ARVO+Genentech Keynote Series

May 3 & 7 | 10:15am MT | Colorado Convention Center

2026 Opening Keynote

Sunday, May 3— bluebird ballroom, Colorado Convention Center 10:15 - 11:45 AM

A Farsighted Perspective on Nucleic Acid Therapeutics for Eye Disease

Thomas R. Cech, PhD
Distinguished Professor of Biochemistry, Univ. of Colorado Boulder
Investigator, Howard Hughes Medical Institute
Nobel Prize in Chemistry, 1989

With the recent success of mRNA vaccines and the invention of CRISPR gene editing, nucleic acid therapeutics are at a crossroads. It’s an exciting time when we simply do not yet know if RNA will become a major paradigm in medicine or will have more limited applications. In his keynote, Cech will first review RNA research discoveries using examples from his recent book, The Catalyst: RNA and the Quest to Unlock Life’s Deepest Secrets (W.W. Norton). He will then discuss the potential of nucleic acid therapeutics to treat hereditary genetic diseases of the eye, including Leber congenital amaurosis (LCA), as well as age-related macular degeneration (AMD). Nucleic acid therapeutics offer novel advantages as well as challenges compared to traditional small-molecule therapeutics.

After his PhD in chemistry from the University of California, Berkeley and postdoctoral research at the Massachusetts Institute of Technology, Cech joined the faculty of the University of Colorado Boulder, where he became a Howard Hughes Medical Institute (HHMI) investigator in 1988 and Distinguished Professor of Chemistry and Biochemistry in 1990.

In 1982, Cech and his research group announced that an RNA molecule from the pond animal Tetrahymena could catalyze biochemical reactions, the first exception to the long-held belief that only proteins could act as enzymes. This work led to the 1989 Nobel Prize in Chemistry, shared with Sidney Altman of Yale University.

In January 2000, Cech moved to Maryland as president of HHMI, the largest private biomedical research organization in the U.S. He returned to research and teaching at CU Boulder in 2009, where he also served as the founding director of the BioFrontiers Institute until 2020.

His passion for communicating science to the general public led him to write The Catalyst which The Economist heralded as one of its Best Books of 2024. Cech’s awards include also the Heineken Prize of the Royal Netherlands Academy of Sciences (1988), the Canada Gairdner International Award (1988), the Albert Lasker Basic Medical Research Award (1988), and the National Medal of Science (1995). In 1987, he was elected to the U.S. National Academy of Sciences and also awarded a lifetime professorship by the American Cancer Society. In 2000, he was elected to the National Institute of Medicine.

Outside the laboratory, Cech enjoys hiking in the mountains, downhill skiing and cooking.

2026 Thursday Keynote

thursday, May 7— bluebird Ballroom, Colorado Convention Center 10:15 - 11:30 AM

Modeling, Targeting and Healing: Stem Cell Strategies to Combat Neurodegeneration in the Brain and Eye

Sally Temple, PhD
Scientific Director
Neural Stem Cell Institute (NSCI)
With commentary from NEI Director Michael F. Chiang, MD, FARVO

Age-related neurodegenerative diseases demand advanced, human-relevant models to understand underlying disease mechanisms and develop therapeutic strategies. We report three interconnected strategies leveraging stem cell technology to address Frontotemporal Dementia (FTD-tau), Alzheimer's disease (AD), and Age-related Macular Degeneration (AMD).

First, we developed a scalable protocol to produce high-quality cerebral cortical organoids and used these to unveil disease mechanisms underlying FTD-tau. Utilizing these organoid models, we engineered fully human bifunctional anti-tau-PEST intrabodies that reduced toxic tau and significantly improved neuronal survival, confirming the efficacy of targeted tau degradation. Second, we used a multi-omic atlas of human cerebrovascular cells to reveal that the APOE4 Alzheimer’s disease risk allele confers a pro-inflammatory phenotype and barrier dysfunction. In iPSC-derived FTD-tau neurovascular models, pharmacological inhibition of JAK-STAT rescued APOE4-linked impairments, establishing a direct therapeutic target. Finally, addressing AMD, we applied our discovery of RPE stem cells (RPESC) to create RPESC-RPE-4W, a post-mitotic progenitor cell product for RPE cell replacement therapy. Our ongoing first-in-human clinical trial shows positive safety and tolerability of RPESC-RPE-4W implantation therapy for dry AMD, with some participants achieving an average gain of 21.67 letters in visual acuity.

Our three-pronged translational approach effectively incorporates current breakthroughs in stem cell brain organoids (for FTD-tau), multi-omic bioinformatics (for AD), and RPESC progenitor progeny (for AMD). Progress across all three interconnected efforts, from understanding disease mechanisms to implementing interventions, benefits from integrating Artificial Intelligence (AI). We utilize AI across the entire translational spectrum, from identifying critical targets in GWAS and modeling complex drug interactions to optimizing the robust manufacturing processes for our RPE progenitor cells. As advances in AI emerge, our field is poised for a significant acceleration in therapeutic development.

At NSCI, Sally Temple leads research programs focused on how stem cells contribute to the development, maintenance, and repair of the central nervous system. She earned her undergraduate degree in developmental neuroscience from the University of Cambridge, UK, and her PhD in optic nerve development from University College London. During her postdoctoral studies at the University of Miami, Temple demonstrated the existence of stem cells in the embryonic mammalian central nervous system, a foundational contribution to the neural stem cell field. In recognition of her pioneering work, she received a MacArthur Fellowship.

Temple has served as both president and board member of the International Society for Stem Cell Research (ISSCR) and was elected to the U.S. National Academy of Medicine. Her current research spans basic and translational neuroscience, including the development of stem cell–based therapies for age-related macular degeneration (AMD) — a clinical-stage program she co-leads with her husband, Jeffrey Stern, MD, PhD — and the use of patient-derived stem cell models to study AMD and tauopathies, with the goal of uncovering disease mechanisms and advancing regenerative treatments.

The ARVO+Genentech Keynote Session is sponsored by the ARVO Foundation through the generous support of Genentech.