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
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
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
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!
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
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!
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
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