A New Pillar of T1D Care: What Gene Therapy Could Look Like in Practice

In this lecture with Dr. Pettus:

The current ceiling of T1D care: 
Dr. Pettus opens with a level-set on where outcomes actually sit today – the share of patients above goal, the residual cardiovascular risk that persists at target A1C, the near-universal insulin resistance, and the weight and hypoglycemia issues that the technology layer alone has not solved.

The case for a new pillar of treatment:
A walk-through of the five categories currently in play for T1D – insulin, technology, immunotherapy, cellular replacement, and metabolic therapy – and where gene therapy slots in as the next one entering clinical development.

How AAV gene therapy works in plain mechanism terms:
A clear explanation of episomal delivery, why this is distinct from gene editing, and why muscle tissue is the chosen delivery site for a durable, post-mitotic expression platform.

The dual-transgene rationale:
Why this program co-delivers an insulin gene and a glucokinase gene, and how glucokinase functions as a tissue-level glucose sensor that may bring glucose-responsiveness to the insulin output.

The preclinical signal:
Canine and murine data showing normalized glucose profiles out to 1,600+ days from a single administration, with the trial-design implications that follow.

The clinical trial that’s now starting:
The phase 1 dose-escalation design, the patient population, the peri-procedural immune modulation strategy, and how the program is built to expand quickly into broader T1D populations.

Meet Your Ultra-Experts

*extra-relatable faculty (he's living with type 1!)
Picture of *STEVEN V. EDELMAN, MD
*STEVEN V. EDELMAN, MD

Founder & Director, TCOYD; Endocrinologist, Clinical Professor of Medicine, University of California, San Diego (UCSD) School of Medicine; Director, Diabetes Care Clinic, VA Medical Center San Diego

Picture of *JEREMY PETTUS, MD
*JEREMY PETTUS, MD

Endocrinologist, Director of Type 1 Track and Professional Medical Education at TCOYD; Associate Professor of Medicine, UCSD School of Medicine, San Diego

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