2026 Plenary Keynote Program

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 Recent Advancements of Oligo-Conjugates Revolutionizing the Field
Mano Manoharan, PhD, Distinguished Scientist & Senior Vice President, Innovation Chemistry, Alnylam Pharmaceuticals
Chemical modification is the key to the success of making drugs out of oligonucleotides. Breakthroughs in LNPs formulation and trivalent GalNAc conjugation of chemically modified oligonucleotides have paved the way to efficient delivery of these therapeutics to liver. Additional ligands and delivery platforms are on the horizon for delivery to extrahepatic tissues. Lipid-conjugated siRNAs for CNS delivery and antibody-conjugated siRNAs for muscle delivery have entered clinical studies. Antibody-conjugated oligonucleotides are also showing promise for Blood-Brain Barrier penetration. An overview of the chemical modifications, linkers, and targeting ligands for efficient delivery will be presented.