Engage with foundations, trusts and patient advocates to appreciate the power of industry and patient Group collaboration

Boston, MA

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SPEAKER LIST & PRE-CONFERENCE WORKSHOP DISCUSSIONS

TESTIMONIALS FROM LAST YEAR


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

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

2022 Key Speakers


 

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

Developing New Outcome Measures, Functional Biomarkers & Clinical Research Regulatory Frameworks for Disease-Modifying Therapeutics Across Neurodevelopmental & Rare, Genetic CNS Disorders

With further clinical data emerging, drug development for rare CNS and neurodevelopmental disorders is advancing from hypotheticals to real-life clinical results, outcomes, and functional biomarkers; catapulting this critical field forwards to change patients’ lives.

The 2nd Rare CNS & Neurodevelopmental Drug Development Summit is the definitive industry-dedicated forum for large pharma, biotech, academia, and patient advocacy groups, paving the way towards symptomatic and successful disease modifying treatments.

Tightly focused on industry progress, join stakeholders exploring ASOs, gene therapies and small molecules across autistic syndromes and seizure phenotypes in at this unique drug sponsor conference.

Don’t miss out on the industry’s definitive opportunity to collaborate with patient groups and gain regulatory clarity on clinical trial design, endpoints and delivery and toxicity concerns as well as deep diving into functional & molecular biomarkers, emerging therapeutic targets and translational biology.

If you are working towards treatments for rare CNS and neurodevelopmental disorders, including (but not limited to) Angelman Syndrome, Rett syndrome, Fragile X, Dravet Syndrome & CDKL5 Syndrome or discovering new frontiers in anti-epileptic therapeutics, this is the platform you need.

What’s New In 2022?

Brand new industry insights include:

Discover emerging technological applications from targeted gene regulation using engineered zinc finger proteins, tRNA and ASOs for endogenous gene activation from Sangamo, Tevard Biosciences and CAMP4 Therapeutics

Explore advances in outcome measures with Roche, Rett Syndrome Research Trust and LouLou Foundation, from digital outcome measures, and application across diseases

Dive into biomarkers with Biogen, Ultragenyx & Anavex Life Science, exploring molecular, quantitative and functional such as sleep, EEG & behaviour

Engage with foundations, trusts and patient advocates to appreciate the power of industry and patient Group collaboration

Expert Speakers Include:

“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

2022 Partners:

Lead Partner

Exhibition Partner

Other Events in the CNS Series: