About Event

Welcome to the 4th RNAi-Based Therapeutics Summit

2022 has been an exciting year for the RNAi field with Alnylam getting both EMA and FDA approval of AMVUTTRATM, Atalanta Therapeutics launching with $110m, Siranomics dosing the first patient with STP705, Arrowhead enrolling their first patient evaluating HZN-457, RNAi therapeutics have reached an inflection point in 2023.

For the very first time, we have seen the release of FDA guidance on clinical pharmacology considerations offering some guidance for drug developers to advance their pipelines.

Despite this, targeted delivery, process development, selectivity, and limited half-life remain big challenges to overcome so the 3-day jam-packed agenda at the 4th RNAi-Based Therapeutics Summit returned to unite 90+ trailblazers, pioneers, and new entrants to turbocharge RNAi drug development.

After listening to your feedback in 2022 and researching with industry leaders, the 2023 conference covered:

Deep dive into delivery mechanisms, including lipid conjugates, that are targeting the skin, eyes, and CNS that is expanding the therapeutic target space for siRNA therapies with, Eli Lilly & Altamira Therapeutics
Hear key clinical updates of siRNA candidates tackling oncology indications as well as new targets that are being explored with Siranomics
Uncover computational modeling approaches to enhance target identification, improve biological understanding, and fast-track siRNA discovery with e-Therapeutics & Tallas Biotech
Revealing next-generation dual targeting siRNAs with improved tolerability and efficacy for robust silencing in the brain with University of Massachusetts & Argonaute RNA
Learn about the strategies for successful scale-up and ways to tackle quick turnaround times to meet the demands for in-person trials whilst ensuring robust production processes with Olix Pharmaceuticals

What Was New for 2023?

A Pre-Conference Focus Day: Unveiling Next-Generation Delivery Methods to Get RNAi Therapeutics Into Extrahepatic Tissues

Whilst GalNAC has proven successful in delivering RNAi therapeutics to the liver, one of the biggest challenges facing RNAi drug developers is achieving targeted delivery to extrahepatic tissues. Join this dedicated focus day to learn about new delivery technologies and platforms targeting the skin, eye, CNS, oncology & beyond.