Nikolai Naryshkin
Company: Ribonaut Therapeutics
Job title: Chief Scientific Officer
Bio:
Nikolai Naryshkin is Chief Scientific Officer at Ribonaut Therapeutics. He has 20+ years of experience in targeting RNA with small molecules and nucleic acid derived therapeutics. At PTC Therapeutics, he led the SMA program that, in partnership with SMA Foundation and Roche, culminated in regulatory approval of Evrysdi (risdiplam), a first in class oral small molecule targeting pre-mRNA splicing, for the treatment of spinal muscular atrophy. A major focus of Dr. Naryshkin’s work has been the discovery and development of drug candidates targeting gene expression pathways in rare genetic disorders and diseases of altered gene expression.
Seminars:
Emerging Modalities Beyond Splice Modulation: Harnessing Covalent Binders, Proximity-Inducing Compounds & lncRNA Targets to Discover Functional RNA Binders 1:00 pm
While splice modulation has paved the way for RNA-targeted therapeutics, the next frontier lies in unlocking the therapeutic potential of directly targeting RNA. This workshop explores innovative strategies exploring covalently binding RNA, bivalent compounds inducing proximity between RNA and effector molecules, and the untapped opportunity of targeting long non-coding RNAs. Attendees will cover: • Exploring…Read more
day: Pre Conference workshop day
Panel Discussion: Building Strategic Collaborations & Investment Pathways to Accelerate RNA-Targeted Drug Discovery Across Biotech & Pharma 8:45 am
• Discussing the importance of pharma-biotech-academic collaborations and what each sector can provide to successfully create RNA-targeted therapeutics • How are large pharma and biotech companies structuring risk-sharing partnerships to access innovation and accelerate RNA-targeted therapeutic pipelines • How can early-stage companies leverage academic collaborations, tool compound sharing and co development deals to validate targets…Read more
day: Conference Day Two