8:00 am Check In & Light Breakfast
09:00 am | Workshop A
Maximizing Bioavailability of RNAi Therapies with Oligonucleotide Conjugates to Reach Previously Undruggable Therapeutic Targets
Synopsis
Whilst the hunt for next-generation delivery platforms continues, and the scope for targeting extra-hepatic tissue increases, the success of RNAi therapeutics is limited if bioavailability is not addressed. For RNAi therapies to successfully induce gene silencing, the payload must be released effectively to the target tissue. As such, this workshop will address:
- Utilizing machine learning and algorithms to identify targets and design precise siRNA sequences
- How siRNA conjugates can improve targeted delivery and bioavailability in extrahepatic tissue
- How to select optimized delivery systems which amplify bioavailability whilst maintaining target specificity
11:00 am Morning Break & Networking
11:30 am | Workshop B
Streamlining Translation & De-risking RNAi Therapeutic Development with Optimal Selection of Preclinical Models & Appropriate Assays to Ensure Tolerable, Potent Therapies
Synopsis
There are many promising RNAi therapeutic candidates at both the preclinical and clinical phase of development, but few drugs have yet been approved. To maximize clinical success, the right preclinical models should be used, mimicking the genotype of human patients. Assays to determine toxicity and efficacy must be precise, and dosing should be considered. Therefore, this Workshop will address:
- How to derisk novel chemical modifications in in vitro and in vivo preclinical models to safeguard against off-target effects and select animal models which mimic human genotype and pathology
- How to identify assays to determine toxicity, immuno-stimulation and therapeutic potency
- How to plan effective clinical trials considering human dose projections, PK and toxicity
- How to consider IND filing and regulatory approval throughout the translational pipeline
1:30 pm Lunch Break & Networking
02:30 pm | Workshop C
Seizing the Value of RNAi Therapeutics for Oncology to Develop Precise, Effective Treatments for Patients
Synopsis
RNAi therapeutics holds promise for treating complex genetic diseases such as cancer by targeting multiple oncogenes, but release of the therapeutic payload must be rapid and precise, and the heterogeneity of tumors must be considered. Therefore, this Workshop will address:
- How to identify genetic targets in heterogenous tumors to increase correlation in response at the preclinical and clinical stage
- How to design miRNA to target multiple oncogenes to overcome tumor resistance and limit toxicity
- How to integrate chemotherapeutic nucleoside analogs into miRNA to amplify tumor killing and induce a durable anti-cancer response
- How to stimulate release of siRNA from the endosome to invoke a rapid response against tumor cells