7:00 am Morning Networking Coffee

7:50 am Chair’s Opening Remarks

Advancements in Targeted Protein Stabilization Showcasing the Therapeutic Potential of Induced Proximity

8:00 am Harnessing Deubiquitinases (DUBs) for Targeted Protein Stabilization (TPS) & Restoration of Protein Function

  • Scott Kanner Associate Director - Platform Development, Stablix

Synopsis

  • Identifying therapeutically relevant proteins for targeted stabilization
  • Profiling novel ligands for DUB recruitment
  • Presenting novel insights into TPS and RESTORAC development 

8:30 am Using Small Molecular Glues for Selective Stabilization of Clinically Beneficial 14-3-3 Protein Interfaces

  • Ying Zhang Executive Director, Ambagon Therapeutics Inc

Synopsis

  • Identifying key 14-3-3 protein complexes that can be stabilized for therapeutic effect
  • Studying the binding grooves that 14-3-3 interaction unveil for targeting through small molecules
  • Hunting for natural and synthetic molecular glues that can stabilize clinically relevant 14-3-3 complexes

9:00 am Presenting Protein Rescue Targeting Chimeras (PRESTACs) for Targeted Protein Stabilization

Synopsis

  • Leveraging the targeted stabilization of therapeutically relevant proteins which can be achieved by recruiting Deubiquitinases (DUBs) to deubiquitinate target proteins and prevent their degradation
  • Exploring how heterobifunctional molecules, called Protein Rescue Targeting Chimeras (PRESTACs), can be generated by chemically linking a DUB ligand to a target protein ligand
  • Understanding how Progenra has discovered and characterized novel small molecule DUB ligands which are used to design and synthesize PRESTACs to stabilize disease-relevant targets
  • Learning strategies for discovering and characterizing novel DUB ligands using Progenra’s UbiProTM platform will be discussed

9:30 am Morning Break & Networking

10:00 am PANEL DISCUSSION: Tackling the Unique Challenges that Novel Proximity Approaches Face for Efficient Drug Development

  • Susanne Saalau Vice President - Biochemistry & Biophysics, Photys Therapeutics
  • Kyle Eastman Senior Director, Halda Therapeutics
  • Nate Hathaway Associate Professor, University Of North Carolina At Chapel Hill
  • Nan Ji CEO, PAQ Therapeutics

Synopsis

Addressing the differences between targeted protein degradation and other modalities, along with the challenges that arise from this, such as:

  • What are the benchmarking standards when creating new protein species to test the efficacy of your drugs?
  • How to address the reversibility of post-translational modifications for sustained therapeutic effect?
  • Increasing the bio-chemical and bio-physical understanding of new reactive protein species to drive specific pharmacological effects

From Post-Translational Modifications to Transcriptional Control: Uncovering Emerging Modalities Pushing the Frontiers of Induced Proximity

10:45 am PANEL DISCUSSION: Finding the Gaps – Discussing the Unmet Needs that Novel Modalities Are Filling to Push the Therapeutic Limits of Induced Proximity

  • Ying Zhang Executive Director, Ambagon Therapeutics Inc
  • Mikko Taipale Assistant Professor - Molecular Genetics & Research Chair - Functional Proteomics & Proteostasis, University of Toronto
  • Goutham Narla Professor, Department of Internal Medicine / Chief, Division of Genetic Medicine, University of Michigan

Synopsis

  • Evaluating hard-to-drug targets that novel approaches can unlock
  • Bridging the gap between a clinically relevant target and a therapeutic modification
  • How to design unbiased screens to find binders across the proteome

11:30 am Regulated Induced Proximity Targeting Chimeras: Using RIPTACs to Selectively Kill Cancer Cells

Synopsis

  • Describing a novel “hold & kill” approach to selectively treat cancers
  • Elucidating the mechanism of action of RIPTACs
  • Accelerating the proof-of-concept gain for further novel RIPTAC therapies

12:00 pm Networking Lunch

1:00 pm Precision Control of Transcription Using Bio-orthogonal Chemical Epigenetic Modifiers

  • Nate Hathaway Associate Professor, University Of North Carolina At Chapel Hill

Synopsis

  • Understanding how bio-orthogonal Chemical Epigenetic Modifiers allow for dose dependent gene activation
  • Leveraging chemical induced proximity with small bifunctional molecules that bridge gene targeting systems with endogenous gene activators
  • Exploring novel translational applications of this technology in human disease 

1:30 pm Showcasing Chemically Induced Proximity & CRISPR Strategies to Regulate RNA Modifications

  • Fu-Sen Liang Associate Professor, Case Western Reserve University

Synopsis

  • Understanding the function and disease relevance of RNA modifications
  • Leveraging an inducible CRISPR platform for m6A editing
  • Engineering a split Cas13b system for RNA regulation

2:00 pm Inducing Dephosphorylation of Kinases by Inducing Proximity Between PP1 & AKT1 & EGFR (Virtual)

Synopsis

  • Showcasing proof of concept for inducing proximity between a phosphatase (PP1) and kinases (AKT and EGFR) Elucidating the chemical strategy for heterobifunctionals that recruit phosphatases
  • How to identify key targets that are solvable through phosphatase activity

2:30 pm Afternoon Break & Networking

3:00 pm Navigating the Interplay of Acetylation & Ubiquitination

  • George Burslem Assistant Professor, Perelman School of Medicine University of Pennsylvania

Synopsis

  • Understanding how acetylation of non-histone proteins has emerged as an important mechanism of regulation for the ubiquitin-proteasome system, particularly at the level of E3 ligase substrate recognition
  • How to identify a subset of proteins with stability regulated at the level of post-translational acetylation
  • Leveraging these acetylation sites that can be altered by induced proximity modalities for desired effects

3:30 pm Targeting Histone Acetylation & Key Structure-Function Relationships

  • Jun Qi Assistant Professor - Medicine, Dana-Farber Cancer Institute

Synopsis

  • Dissecting the structural function of HAT containing protein
  • Using induced proximity to change the core-regulatory circuitry network
  • Studying proximity induced degradation impact on the protein complex in cancers

4:00 pm Examining Proof of Concept (PoC) Studies of Catalytic, Proximityinduced, Autophagy-dependent Degradation of Proteins & NonProtei

  • Nan Ji CEO, PAQ Therapeutics

Synopsis

  • Comparing and contrasting the ubiquitin-proteosome system vs autophagy-lysosome system
  • Studying the PoC studies of proximity-induced, autophagy-dependent degradation
  • Elucidating the potential of autophagy-based, targeted degradation

4:30 pm Chair’s Closing Remarks & End of Day Two