Pre-Conference Workshop Day

Tuesday, December 7 | 2021

8:30 am | Registration Opens & Welcome Coffee

9:00 am | Antisense Oligonucleotides: Successes & Cross Learnings for RNA Small Molecule Approaches

Antisense oligonucleotides (ASOs) bind to a target RNA in order to modulate protein expression. This rational is not dissimilar to that being employed by the small molecule community, and similarly both modalities exist with challenges preventing clinical success. With this, employing ASO’s to modulate RNA activity is a therapeutic approach which offers valuable insight and cross learning opportunities to be applied to small molecules targeting RNA. This session will consider the various challenges with ASO’s, outline the significant benefit of this approach over small molecules, and most importantly consider what the ASO community can offer to aid the acceleration of small molecules to the clinic.

In this workshop we will discuss:

  • Data driven evidence of preclinical and clinical successes within the RNA ASO community
  • Challenges and bottlenecks of ASO’s targeting RNA
  • Valuable lessons and cross-modality learnings for RNA small molecule approaches

Workshop Leaders:


Satya Kuchimanchi
Vice President, CMC
Triplet Therapeutics


Richard Gregory
Professor, Harvard Medical
School, Co-Director
HMS Initiative for RNA

11:00 am | Morning Refreshments & Networking

12:00 pm | The Use of RNA Analytics to Assess Target Engagement & Enable the Development of PD Biomarkers

Small molecule targeting of RNA, either through direct binding to RNA or through indirect mechanisms such as RNA processing or modifying proteins, has opened major new opportunities for drug discovery. However, one of the major challenges in the field is the development of quantitative and high throughput technologies that allow the accurate determination of small molecule effects on target RNAs. Establishing such technologies is essential for the assessment of target engagement in cells and tissues, and for the development of PD biomarkers.

In this workshop we will discuss:

  • The use of mass spectrometry and direct RNA sequencing to quantify small molecule effects on RNA modifications to determine substrate kinetics and PK/ PD relationships
  • Technologies to characterize small molecule binding to RNA and assess its effect on secondary and tertiary RNA structure
  • Technologies to assess targeting of specific RNAs
  • Targeting and using the RNA-bound proteome

Workshop Leaders:


Oliver Rausch
Chief Scientific Officer
Storm Therapeutics

Byron Andrews, STORM

Byron Andrews
Principal Scientist, RNA
Storm Therapeutics

2:00 pm | Lunch Break & Networking

3:00 pm | Unbossing RNA Biology: Empowering & Employing LMW Drug Discovery in the RNA Biology Space: Enabling Drug Discovery with an RNA Chemical Biology Mindset

RNA biology is not only pervasive, but highly relevant to drug discovery as RNA biology nodes can be associated with or cause various diseases. The complex interplay of RNA and RNA binding proteins in regulating phenotypes may allow for multiple points and MoAs of intervention for a difficult-to-drug targets. From a chemical biology perspective, unlocking RNA biology as a target for LMW would considerably reduce the undrugged space, and can be distilled into 3 key aspects: 1) identification of the most disease-relevant RNA biology phenotypes, 2) elucidation of molecular targets (RNA, RNA-protein interactions) that control these phenotypes, and 3) discovery of chemical matter capable of modulating critical RNA-protein complexes.

Seamless navigation around this phenotype-target-compound triangle is critical to drug discovery efforts in the RNA biology space.

In this workshop we will discuss:

  • Case studies of how navigation around the triangle can look like
  • Role of various assays, technologies in enabling successful navigation around the triangle
  • Challenges and opportunities


Workshop Leaders:

Andrew Krueger, Novartis

Andrew Krueger
Principal Scientist II
Novartis Institutes for Biomedical Research

Connie Wu, Novartis

Connie Wu
Principal Scientist II
Novartis Institutes for Biomedical Research

5:00 pm | End of Pre-Conference Workshop Day