Pre-Conference Workshop Day
Tuesday, April 19

8:30am Morning Networking Coffee

Workshop A - Delving into the Molecular Mechanisms & Biological Functions of siRNA

Tuesday, April 19 | 9:00am

With the current developments in the siRNA field, there is a lack of clarity in the fundamental underpinnings to the mechanistic basis of siRNA transport and delivery. Here we go back to basics, revealing the missing points to loading dsRNAs, understanding the mechanisms of RISC complex loading and deep diving into gene silencing mechanisms to advance better developed drugs with therapeutic relevance.

• Leveraging understanding of the endogenous intracellular mechanisms of siRNAs.

• Investigating the mechanisms underlying activation of transcription by RNAi complexes.

• From a systems biology approach, exploring the mechanistic gene silencing induced by RNAi complexes.

Picture1

Xiabo Zhong
Professor of Pharmacology & Toxicology
University of Connecticut

11:00am Morning Networking Break

Workshop B - Pre-Clinical, Clinical Development & Trajectories of RNAi Therapeutics
for Cardiovascular Disease
Tuesday, April 19 | 12:00pm 

RNA therapeutics allow targeting of previously undruggable mechanisms leading to a next generation of mechanism-focused approaches. Here we will discuss chances and bottlenecks of RNAi preclinical (including discovery, preclinical development and toxicology studies) and clinical development (phase 1, phase 2) of RNA therapeutics for cardiac diseases with a special focus on common and rare heart failure-related diseases.

• How to build an optimal pipeline for preclinical development of RNAi therapeutics.

• Update on targeted delivery approaches to the cardiovascular system.

• Optimal design of early clinical trials of RNAi approaches in the cardiovascular disease space.

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Thomas Thum
Professor & Institute Director
Hannover Medical School
& CSO & Co-Founder
Cardior Pharmaceuticals

2:00pm Networking Lunch

Workshop C - siRNA-Based Modulation of Emerging Disease Targets: Case Studies of
Three Unique Challenges
Tuesday, April 19 | 3:00pm 

This workshop uses three case studies to underscore and describe challenges commonly faced during the development of siRNA therapeutics. The case studies discussed will be:

1) siRNA efficacy in the nucleus: The story of HTT1a

2) RNAi based Immunomodulation of emerging targets for the treatment of vitiligo and other autoimmune disorders

3) Establishing a framework for antiviral siRNA discovery and development for SARS-CoV-2

• What are the factors currently limiting siRNA efficacy and how can we can overcome them?

• What are the potential side effects of systemic vs local delivery strategies?

• What are the challenges facing anti-viral siRNA development in the face of rapidly evolving pandemics?

daniel

 

Daniel O’Reilly
Post Doctoral
Associate
Khvorova Lab RNA Therapeutics Institute

vignesh

Vignesh N. Hariharan
Post Doctoral
Associate
Khvorova Lab RNA Therapeutics Institute

qi

Qi Tang
Post Doctoral Associate
Khvorova Lab RNA Therapeutics Institute

5:00pm End of Pre-Conference Workshop Day