In-Person Registration: Closes Friday, October 6th, 2023.

Virtual Registration: Closes Friday, October 13th, 2023.

Background: Oligonucleotides represent a novel pharmacotherapeutic drug class. While based in the specificity of Watson-Crick base pairing, construct (single and double stranded) and chemical modifications vary and result in distinct advantages but also specific challenges. Single stranded antisense oligonucleotides (ASO) were the first in this drug class. More recent are small interfering RNAs (siRNAs), with the first siRNA approval in 2018. Oligonucleotides have both small molecule and biotherapeutic attributes and there are no class-specific ICH or FDA guidance documents to provide a clear blueprint for nonclinical safety testing, including for developmental and reproductive toxicity (DART) evaluations. Currently, these compounds are regulated as small molecules, but certain compound characteristics aligning to that of biologics raises the need to evaluate if a traditional small molecule DART assessment is appropriate.

Meeting Purpose & Highlights: This workshop will consider application of DART principles to fit attributes of ASOs, siRNAs and other oligonucleotide therapeutics and highlight gaps and challenges associated with determining the most appropriate strategies to account for potential risk to a varied patient population and spectrum of disease conditions. Considerations unique to oligonucleotide therapeutics for DART evaluations that will be discussed include mechanism of action/target engagement, dosing schedule, toxicokinetics and biodistribution, and species or model selection and validation. Speakers will represent institutions actively working in oligonucleotide therapeutic development and will deliver case study examples to illustrate various DART considerations. It is anticipated that most of the session will be reserved for discussion rather than presentation. This workshop will be timely as RNA-based therapeutic development is expanding. Reviewing existing DART strategies and initiating discussion on how to address gaps and challenges will help advance the field toward a fit for purpose and robust characterization of DART risk for this class of compounds.

The workshop will conclude with a scientific panel (forward thinking) discussion based on emerging themes from the session. Panelists will include speakers and regulatory representatives who will provide their opinion (not necessarily that of their organizations) on key questions and challenges that have arisen in the meeting.

Meeting format: In-Person at HESI Offices in Washington, DC and Virtual. In-Person attendance is limited to 50 people.

Anticipated Attendance: This meeting will engage representatives from the HESI DART committee and other invited experts and stakeholders. Registration will be open to virtual and in-person attendees.

Workshop Outputs: The goal will be to develop and submit a manuscript for peer review highlighting key workshop recommendations, gaps, and opportunities.

Information on the HESI Developmental and Reproductive Toxicology Committee can be found HERE.

Hotel Booking Information

A hotel room block is available for the workshop:

The Madison
1177 15th St NW
Washington, DC 20005
(location)
$299/rate

To book, please us this link: https://book.passkey.com/e/50664440

We have a limited number of rooms blocked off, so if you would like to take advantage of this room block I recommend you book your room soon.