8:30 am | Registration Opens & Morning Coffee Networking

Insight Guided Small Molecule Design & Optimized Strategies for RNA Targeting

9:00 am Sequence-Based Design of Small Molecules Targeting RNA


  • Design of small molecules targeting RNA
  • Factors affecting bioactivity
  • Conversion of binders into potent and specific degraders

9:30 am Enabling Technologies for Targeting RNA-Protein Interaction


  • Highlighting the role of RNA-binding proteins in RNA biology and their druggability
  • Introducing new technologies for assaying RNA-protein interactions in live cells
  • Discussing lessons learned from screening against RNA-protein interactions

10:00 am Targeting RNA with Small Molecules: Tools & Tactics for Medicinal Chemistry


  • Exploring efforts to discover and characterize small molecules that bind to bacterial and human RNAs
  • Discussing our program employing small molecule microarrays to discover and profile RNA binding molecules
  • Discussing efforts to develop structure-guided design of RNA binders

10:30 am | Morning Break & Structured Networking


As the RNA community is reunited, this valuable session will ensure you can reconnect with your peers in the room to make new and lasting connections. Running concurrently as an in-person and digital session, all attendees will have the opportunity to meet and network with their academic and industry colleagues.

Optimizing Selectivity, Specificity & Toxicity & Novel Approaches to Address Unmet Clinical Needs

11:15 am Noncoding RNA Therapeutics – Challenges & Potential Solutions


  • Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of cancers, as well as many other diseases
  • Alternative entities such as anti-miRNAs are undergoing clinical testing, and lncRNA-based therapeutics are gaining interest
  • In this talk, I will discuss key challenges facing ncRNA therapeutics – including issues associated with specificity, delivery, and tolerability and focus on promising emerging approaches that aim to boost their success

11:45 am Modulation of RNA Based Targets in The Area of Cardiovascular, Renal & Metabolism Disease Areas

  • Malin Lemurell Executive Director & Head of Medicinal Chemistry, AstraZeneca


  • A background to the biology and the rational for the approaches taken
  • Examples of modulation of miRs using small molecules
  • Aspects of assays and screening

12:15 pm Targeting mRNA Biology in a Tissue Selective & Disease-Specific Manner with Small Molecules


• mRNA Lightning: a novel platform for the discovery of selective small molecule mRNA drugs and their mechanisms of action
• Selectivity of mRNA biology: tissue selectivity in mRNA translation regulation, in mRNA transport and localization
• Wholly owned pipeline of 18 programs including among others: fibrosis (collagen I translation inhibitors for fibrotic diseases), oncology (c-MYC and K-RAS translation), and neuroscience (Tau translation inhibitors)

12:45pm | Lunch Break & Networking

1:30 pm Fireside Chat & Networking Session: Biopharma & Investment Partnering – Discussing the Future Landscape of Investment & Collaborations for RNA Small Molecule Space

  • Yang Zheng Head of Business Development, Nymirum
  • Yochi Slonim Co-Founder & CEO, Anima Biotech
  • Beth Shafer Head of Neuroscience, Drug Discovery Sciences, & Externalization Business Development, Takeda Pharmaceuticals International Co.


Small molecule drugs exist as a critical element under consideration for the biopharmaceutical industry. This community thrives on strategic partnerships, permitting cross-functional sharing of knowledge, skills, and experience across multiple disciplines to truly advance RNA targeted small molecules to the patient. This session is dedicated to shining a spotlight onto the advantages and challenges of biopharma partnering and opening the floor to discussions with peers surrounding experiences with partnering and facilitating industry connections.

2:15 pm Panel Discussion: Accelerating the Preclinical Translation of RNA Small Molecule Drugs


Despite the continuous work and successes within RNA small molecule drugs, streamlining RNA therapeutics through preclinical translation remains a critical challenge. Target selectivity and specificity largely exists as a significant and critical hurdle within this community and salient questions remain unanswered.

This panel will discuss:
• How we can optimize the selectivity, specificity, toxicity and advance RNA small
• molecules into the clinic?
• Consider existing literature, novel
• approaches and future landscape to successfully streamline RNA targeted drugs into the clinic

3:00 pm | Afternoon Break & Networking

Navigating the Existing & Future Landscape of Innovative Approaches to Target RNA with Small Molecules

3:30 pm RMP-Targeted Cancer Drug Discovery


  • RNA modifying proteins (RMPs) represent a large class of novel targets for oncology and other disease indications that can be targeted with small molecule inhibitors
  • The enzyme ADAR1 (Adenosine Deaminases Acting on RNA) catalyzes the majority of A-to-I editing, where it has been demonstrated to effect coding sequence, miRNA function and the detection of repetitive elements by innate immune pathways
  • Inhibition of ADAR1 has potential as both a monotherapy for tumors with high Type I interferon signaling and in combination with checkpoint inhibitors in relapsed/refractory tumors; target validation and current efforts in drug discovery

4:00 pm RNA Small Molecule Targeting – Exploring Ladder Therapeutic’s Novel Approach to Target RNA


  • Building a “digitally native” RNA-targeting company – working at the intersection of ML / Biology
  • Discussing the challenges and opportunities from targeting RNA with small molecule
  • Reviewing our approach to target RNA

4:30 pm Approaches to Modulating RNA Processing

  • Peter Smith Chief Scientific Officer, Remix Therapeutics


  • Understanding data science approaches to interpreting genomic data for RNA processing variation
  • Using small molecules as tools for RNA processing research
  • Leveraging opportunities for therapeutic intervention

5:00 pm End of 4th RNA-Targeted Drug Development Summit