Conference Day One

Day One

Wednesday June 16, 2021

9:00 am Registration Opens & Chair’s Opening Remarks

Outlining the Successes & Challenges of Effective RNAi Based Therapies

9:30 am Alnylam & the Past, Present & Future of RNAi Therapeutics

  • Greg Hinkle Vice President, Research Informatics, Alnylam Pharmaceuticals

Synopsis

  • RNAi Therapeutics are a bona fide, novel drug modality
  • Alnylam’s success is based on a marriage of biology and chemistry
  • Alnylam is working to expand the application of RNAi therapeutics beyond the liver

9:55 am MicroRNA Therapeutics: Targeting the Pathways of Human Disease

  • Stefan Engelhardt Professor & Chair of Pharmacology and Toxicology Technical , University of Munich

Synopsis

  • MicroRNAs are short non-coding RNAs that regulate biochemical pathways by RNA interference (RNAi). Dysregulation of microRNAs is associated with many diseases
  • MicroRNA-21 has been implicated with fibrotic diseases and cancer
  • Discussing the development of an anti-fibrotic antisense oligonucleotide (anti-miR-21) for the treatment of a genetic fibrotic kidney disease called Alport Syndrome, and summarizes the potential of anti-miR-21 as a drug to treat hepatocellular carcinoma

10:20 am KRAS-LODER to Target Pancreatic Cancer

Synopsis

KRAS-LODER to Target Pancreatic Cancer:
Ongoing Clinical Results from Phase 2
• LODER is a drug delivery product for regional delivery of RNAi-based drug
• KRAS-LODER shows encouraging clinical results in pancreatic cancer
• Utilization of KRAS-LODER to target NSCLC is in development

10:45 am Live Panel Q&A – Ask the Speakers your Burning Questions!

  • Greg Hinkle Vice President, Research Informatics, Alnylam Pharmaceuticals
  • Stefan Engelhardt Professor & Chair of Pharmacology and Toxicology Technical , University of Munich
  • Rebecca Miles Senior Research Scientist , Eli Lilly & Company
  • Amotz Shemi CEO, Silenseed

11:00 am Morning Break & Virtual Speed Networking

Developing Extrahepatic RNAi Therapeutics: Can RNAi Have Significant Impact on Diseases Outside of the Liver?

12:00 pm Pulmonary Delivery of siRNA Against Beta Corona Viruses

Synopsis

  • Respiratory viruses lack specific therapeutics despite the easy accessibility of the lung
  • Inhaled siRNA nanoformulations could change the therapeutic landscape for lung infections
  • We have developed nanoformulations of siRNA against betacoronaviruses and have tested them in biomimetic models of the lung and in ex vivo human tissue. Additionally, we have developed dry powder formulations for inhalation and improved shelf life

12:25 pm Developing Novel Chemical Architectures for Extra-Hepatic Delivery of siRNA

  • Julia Alterman Senior Scientist Khvorova Lab, University of Massachusetts Medical School

Synopsis

  • Discussing the chemical engineering of therapeutic siRNAs
  • Exploring the therapeutic potential of extrahepatic delivery
  • Outlining existing work and approaches for effective CNS siRNA delivery

12:50 pm Live Panel Q&A – Ask the Speakers your Burning Questions!

  • Olivia Merkel Professor of Drug Delivery, LMU Munich
  • Julia Alterman Senior Scientist Khvorova Lab, University of Massachusetts Medical School
  • Rebecca Miles Senior Research Scientist , Eli Lilly & Company

1:05 pm Networking Lunch

2:05 pm PANEL DISCUSSION: From Theory to Reality: How to Target the Central Nervous System with RNAi Based Therapies

  • Olivia Merkel Professor of Drug Delivery, LMU Munich
  • Julia Alterman Senior Scientist Khvorova Lab, University of Massachusetts Medical School
  • Sean Bedingfield Research Engineer, New Therapeutic Modalities, Cambridge, Eli Lilly and Company
  • Irina Antonijevic Chief Medical Officer, Triplet Therapeutics

Synopsis

In the past few years, RNAi therapeutics have rocketed as a widely promising potential therapy for several diseases. With the major shift of interest to target the central nervous system, it is critical to now consider the viability of targeting the CNS for therapeutic intervention and debate the important next steps to ensure RNAi CNS drugs become a reality.

3:05 pm Afternoon Break & Virtual Networking

Therapeutic Development of Efficient Gene Silencing Drugs

3:35 pm Design of Stable & Functional siRNA-Oligonucleotide Conjugates by Screening Small Molecules that Bind to the Human PAZ Domain of Ago2

Synopsis

  • Outlining a new generation of siRNA oligonucleotide conjugates
  • Discussing the design through screening of small molecules that bind to siRNA binding protein
  • Combining the worlds of small molecule drug discovery and innovation in the field of biological drugs

4:00 pm Therapeutic Development Using Chemically Modified Asymmetric siRNAs

Synopsis

• Asymmetric siRNA (asiRNA) triggers efficient target gene silencing with reduced non-specific effects
• OliX’s proprietary cp-asiRNA for local administration therapeutics, and GalNAc-asiRNA for liver targeting therapeutics
• Describe OliX’s pre-clinical and clinical developments against various disease indications

4:25 pm Live Panel Q&A – Ask the Speakers your Burning Questions!

4:40 pm Chair’s Closing Remarks

5:00 pm End of Conference Day One