mean
Let's break down the key parts of AAV gene therapy with the facts you need to know.
🧬 Gene Therapy: The “Blueprint”
Think of this as delivering a new “blueprint” (a DNA gene) to your cells. This blueprint tells your cells how to build a protein, like insulin, that your body is currently missing.
🚚 AAV Vector: The “Delivery Truck”
This is the “good” virus. It’s an engineered, harmless virus (Adeno-Associated Virus) that acts as a delivery vehicle. It’s been gutted of all its own “bad” stuff and filled with the good “blueprint” (the gene) you need.
🛠️ “One and Done”: The “Long-Term Fix”
What makes this new diabetes treatment so exciting is that it is a one-time procedure. Because the DNA blueprint is delivered directly to your cells, they can continuously create the protein on their own. It’s not a drug that wears off; it’s designed to work for many years, maybe even a lifetime.
How AAV Gene Therapy Works for Diabetes: A Practical Q&A
Here are the questions about how this therapy is applied to diabetes.
How is it given?
AAV gene therapy is delivered via an injection into muscle tissue, a very accessible and large part of the body.
How does it know when to work?
It’s not just a “dumb” insulin factory. The therapy is designed with a built-in “glucose sensor” (like the glucokinase enzyme) so it’s smart enough to produce insulin only when your blood sugar is high. The incredible part is that this “sensing” all happens within your own cells.
What about exercise?
This has been tested. In animal studies, dogs on treadmills showed stable blood sugars, suggesting exercise won’t cause problems or disrupt the therapy.The process has been extensively tested with excellent results. In fact, most animal participants showed long-term efficacy.
Is this new diabetes treatment permanent? Can it be turned off?
AAV gene therapy is long-lasting, but it can be reversed. Because it’s given in a localized area of muscle, doctors have developed ways to “turn off” those specific cells if needed.
Does it matter how long I've had diabetes?
No. This therapy works by adding a new function, not by trying to save old beta cells. You could have had diabetes for decades and still be a candidate.
Does this new diabetes treatment sound interesting? Watch to the whole episode here!
From Today's Treatments to Tomorrow's Cured-Reality
This approach is a new paradigm, different from today's management tools and other "curative" research paths.
Current Tech (Pumps/CGMs)
These tools are fantastic for managing diabetes by helping you react to blood sugars with external insulin. AAV gene therapy is a proactive approach to have your body make its own.
Islet Cell Transplants
This is another exciting “curative” approach that demonstrates real progress toward curing diabetes. Advances in islet cell transplantation offer hope for restored insulin production, and continued research in this area is bringing us closer to more breakthrough therapies.
The Timeline: 2026
This is moving from successful animal models (rodents, dogs, primates) to human clinical trials planned for 2026.
Your Docs Who Get It
Dr. Steven Edelman and Dr. Jeremy Pettus aren’t just endocrinologists—they’re your guides on the journey of Taking Control Of Your Diabetes (TCOYD). Their mission is simple: to provide clear, entertaining, and empowering education that helps you live a healthier, happier life with diabetes.
The TCOYD team understands how many people can be skeptical of the hype behind new diabetes treatments, but both Dr. Edelman and Dr. Pettus have looked at the initial research data involving animal trials and truly believe that people with diabetes have reason to be hopeful about this breakthrough.
Meet Our Special Guest
On this podcast episode, you’ll meet Dr. Fraser Wright, a career gene therapist with over 30 years in the field. He is the co-founder and Chief Gene Therapy Officer at Kriya Therapeutics. He also co-founded Spark Therapeutics, which brought forward Luxturna, the first FDA-approved gene therapy for a genetic disease.
Steve and Jeremy
Great update on gene therapy as a potential modality for diabetes. Something to look forward to. One question, would any type of immunosuppressive therapy likely be required? Thanks guys.
My question exactly:”…would any type of immunosuppressive therapy likely be required?” Also, is there an upper age limit for patients? I’ve aged out of a lot of clinical trials, but it would be great to just have a few years free of a disease, even if the method wasn’t proven. I’ve had T-1 for 59 years.
AAV gene therapy is something I would love to try. Just wanted to know which muscle will you inject the sensor into, what does the sensor looks like, and how is it removed if needed to be? I didn’t hear any side affects! Do you have to have a percentage of muscle mass to be a candidate?
In 1966 when I was diagnosed, the doctor said, “Oh, don’t worry. There will be cure for it within the next ten years!
I always take possible cure announcements with a grain of salt.
Im watching this closely having first invested in islet technology some 20 + yrs ago. I appreciate the rode your dedicated team are traveling. May God Bless you,your team and all others involved in this endeavor towards a successful journey.
Thank you Chris, appreciate it!
I would love to be on your trial list.ive had type1 for 50 years now,I’ve had heart bypass surgery in 2009 .I’m wearing the omnipotent which is a blessing with the dexcom g7 sensor.Thanks for all you do.
Thanks Dorothy! If you’d like to be involved in a clinical trial, you can search “type 1 diabetes” on the clinical trials website here to find active trials and those currently recruiting:
https://clinicaltrials.gov/
Exciting and definitely “hope” worthy. Is there any additional information on the human trials? Where? Who can participate, etc? Thank you so much for always keeping us updated on meaningful news.
Hi Michele,
Thanks so much for your comment! If you’d like to be involved in a clinical trial, you can search “type 1 diabetes” on the clinical trials website to find active trials and those currently recruiting:
https://clinicaltrials.gov/
Gene Therapy sounds great to me. I had Type 2 Diabetes for close to 30 years (diagnosed around the age of 42, not overweight). About 1 year ago my doctor told me that I had transitioned to Type 1. How common is this? I am on an insulin pump now. I also have a pacemaker. I doubt that I would be a good candidate for gene therapy at the age of 72, but I will be watching the news of progress with gene therapy.
Would AAV Gene Therapy be unaffected by the autoimmune islet-cell destroying antibodies individuals with Type-1 carry?