2026年全體會議（主題演講）

WEDNESDAY, MARCH 18

4:00pm Welcome Remarks by Conference Director
Gemma Smith, Senior Conference Director, Production, Cambridge Healthtech Institute

4:05pm Chairperson's Remarks
Adrian Krainer, PhD, St. Giles Foundation Professor, Cold Spring Harbor Laboratory, CSHL Cancer Center

4:10pm N-of-1 Therapeutics: Progress, Pitfalls, and Prospects for Future Individualized Medicines
Timothy Yu, PhD, Associate Professor Pediatrics, Genetics & Genomics, Boston Children's Hospital
Successes in oligonucleotide therapeutics have spurred the creation of bespoke therapies for rare genetic conditions, even for single patients. This talk will review lessons, challenges, and opportunities stemming from these pioneering efforts, and offer perspectives on the ethical and regulatory hurdles to be overcome to realize a future of individualized medicines, whether as proof of concept or provision of care.

4:50pm ADAR RNA Editing: Applying Current Knowledge to Future Applications
Brenda Bass, PhD, Distinguished Professor, Biochemistry, University of Utah
Much is known about biochemical properties of ADAR RNA editing enzymes from decades of in vitro studies, but how these properties correlate with in vivo editing is not always clear. Properties established in vitro will be compared with observations made in vivo, with a focus on properties relevant to therapeutic applications, such as guided RNA editing. Recent progress on how inosine precludes activation of an immune response will be presented.

THURSDAY, MARCH 19

1:20pm Chairperson's Remarks
David Corey, PhD, Professor, Department of Pharmacology, UT Southwestern

1:25pm Venture Philanthropy in Drug Development from a Rare-Disease Patient-Advocacy Perspective
Debra Miller, Founder & CEO, CureDuchenne
CureDuchenne, the leading Duchenne patient advocacy organization, will discuss its initiatives to accelerate the development and regulatory approval of the first drugs to treat Duchenne muscular dystrophy, in addition to its recent efforts supporting the next generation of improved therapeutic products. The presentation will outline existing gaps and strategic opportunities within the development pipeline, focusing on efforts to establish effective treatment options for all Duchenne patients, regardless of their genetic mutation.

2:05pm Oligonucleotide and Delivery Chemistry for siRNA Conjugates: Past Innovations and Future Opportunities
Weimin Wang, PhD, Founder & CEO, Sanegene Bio
RNAi technology is at a crossroads; the focus is shifting from rare genetic disease applications to common chronic indications and general medicine. This transition will need to build upon previous technology by applying new chemistries which further increase the efficacy, safety, tunability and patient accessibility. In this presentation, we will review key medicinal chemistry concepts used in current RNAi medicines and describe next-generation approaches to enable this powerful modality to become a central component of standard-of-care for obesity and cardiovascular therapy.