Main Conference Day 3
- Yi Shi, PhD - Professor of Pharmacological Sciences, Icahn School of Medicine at Mount Sinai
- Fabio Macciardi, MD, PhD - Chief Scientific Officer, Cognigenics
- Masha Stromme - CEO, Black Swan Biotech
- Shigeo Matsuda, PhD - Vice President, Chemistry, City Therapeutics
- Yasuhiro Kajihara, PhD - Professor, Department of Chemistry, Osaka University
- Yingnan Zhang, PhD - Senior Principal Scientific Manager, Genentech
- Rita Chen, PhD - Research Fellow, Academia Sinica
- Sridhar Vaddeboina, PhD - Senior Vice President, CMC Operations, Wave Life Sciences
- Maryke Fehlau - CTO, BioNukleo
Luxna Biotech is advancing LuxiAP™, a next‑generation oligonucleotide chemistry platform built around four proprietary nucleic acid modifications—AmNA, scpBNA, GuNA, and 5′‑CP—to boost potency, stability, and tolerability across antisense modalities. We will present our recent results with GuNA™ and 5′‑CP™ showing their versatility in gapmers and other ASO designs.
- Ajaya Shrestha, PhD - Chemistry Group Lead, Luxna Biotech
Peptides have long been ignored as good drug leads for gastrointestinal disorders due to their intrinsic metabolic limitations. However, we argue that peptides could be ideal drug candidates for gut-specific treatments and have developed methods to render them gut-stable for oral treatment. We exemplify this new frontier with several examples, including the trefoil factor family, blockbuster linaclotide, oral oxytocin for abdominal pain, and new treatment opportunities for diseases associated with intestinal biofilms.
- Markus Muttenthaler, PhD - Professor, Neuropeptide Research, The University of Queensland and University of Vienna
Targeted radiopharmaceuticals pair tumor-selective ligands with diagnostic or therapeutic radionuclides, enabling whole-body target mapping and matched therapy. We describe a phage-display pipeline that transformed PD-L1 peptide-macrocycle hits into single-digit-nanomolar ligands through focused libraries, synthetic SAR, structural biology and mouse imaging. Building on these results, our TRP program integrates medicinal chemistry with DNA-barcoded Liquid Molecular Arrays to compare up to 100 vectors simultaneously in vivo, accelerating translation toward viable clinical candidates.
- Ratmir Derda, PhD - Founder and CSO, 48 Hour Discovery, University of Alberta
Gate2Brain is developing a proprietary peptide-shuttle platform to transport therapeutics across the blood-brain barrier. This presentation will discuss the translation of BBB shuttle peptides from academic discovery into drug development, with a focus on G2B-002, our lead program for pediatric high-grade gliomas. I will share key learnings in peptide design, CMC, preclinical development and regulatory strategy as we advance toward a multiregional Phase I/IIa clinical trial.
- Meritxell Teixidó, PhD - CEO and CSO, Gate2Brain
Design of Experiments (DoE) was applied to optimize the manufacturing process of dual-cargo LNPs for gene editing. While critical process parameters (CPPs) were successfully established to achieve target physico-chemical CQAs—such as size, PDI, and encapsulation efficiency—in vivo potency was significantly impacted by process conditions. These findings highlight that standard physical characterization alone is insufficient, underscoring the necessity of advanced analytical tools to understand structure-activity relationships (SAR) and preserve biological activity during scale-up.
- Qook Lee, PhD - Sr. Director, HPI (BioPharma)
- Keith Bowman - Vice President of Process Development, Wave Life Sciences
China has become an increasingly important market for oligonucleotide therapeutics, accompanied by a rapidly evolving regulatory environment. Following publication of the CDE's first dedicated CMC guideline for chemically synthesized oligonucleotide drugs in Feb 2026, greater clarity has emerged regarding regulatory expectations for the development, manufacture, and control of this modality. This presentation will discuss key China-specific CMC considerations for oligonucleotide therapeutics, drawing on both the newly published CDE guideline and practical experience from a very recent NDA review of an innovative ASO product submitted shortly after issuance of the guideline. Topics will include impurity control, starting material designation, comparability, process validation, and lifecycle management, with a particular focus on considerations relevant to global developers seeking registration in China. The presentation aims to provide practical insights into navigating China's evolving regulatory landscape and supporting efficient global development strategies for oligonucleotide products
- Yixuan Zhang - Associate Director, Regulatory CMC, GSK
Priavoid develops disease-modifying therapeutics for neurodegenerative diseases. Its all-D-peptide platform targets pathogenic protein aggregation by stabilizing monomeric proteins and detangling aggregates. Building on clinical-stage PRI-002 for Alzheimer’s disease, the platform has identified Parkinson’s disease candidates that reduce α- synuclein pathology and show brain penetration after oral administration in mice.
- Mathias Wendt, PhD - Senior Peptide Chemist, Priavoid GmbH
- Nicolas Gilles, PhD - Research Director, CEA & Co-Founder & CSO, V4Cure
- Yanjie Li, PhD - Scientist IV, Genentech