8:00 am Registration & Morning Coffee

8:50 am Chair’s Opening Remarks

Leveraging Key Learnings from Discovery to Commercialization to Fast-Track Next Generation Small Molecules Targeting RNA

9:00 am Early Discovery Leads to Drug Candidates to Clinic


  • How many of these early discoveries have turned into like late lead drug candidates?
  • How many of those late lead candidates have resulted in upcoming clinical trials?

9:30 am Early Discovery Lessons Learned Along the Path to Risdiplam’

  • Matt Woll Vice President, Chemistry, PTC Therapeutics


  • With the discovery and commercial success of Evrysdi® (risdiplam) in treating SZA, the use of small molecules to influence pre-mRNA splicing has become a fertile ground for drug discovery
  • With hindsight vision, some of the key determinants to successful identification of risdiplam precursors will be discussed, including the importance of a sensitive screening assay and splicing selectivity
  • Discussing key SAR breakthroughs

10:00 am Morning Break & Speed Networking


As the RNA-Targeted community is reunited, this valuable session will ensure you can reconnect with your peers in the room to make new and lasting connections. All attendees will have the opportunity to meet and network with their academic and industry peer

Early Discovery

Optimizing Assays to Determine Functional Effect in Cellular Conditions for Improved Selectivity

11:00 am Utilizing Screening Methodologies to Study Physiological Responses


  • Using RNA-sequencing to determine the specific effects elicited by the small molecules when interacting with the target RNA
  • Identifying the correct confirmations with the use of cell screening for higher predictabilit
  • How to use cell-free RNA-binding assays to identify small molecules that bind RNA and elicit a desired on-target response in cellular conditions

11:30 am Reserved for Momentum



11:50 am Leveraging the Biophysics of RNA Structure & Dynamics to Improve Small Molecule Potency & Selectivity

  • Isaac Kimsey Vice President of Drug Discovery, Base4 Biotechnology


  • Replicating RNA-small molecule binding seen in vitro, in cellular conditions
  • Leveraging current understanding of RNA structure and cellular environment to improve selectivity
  • Understanding novel technologies used to determine small molecule binding RNA targets in the cell

Preclinical & Translation Chair: Li Zhang, Director, Quantitative Clinical Pharmacology, Daiichi Sankyo

Evaluating Model Systems to Accurately Test PK-PD & Improve Therapeutic Index

11:00 am Fire-Side Chat: Understanding Off-Target Effects to Reduce Toxicity In Vivo

  • Anu Bhattacharyya Executive Director Biology/Nucleotide Repeat Disorders, PTC Therapeutics
  • Paul August Chief Scientific Officer, ReviR Therapeutics


  • Assessing how to reduce off-target effects to improve safety and efficacy of drugs and allow for a more targeted approach
  • Utilizing small molecule bioinformatics to better predict offtarget interactions
  • How to increase therapeutic index in preclinical models that successfully translate into the clinic?

11:30 am Reserved for Promega



12:00 pm Using Appropriate System Models to Test Pharmacokinetics & Pharmacodynamics, Allowing Robust Translatability into Humans

  • Simon Xi Chief Executive officer, Rgenta Therapeutics


  • Exploring how PK-PD relationship can be tested within system models & how these can be translated into in vivo models
  • Understanding the relevance of the chosen animal model and whether they express the relevant to perform disease efficacy studies
  • Evaluating tools to test PK-PD & efficacy of RNA-targeting small molecules to improve clinical outcomes

12:30 pm Lunch & Networking Break

Utilizing Computational Tools to Predict Bioactivity & Fast-Track Lead Optimization

1:30 pm Improve Hit Discovery Efficiency & Success Rate Through In Silico RNA Splicing Fingerprints


  • Understand how to carry out phenotypical screens, large screens, NMR-based screens, and Mass-spec based screens to evaluate and determine good bioactivity
  • Exploring a more focused approach to achieve high-value hits to narrow down potential small molecule drugs that can successfully target RNA

2:00 pm Optimizing Selective Binding of Small Molecules to Reduce Off-Target Effects within the Cellular Environment

  • Martina Roos CEO, Co-Founder, President, Sardona Therapeutics


  • Understanding how to determine and select small molecules with high selectivity for RNA targets?
  • Utilizing bioinformatics to understand small molecules with minimal off-target effects, therefore, also predicting the best targets for these small molecule drugs

Bridging the Gap Between Pre-clinical Models & Humans to Improve Translation into the Clinic

1:30 pm Understanding Conservation of RNA Sequence in Preclinical Model Systems

  • Anu Bhattacharyya Executive Director Biology/Nucleotide Repeat Disorders, PTC Therapeutics


  • Analyzing animal model data with cross-species conservation of RNA sequence to aid translatability
  • How can animal models be further manipulated to more accurately replicate human systems?
  • How relevant are the mouse models to the different disease areas?

2:00 pm Small Molecules Targeting RNA Expression: Evaluation of Preclinical Therapeutics in Pathophysiological Models of Disease

  • Paul August Chief Scientific Officer, ReviR Therapeutics


  • Examining translational medicine approaches to demonstrate human efficacy in vitro by utilizing IPSC models that asess small molecule RNA therapeutics for Huntington’s disease
  • Leveraging unique ‘omics analyses to demonstrate robust target engagement and target specificity profile to enhance translational confidence to the clinic
  • Limitations and challenges when utilizing patient derived models in vitro vs. in vivo assessments followed by direct omic comparisons

2:30 pm Afternoon Networking Break

Trailblazing the Future of RNA-Targeted Drug Discovery & Development for an Accelerated Path to the Clinic

3:30 pm Panel Discussion: Overcoming Key Challenges to Improve Drug Design & Discovery for Novel Approaches


  • Navigating the current landscape of small molecules targeting RNA
  • How can the unlimited potential to target RNA with small molecules be explored and exploited to develop novel approaches?
  • What are the lessons from other modalities that can be leveraged to develop small molecules that target RNA? What needs to happen for this modality to become widely adopted?
  • Analyzing the potential of small molecule drugs to treat diseases with unmet medical needs, such as oncology, neurodegenerative diseases and rare & genetic diseases

4:30 pm Examining the Intricacies of the Small Molecule Landscape to Influence Future Direction of RNA-Targeted Drug Discovery

  • Jennifer Petter Founder and Chief Innovation Officer, Arrakis Therapeutics Inc.


  • Where is the RNA-targeting small molecules field headed?
  • Understanding the complexities and risks when developing new approaches to target RNA
  • What are the shortcomings of using small molecules to target RNA that need to be understood and overcome?

5:00 pm Chair’s Closing Remarks & End of Conference Day One