PJ Brookes

Company: National Center for Advancing Translational Sciences, National Institutes of Health

Job title: Program Director, Office of Rare Diseases Research

Seminars:

Accelerating Clinical Trials in Rare Diseases by Moving Beyond One Disease at a Time 8:40 am

Exploring platform approaches to gene therapies to accelerate therapeutic development for monogenic diseases How much can we push platform capacity of AAV’s? How can we run clinical trials more efficiently? The need to streamline our current approaches and enable a more scalable, and inherently patient-centric approach Evaluating broader collaborative approaches that the NIH are taking…Read more

day: Day Two

Panel Discussion – Diving into Specific Therapeutic Modalities 10:40 am

Advantages and disadvantages across small molecules, gene therapy, antisense oligonucleotides, gene replacement, stem cell therapy and hybrid strategies With each modality, what needs to happen before the trial, concurrently with the trial and after? What’s the longevity of these approaches? What are the knock on effects of taking these genes out? What is a good…Read more

day: Day Two