Pre-Conference Workshop Day

Tuesday, December 13 | 2022

8:00 am | Registration Opens & Welcome Coffee

8:30 am | Workshop A – High-throughput Methods for Unraveling Mechanisms & Biological Functions of RNA & Protein Interactions

For most RNAs, defining RNA function and regulatory mechanisms is the principal challenge in advancing from discovery to therapeutic targeting. Recent years have seen the development of diverse sequencing-based assays for integrating nearly every aspect of RNA biology. Here, we discuss several established and emerging technologies for revealing RNA regulation and their application for advancing better-developed drugs with therapeutic relevance.

  • Leveraging an understanding of the endogenous intracellular functions and mechanisms of RNA activity
  • Delving into RNA organization, signaling, and operation to improve drug targeting

Workshop Leaders:

Anthony Mustoe

Anthony Mustoe
Assistant Professor
Baylor College of Medicine

10:30 am | Morning Networking Break

11:30 pm | Workshop B – Delving into the Molecular Toolbox for Drug Discovery: From Targets to Drug Products

As drug discovery remains the touch point of the RNA targeting community, the focus is now on improving tools to ensure that trajectories remain to accelerate toward the clinic. As such, there is a greater need to achieve efficient proof-of-concept to find the right target and be able to drug the undruggable with RNA targeting small molecules. This workshop will delve deep into developing an effective toolbox for drug discovery and beyond.

  • How to achieve a successful proof of concept with validated and prioritized targets?
  • How to decipher different validation levels and hit characterization while improving time efficiency?

Workshop Leaders:


Jennifer Petter
Founder & CIO
Arrakis Therapeutics

Zbigniew Zaslona

Zbigniew Zaslona
VP – Research Biology

1:30 pm | Afternoon Networking Break

2:30 pm | Workshop C – Advancing Preclinical Translation of RNA Small Molecules to Improve Clinical Outcomes

Given the plethora of RNA-targeted therapeutics in preclinical investigations and clinical trials, it is clear that these medicines will likely become a significant therapeutic intervention in the fight against the disease. Despite the enormous therapeutic promise, there are important considerations and substantial obstacles that the industry must address, and a closer look at the progression from preclinical to clinical trials would be extremely helpful in getting these medicines to patients, as will be discussed in this workshop.

  • How to use what we know about RNA binding to a small molecule targeting model in vivo?
  • How to analyze animal model data with cross-species conservation of sequence and structure to aid translatability?
  • What are the right system models to use to test pharmacokinetics, pharmacodynamics, and efficacy

Workshop Leaders:

Anu Bhattacharya

Anu Bhattacharyya
Executive Director – Biology
PTC Therapeutics

Jana Narasimhan

Jana Narasimhan
Executive Director – Pharmacology
PTC Therapeutics