Podcast Summary: "Gene Therapy as a Potential Cure for Diabetes is no Longer Science Fiction"

Podcast: Taking Control Of Your Diabetes® - The Podcast!
Hosts: Dr. Steve Edelman & Dr. Jeremy Pettus
Guest: Dr. Fraser Wright, Chief Gene Therapy Officer at Kriya Therapeutics
Release Date: October 27, 2025

Episode Overview

This episode dives deep into the emerging world of gene therapy as a potential game-changer for diabetes—particularly, type 1 diabetes. Host endocrinologists (and people living with type 1 themselves) Drs. Steve Edelman and Jeremy Pettus are joined by top gene therapy researcher Dr. Fraser Wright, exploring what gene therapy actually is, how it's been used to treat other diseases, and why we may be on the verge of seeing trials using this technology as a one-time, potentially lifelong therapeutic for people with diabetes.

Key Discussion Points & Insights

1. Introduction to Gene Therapy and the Current Landscape

[00:58]

• The episode establishes the unique excitement around gene therapy for diabetes, stating that most clinicians and patients know little about it.
• The notion: "By the end of this, you're going to be experts on this topic..." – Dr. Jeremy Pettus

[02:58]

• Dr. Fraser Wright shares his background in gene therapy (30+ years), involvement in academic and industry settings (Pennsylvania, Stanford, Spark & Kriya Therapeutics).
• Notably, he's helped launch FDA-approved therapies using adeno-associated virus (AAV) and lentivirus vectors.

2. What is Gene Therapy?

[05:15]

• Gene therapy defined: Instead of giving patients proteins (like insulin) made in factories, gene therapy delivers DNA instructions directly into the patient's body, enabling their own cells to produce therapeutic proteins.
• This is called a "one-and-done" approach—ideally a single procedure for lasting benefit.

Dr. Fraser Wright [07:10]:
"We engineer [viruses] extensively so that they become...a delivery vehicle. ...It's a viral vector expressing something that's going to be beneficial to a person. ...It is non-replicating, it doesn't spread, it doesn't amplify. ...It's very inert."

• AAV vectors are safe, widely studied, and do not cause disease.
• Viruses are used simply because they're extremely efficient at delivering genetic material into cells.

3. Successful Real-World Examples of Gene Therapy

[09:54]

• Seven AAV-based gene therapies have been FDA-approved as of 2025.
• Dr. Wright was closely involved in therapies for:
  • RPE65-related blindness ("Leber congenital amaurosis type 2")
  • Hemophilia B
• The process involves rigorous research, animal and human trials, and regulatory approvals—demonstrating the technology's safety/efficacy.

[12:16]

• RPE65 blindness is a childhood-onset condition; gene therapy involves a subretinal injection.
• Results are dramatic: within weeks, patients experience improved vision; durability is now 10+ years.

Memorable Story [15:44]:
"One of the young women who received it had...given up on the idea of moving past high school...but this changed it for her and she was able to move forward...I mean some of the stories were bringing even tears to our eyes."
— Dr. Fraser Wright

4. Pivot: From Rare Genetic Diseases to Diabetes

[17:45]

• Historically, gene therapy targeted rare, single-gene ("monogenic") diseases—easier to 'fix.'
• The field has matured; multi-year, even decade-long benefits shown in humans.
• Now, the same logic and delivery technology can be applied to more common diseases like diabetes.

Dr. Wright [18:58]:
"The time now has come to take this validated therapeutic modality...into prevalent diseases."

5. Gene Therapy for Type 1 Diabetes: How Would It Work?

[21:09]

• The hosts explain that robust animal data support the concept.
• In these studies, animals with type 1 diabetes (no insulin production) received gene therapy targeted at muscle tissue.
• Muscle cells are engineered to secrete insulin, effectively taking over a pancreas-like role.
• Results: Animals (mice, dogs, primates) stopped needing insulin entirely and maintained normalized blood sugars.

Dr. Pettus [22:45]:
"...the hope is that it would at least reduce the amount of insulin you would have to take. And ultimately ... not need any insulin at all."

• Clinical trials in humans are expected to commence in 2026.

6. How Is Insulin Production Controlled? Safety Mechanisms

[24:32]

• The biggest concern: Would this cause low blood sugar (hypoglycemia) if insulin release wasn't tuned?
• Answer: The gene therapy approach may co-deliver sensor enzymes (like glucokinase) so that insulin production is regulated and responsive to blood glucose.
• Low doses are needed; the system is localized and designed for fine control.

Dr. Wright [24:56]:
"It's not just simply a delivery of ... insulin. It may involve insulin and involves other enzymes that have a sensor function..."

[27:22]

• If "overdose" of insulin production occurred, the system is reversible—muscle tissue with inserted genes can be deactivated with local therapy.

7. Practical Concerns & Study Design

[28:11]

• What if gene therapy is delivered to leg muscles—does exercise affect therapy?
  • Animal testing (dogs running on treadmills) indicates no adverse effect.
  • Therapy is localized; normal activity seems safe.
• Sites of gene insertion will be marked for monitoring and possible intervention.

[29:12]

• Timeline:
  • Human trials in 2026.
  • Potentially available before 2030, pending trial results.

Dr. Pettus [29:33]:
"This is actually happening now ... if everything went well, you're looking at sometime before 2030 ... that's here. We've been promised so much, you know, for so long."

8. Beyond Type 1: Future of Gene Therapy in Metabolic Diseases

[30:38]

• The same technology could potentially address type 2 diabetes and other hormone-related conditions.
• In the future, gene therapy could become as routine as a pill, shot, or insulin pump.

9. Humor and Human Touch

• The hosts and guest regularly joke and keep the tone light, reflecting their philosophy that, in diabetes, sometimes you simply have to laugh.
• Memorable joke:
  Dr. Edelman [04:38]: "When you're bouldering, what kind of jeans do you wear?"
  Dr. Wright: "I don't wear jeans."

Notable Quotes & Memorable Moments

• Dr. Wright on gene therapy's potential:
  "I think we're emerging here with the gene therapy approach and, you know, if we understand the mechanisms ... it just makes sense to move forward into prevalent diseases."
  — [20:50]

• On practical hope:
  "We've been promised so much, you know, for so long. ...This is like the speed that it could potentially happen."
  — Dr. Jeremy Pettus [29:33]

• On collaboration and humility:
  "I must say I've been very lucky in my career. I followed my nose ... I have to acknowledge the many great individuals who have really advanced gene therapy."
  — Dr. Fraser Wright [33:30]

Key Timestamps for Major Segments

• 00:58 — Introduction of gene therapy as a topic, why it excites the hosts
• 02:58 — Dr. Fraser Wright's background and experience
• 05:15 — Simple definition and explanation of gene therapy
• 09:54 — Real-world gene therapy success stories and FDA approval
• 12:16 — How gene therapy restored sight to children with genetic blindness
• 17:45 — Moving from rare diseases to diabetes
• 21:09 — How diabetes gene therapy would work (animal data, projecting to humans)
• 24:32 — Controlling insulin release, preventing hypoglycemia
• 27:22 — Can gene therapy be reversed if needed?
• 28:11 — Exercise and practical living with gene therapy
• 29:12 — Realistic timeline & hope for people with diabetes
• 30:38 — Broader applications and future vision

Closing Thoughts

This episode provides a thorough, accessible, and sometimes humorous masterclass on what gene therapy is and how it is transitioning from "science fiction" to practical possibility for diabetes. It's an eye-opening dose of hope, tempered with realistic caveats about the time needed for clinical trials, but grounded in decades of real-world scientific progress.

The practical upshot for listeners: gene therapy for diabetes is now entering human trials, with the real potential for one-and-done therapies that could dramatically reduce or eliminate daily insulin needs—perhaps even within this decade.

Recommended further action:
Visit Kriya Therapeutics for updates, and stay tuned for future podcast episodes as this story develops.