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Monday, June 15 • 1:30pm - 2:30pm
#121 SL: Non-Profit Organizations Role in Accelerating Preclinical Compound Testing to Fuel the Rare Disease Clinical Pipeline

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-20-681-L04-P; CME 1.00; IACET 1.00; RN 1.00

A panel will review examples of preclinical drug screening platforms and consortia organized by non-profit organizations. Experts will present their experience and describe the challenges encountered, solutions found, and best practices defined.

Learning Objectives

Discuss how rare disease non-profit organizations accelerate drug development; Develop consensus with industry, academia and patients; Identify unmet needs, optimize translational methods, criteria for compound selection, addressing gaps, ensuring quality, reproducibility, robustness and interpretation of data; Discuss challenges and solutions to transparency, data sharing and sustainability.


Daniela Brunner, PhD


Dean Aguiar, PhD

Salvatore La Rosa, PhD

Dominique Pichard, MD

avatar for Daniela Brunner

Daniela Brunner

Founder/CIO, Science Forward/PsychoGenics
Daniela Brunner, PhD is the Chief Innovation Officer of PsychoGenics inc. and founder of ScienceForward, which is focused on the development of digital health technology and treatments for rare disorders and underserved populations. She is part of the Business Advisory Board of the... Read More →
avatar for Salvatore La Rosa

Salvatore La Rosa

Chief Scientific Officer, Children's Tumor Foundation
Salvatore La Rosa, PhD, serves as the Chief Scientific Officer at the Children’s Tumor Foundation (CTF) and he is responsible for the implementation of the Foundation’s business strategy into research projects. He manages the foundation’s research activities, providing scientific... Read More →
avatar for Dominique Pichard

Dominique Pichard

Chief Science Officer, Rettsyndrome.org
Dominique Pichard, MD is the Chief Science Officer at the International Rett Syndrome Foundation (IRSF). In this role, she is responsible for the research program that includes funding of basic, translational, clinical, and neurohabilitation research, the Scout program to accelerate... Read More →
avatar for Dean Aguiar

Dean Aguiar

Director, Preclinical Research, Tuberous Sclerosis Alliance
Dean Aguiar, PhD is the Director for Preclinical Research at the Tuberous Sclerosis Alliance, where he is responsible for leading the Tuberous Sclerosis Complex Preclinical Consortium, a private-public partnership developed to stimulate collaboration and innovation to find a cure... Read More →

Monday June 15, 2020 1:30pm - 2:30pm EDT
TBD Virtual Event Horsham, PA 19044
  06: PreClin Dev-EarlyPhaseCR, Forum