Welcome to the Inaugural Neurodevelopmental Drug Development Summit

Advancing Symptomatic & Disease Modifying Treatments Across Neurodevelopmental Disorders

As investment in rare neurodevelopmental disorders is surging, there has never been a better time to unite all the key stakeholders across this fast-evolving community. 

The inaugural 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.

Developing new outcome measures that measure incremental change and target the neurodevelopmental disease itself, driving design innovation across disease-specific clinical trials, and capitalizing on the advances in new therapeutic modalities to drastically change the lives of patients in need.

With increased understanding of genetic mechanisms sharpening our thoughts on targets and disease modifying approaches, we are now closer than ever to discovering treatments for the highest unmet need across neurodevelopmental disorders.

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

The Neurodevelopmental Drug Development Summit Will Provide a Platform for Industry Thought-Leaders to:

  • Discover how STALICLA, Autifony Therapeutics & Anavex are leveraging precision medicine to treat rare neurodevelopmental diseases
  • Push the development of translational and quantitative biomarkers to strengthen sensitivity and specificity of outcomes measures with Biogen, GeneTx, Harvard Medical School & Yale
  • Dive into the nuances of specific therapeutic modalities including small molecules, gene therapy, antisense oligonucleotides, gene replacement, stem cell therapy and hybrid strategies to discuss longevity of approaches and what is a good candidate for a gene altering therapy or a disease modifying therapy with PJ Brooks, Steven Gray & Barbara Bailus
  • Investigate symptomatic and disease modifying treatments and how our increased understanding of genetic mechanisms has sharpened our thoughts on targets and disease modifying approaches with Ultragenyx, Biogen, Randy Carpenter , Ovid Therapeutics & Kaerus Biosciences

2021 Expert Speaker Faculty

Allyson Berent

Chief Scientific Officer, FAST

Chief Operating Officer, GeneTx Biotherapeutics

Brian Ranes

Principal Investigator, Imaging Core

Amicus Therapeutics

Elizabeth M. Berry-Kravis

Professor, Co-director, Molecular Diagnostics Section of the Genetic Laboratory | Director Fragile X Clinic & Research Program; Co- Director

Rush University Medical Center | Angelman Syndrome Clinic & Research Program

Samir Koirala

Director of Research, Genetic & Neurodevelopmental Disorders Unit


Audrey Thurm

Director, Neurodevelopmental & Behavioral Phenotyping Service, Office of the Clinical Director

National Institute of Mental Health, NIH

Charlene Son Rigby

CEO | President & Founding Board Member

RARE-X | STXBP1 Foundation

Chrisopher U Missling

President & CEO

Anavex Life Sciences

Jeremy Levin

CEO & Chairman

Ovid Therapeutics

Yael Weiss

Vice President Business Development


Charles Large

Chief Executive Officer

Autifony Therapeutics

Greg Dillon

Senior Scientist


Kimberly Parkerson

Senior Medical Director

Stoke Therapeutics

Massimiliano Bianchi

Founder, President & CEO

Ulysses Neuroscience

Randall Carpenter

Co-Founder | CMO

Allos Pharma | Rett Syndrome Research Trust

Companies in Attendance:

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bridge therapeutics
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jaguar gene therapy
ovid therapeutics
shape therapeutics
vyant bio

2021 Partners

Other Events in the CNS Series: