8:00 am Registration & Morning Coffee

8:50 am Chair’s Opening Remarks

Emerging Non-Coding RNA Therapeutics: Current Findings & Looking into the Future

9:00 am Breakfast Panel: What does the Future Landscape of Emerging Non-Coding RNA Therapeutics Look Like?

Ranjan Perera
Director RNA Center & Associate Professor, Johns Hopkins University



  • What are considered as emerging non-coding RNA therapeutics and where is innovation expected to come from?
  • Outlining the advantages and opportunities that emerging long non-coding RNA therapeutics and microRNAs offer
  • Discussing what the next steps are in the research to accelerate these novel therapeutics into the clinic

10:00 am Morning Break & Speed Networking


This session is the perfect opportunity to get face-to-face time with the key opinion leaders in non-coding RNAs.

Establish meaningful business relationships to build upon for the rest of the conference and gain individual insight into the pioneering work ongoing.

Nucelic Acid Targeting Approaches for Improved Target Validation of Non-Coding RNA Therapeutics

11:00 am Therapeutic Targeting of LncRNAs with Next Generation Antisense Oligonucleotides in Cancer


  • Discussing the potential of targeting lncRNAs with next generation antisense drug for cancer
  • The technology also enables the rapid target validation of candidate lncRNAs in vitro and in vivo with the FLAME discovery engine
  • Identification and selection of clinical candidate Malat1rx for the treatment of cancer

11:30 am Targeting Immune Priming LncRNAs (IPLs) with Antisense Oligonucleotides to Treat Inflammatory Diseases


  • Lemba has developed the MOSAICTM machine learning AI-engine to identify ASO lead candidates
  • Lead ASOs reduced systemic inflammation and decreased mortality in a humanized model of acute inflammation
  • Targeting IPLs reduced inflammation-mediated choroidal neovascularisation in a porcine model of wet age-related macular degeneration (AMD)

12:00 pm High-Throughput Discovery & Characterization of Long Non-Coding RNA Targets Using Arrayed CRISPR Screens

  • Pieter Mestdagh Professor; Principal Scientist, Ghent University; CellCarta


  • Arrayed CRISPR-interference for lncRNA target knockdown
  • Cellular and molecular phenotyping
  • Live cell imaging and high-throughput RNA-sequencing

12:30 pm Networking Lunch

Expanding Long Non-Coding RNAs Druggability Beyond Oncology

1:30 pm Transformational RNA-Guided Programmable Therapeutics to Target Disease-Causing Cell-States

  • Samir Ounzain Co-founder, CEO & Board Director, HAYA Therapeutics


  • LncRNAs as druggable ‘information processing units’ for programming cell-states
  • High priority lncRNA target discovery in fibrosis associated cell-state changes
  • Considerations for lncRNA ‘druggability’

2:00 pm A LncRNA-Based Treatment of Hypertrophic Cardiomyopathies


  • Delving into lncRNA-based treatment of hypertrophic cardiomyopathies
  • Optimized delivery of lncRNAs to cardiac muscle
  • Exploring mode of action studies

2:30 pm Afternoon Networking Break & Poster Session

Identification & Selection of Potent Long Non-Coding Therapeutic Targets & Clinical Candidates in Oncology

3:30 pm Advancing Novel Target & Drug Discovery Engine to Drug Non-Coding RNAs


  • Non-coding RNAs and their interacting proteins represent a vast class of therapeutic targets
  • NextRNA leverages a proprietary target validation platform to identify and prioritize long non-coding RNA therapeutic programs
  • Developing small molecules to specifically disrupt ncRNA-protein interactions

4:00 pm The Therapeutic Potential of the LncRNA lnc-HLX-2-7 in Group 3 Medulloblastomas in Children

  • Ranjan Perera Director RNA Center & Associate Professor, John Hopkins University


  • Delving into medulloblastoma (MB), an aggressive brain tumor that predominantly affects children
  • Identifying a novel oncogenic lncRNA, lnc-HLX-2-7, as a potential therapeutic target in group 3 MBs
  • Analyzing the importance of the lnc-HLX-2-7-HLX-MYC axis in regulating group 3 MB progression
  • Providing a strong rationale for using lnc-HLX-2-7 as a specific and potent therapeutic target for the group 3 MBs in children

4:30 pm Targeting LncRNAs to Impact Breast Cancer Progression

  • David Spector Professor & Director of Research, Cold Spring Harbor Laboratories


  • LncRNA misregulation is associated with a wide range of cancer types
  • LncRNAs represent a relatively overlooked class of therapeutic targets
  • Nucleic acid therapeutic targets and nucleic acid therapeutics

5:00 pm Chair’s Closing Remarks & End of Conference Day One