Overview

This webinar will examine how oligonucleotide therapeutics can be designed to modulate gene expression through multiple mechanisms and beneficially impact genetically defined rare diseases. Our first presenter will provide an overview of the novel opportunites provided by RNA therapeutics in rare diseases, while our second from FDA will discuss aspects unique to rare disease indications from the FDA regulatory perspective. The final presenters will describe development of clinical candidates for rare diseases.

Featured topics

Overview of RNA Oligonucleotide Therapeutics

FDA’s perspective on Therapeutics in Rare Diseases

Progress in SMA

RNA Editing Oligonucleotides: A New Class of Medicines

Who should attend?

Professionals who work in the area of:

Biotechnology

Clinical Pharmacology

Clinical Research

Chemistry, Manufacturing, and Control

Clinical, Regulatory, and Business Development

Delivery Technologies

Drug Discovery

Preclinical

Quality Assurance

RNAi

Vaccines

Learning objectives

At the conclusion of this webinar, participants should be able to:

Understand the broad scope of opportunities and challenges faced in rare disease indications

Assess the experience of development of clinical canidates in rare diseases

Understand current approaches for modulating gene expression in genetically defined rare diseases

Have an account?

Complimentary - Oligonucleotide-Based Therapeutics in Rare Diseases

Overview

Featured topics

Who should attend?

Learning objectives

Be informed and stay engaged.