Meeting Structure

Working group members, from academia, industry, and government in multiple fields, have been meeting since February 2021 to identify roadblocks and possible solutions. The working groups will be presenting across the three-day meeting. 

June 3rd will focus on defining:

• Who are the individuals that could benefit from gene-targeted therapies – now and in the future? 

• What are the rare diseases or categories of rare diseases that are most amenable to gene-targeted therapies – now and in the future?

• When is the optimal time to identify individuals who could benefit from gene-targeted therapies (e.g., newborn screening)

June 10th will focus on identifying existing systems that can be leveraged or adapted to bring treatments to the individuals who can benefit from them.

June 17th will focus on identifying gaps that need to be addressed to facilitate the effective dissemination of treatments in an equitable manner.

More information and the meeting structure can be found here!