About Event

Welcome to the 2nd Rare CNS & Neurodevelopmental Drug Development Summit

Returning in Boston for the second edition, the 2nd Rare CNS & Neurodevelopmental Drug Development Summit is the only meeting focused on advancing symptomatic and disease modifying treatments across rare genetic epilepsies and neurodevelopmental disorders.

Don’t miss out on the chance to unite with drug sponsors, researchers, foundations, and patient advocacy representatives to discuss case studies and lessons learned. This is the industry’s definitive opportunity to collaborate with patient groups and gain regulatory clarity on clinical trial design and endpoints, biomarkers and outcome measures, delivery and toxicity concerns.

This is your opportunity to:

Establish lasting connections with this dedicated community at the first ever-industry led meeting focused on advancing symptomatic and disease modifying treatments across neurodevelopmental disorders

Gain an important market update on the progress of neurodevelopmental drug development through pioneering clinical case studies leveraging diverse approaches across gene therapy, gene replacement, ASO’s and small molecules

Engage in critical discussions with world leading industry and academic experts and discover novel outcome measures that measure incremental change and how the industry is driving design innovation across disease specific clinical trials

1st Rare CNS & Neurodevelopmental Drug Development Summit Reviews:

“Great set of talks and glad that people presented unpublished work”
Scientist, Biogen

“Cutting edge, informative and interactive”
Ultragenyx

“It was a detailed and enlightening summit on neurodevelopment disorders”
Genentech

“Thrilled to see a focus on Neurodevelopmental therapeutic development”
STXBP1 Foundation

“High quality and cutting-edge content for application in the coming year in neurodevelopmental drug development”
To Cure a Rose Foundation

“I learned a great deal ranging from the preclinical to the clinical work for neurodevelopmental disorders, with great detail on all stages”
Tufts University

“The focus on endpoint and biomarker development was constructive, it’s helpful to hear how other organisations are approaching this”
Ultragenyx

“The Neurodevelopmental Drug Development Summit was a fantastic opportunity to share learnings among an outstanding group of academic, industry and patient advocacy experts working toward a common goal of transformative therapies for neurodevelopmental disorders”
Director, Biogen