Friday, September 25 - EST (Eastern Time, UTC-05:00
- PGN-EDODM1 represents an advanced peptide therapeutic engineered with enhanced properties for treating Duchenne muscular dystrophy type 1 (DMD1), incorporating optimized cell-penetrating sequences and improved stability that enable efficient delivery to skeletal and cardiac muscle tissues, with preclinical studies demonstrating superior dystrophin restoration, functional muscle improvement in animal models, favorable pharmacokinetic profiles, and a well-characterized safety profile that supports progression into clinical development where the therapeutic aims to address the underlying pathophysiology of DMD1 and provide meaningful clinical benefit to patients suffering from this progressive and life-threatening neuromuscular disorder.
- Jane Larkindale - VP Clinical Science, PepGen Inc.
- An overview of advancing SGB-7342 into Phase 1 clinical trials for the treatment of overweight and obesity, focusing on the RNAi mechanism, study design, patient selection, dose escalation strategy, and key endpoints for assessing safety and pharmacological activity.
- Weimin Wang, PhD - Founder & CEO, Sanegene Bio
This presentation explores next-generation siRNA manufacturing strategies that address growing industry demand for scalable, cost-effective production. By integrating enzymatic ligation and thermostable enzyme technologies, we demonstrate pathways to improve efficiency, reduce cost of goods, and enable flexible manufacturing at scale.
Transition from conventional SPOS to hybrid enzymatic ligation for improved yield, purity, and sustainability
Design and screening of thermostable ligases to enable high-temperature, high-efficiency ligation
Impact of elevated temperature on impurity control, fragment selectivity, and ligation performance
Scalable manufacturing strategies (C-to-C, C-to-P, P-to-P) to optimize cost, robustness, and process efficiency
- Xiaocen (Chris) Li, PhD - Director of Oligonucleotide Science and Technology, Hongene
