8:30 am Morning Networking Coffee

8:50 am Chair’s Opening Remarks

Overcoming Delivery, PKPD & Translational Challenges of Non-Coding RNAs towards Successful Clinical Development

9:00 am Translational Medicine with INT-1B3 (LNP-Formulated miRNA-193a-3p Mimic): From Bench to Bedside

Synopsis

  • MicroRNAs as innovative approach for therapeutic intervention in oncology
  • Pre-clinical development of microRNA mimic in oncology
  • Lessons learnt on translation from pre-clinical to clinical PKPD/toxicology

9:30 am Accelerating Non-Coding RNA Therapeutics into Clinical Stages

Synopsis

  • Moving miRNAs from bench to bedside
  • Next generation heart failure therapeutics
  • Clinical phase 2 design of a miRNA-based program

10:00 am Genome-Wide Screening for Therapeutic Anti-Glioma miR & Exploring Engineered Exosomes for Delivery of miRs in Brain Tumors

Synopsis

  • Screening microRNA library for therapeutic anti-glioma miR
  • Developing engineered exosomes (eExos) that incorporate viral proteins to successfully package, deliver, and express episomal plasmid DNA
  • Packaging of polycistronic miRNAs in engineered exosomes leads to successful delivery to tumours

10:30 am Morning Networking Break

11:30 am Progressing Pre-Clinical & Translational Applicability of Non-Coding RNAs Towards Clinical Development

Synopsis

  • LncRNA are an attractive new area for oncology therapeutics
  • Antisense oligonucleotides can play a key role in inhibiting lncRNAs
  • First lncRNA ASO identified that is progressing to human clinical trials

12:00 pm MicroRNA Delivery & Reprogramming in the Tumor Microenvironment

  • Mansoor Amiji University Distinguished Professor, Professor of Pharmaceutical Sciences, & Professor of Chemical Engineering, Northeastern Univeristy

Synopsis

  • Targeted microRNA delivery to tumor-associated macrophages
  • Role of extracellular vesicles in microRNA transfer in vivo
  • MicroRNA delivery in inflammatory diseases and wound healing
  • Non-viral delivery systems for microRNA therapeutics

12:30 pm Lessons Learned from Pre-Clinical & Clinical Development of MicroRNA Therapeutics

Synopsis

  • Improving screening cascade to mitigate potential off-target effects
  • Harnessing the pleotropic nature of microRNAs
  • Future directions

1:00 pm Networking Lunch Break

Realizing the Future Therapeutic & Investment Opportunities Offered by Non-Coding RNAs

2:15 pm Roundtable Discussion: Communicating the Importance of Non-Coding RNA Therapeutics Compared to mRNAs to Investors

Synopsis

  • Outlining the advantages of different emerging non-coding RNAs in comparison to mRNAs
  • Unravelling different approaches of communicating the importance of non-coding RNA therapeutics to the industry
  • How can more interest be raised for investors?

3:15 pm Regulating Gene Expression by Targeting Regulatory RNAs: A Novel Therapeutic Approach

Synopsis

  • RegRNAs are non-coding transcripts derived from gene regulatory elements that function as key regulators of transcription
  • Targeting regRNAs with antisense oligonucleotides leads to specific modulation of gene expression
  • Therapeutic modulation of gene expression using this approach has been achieved in vivo demonstrating the potential of regRNA targeting to treat a range of diseases

3:45 pm Chair’s Closing Remarks

4:00 pm End of Emerging Non-Coding RNA Therapeutics Summit