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Frontiers in Rare Diseases: 2020 Innovators in Science Award Symposium

WEBINAR

Only

Frontiers in Rare Diseases: 2020 Innovators in Science Award Symposium

Friday, October 2, 2020, 9:00 AM - 1:45 PM EDT

WEBINAR

Presented By

Takeda Pharmaceutical Company Limited

The New York Academy of Sciences

 

The Innovators in Science Award was established in 2016 as a partnership between the New York Academy of Sciences and Takeda Pharmaceuticals to recognize creative and impactful research being conducted around the world. This symposium will showcase the achievements of the 2020 Innovators in Science Award Winners in Rare Diseases, Dr. Adrian Krainer and Dr. Jeong Ho Lee, and feature presentations from world leaders in rare diseases research.​

Please join us virtually for a unique opportunity to engage with leading researchers, clinicians, and prominent industry stakeholders from around the world about the latest breakthroughs in the scientific understanding and clinical treatment of genetic, nervous system, metabolic, autoimmune, and cardiovascular rare diseases.

The Winners of the 2020 Innovators in Science Award will be honored at the 2020 Innovators in Science Award Ceremony, to be held on Thursday, October 1, 2020, 7:30-8:30pm. Please go to nyas.org/InnovatorsCeremony20 for more information and to register for this free event.

For information on poster presentations and networking, please visit our Event Guide


Award Program

The Innovators in Science Award recognizes a promising Early-Career Scientist and an outstanding Senior Scientist for their contributions to science in the therapeutic areas of Neuroscience, Regenerative Medicine, Rare Diseases, Gastroenterology, and Oncology. Each Award Winner receives a prize of US$200,000 intended to support their commitment to innovative research.

Academic, government, and non-profit research institutions from around the globe, as well as the Award’s own distinguished Scientific Advisory Council, nominated candidates who are conducting groundbreaking research in the field of rare diseases.


This is a free event, but advance registration is required.

Registration

Individual
$0

Winners

Adrian Krainer, PhD
Adrian Krainer, PhD

Cold Spring Harbor Laboratory

Jeong Ho Lee, MD, PhD
Jeong Ho Lee, MD, PhD

Korea Advanced Institute of Science and Technology

Symposium Speakers

Annemieke Aartsma-Rus, PhD
Annemieke Aartsma-Rus, PhD

Leiden University Medical Center

Norman Barton, MD, PhD
Norman Barton, MD, PhD

Takeda Pharmaceuticals

Don Cleveland, MD, PhD
Don Cleveland, MD, PhD

UC San Diego

 Daniel Curran, MD
Daniel Curran, MD

Takeda Pharmaceuticals

Steve Hitchcock, PhD
Steve Hitchcock, PhD

Takeda Pharmaceuticals

Huda Zoghbi, MD
Huda Zoghbi, MD

Baylor College of Medicine

Brad Margus
Brad Margus

Cerevance

David Fajgenbaum, MD
David Fajgenbaum, MD

University of Pennsylvania

Graciana Diez-Roux, PhD
Graciana Diez-Roux, PhD

Telethon Institute of Genetics and Medicine

Sarah Sheikh, MRCP
Sarah Sheikh, MRCP

Takeda Pharmaceuticals

Anne Heatherington, PhD
Anne Heatherington, PhD

Takeda Pharmaceuticals

Organizers

Andrew Plump, MD, PhD
Andrew Plump, MD, PhD

Takeda Pharmaceutical Company Limited

Mohannad Shawer, PhD
Mohannad Shawer, PhD

Takeda Pharmaceutical Company Limited

Amanda Sadacca, PhD
Amanda Sadacca, PhD

New York Academy of Sciences

Brittany Aguilar, PhD
Brittany Aguilar, PhD

New York Academy of Sciences

Friday

October 02, 2020

9:00 AM

Welcome Remarks & Introduction to the Innovators in Science Award

Session 1: Presentation of Early-Career Scientist Award Winner

9:15 AM

Presentation of Early-Career Scientist Award Winner

Speaker

Daniel Curran, MD
Takeda Pharmaceuticals
9:20 AM

Deciphering Brain Somatic Mutations in Neurological Disorders

Speaker

Jeong Ho Lee, MD, PhD
Korea Advanced Institute of Science and Technology
9:40 AM

Early-Career Scientist Winner Q&A

Speaker

Jeong Ho Lee, MD, PhD
Korea Advanced Institute of Science and Technology

Session 2: Presentation of Senior Scientist Award Winner

9:50 AM

Presentation of Senior Scientist Award Winner

Speaker

Steve Hitchcock, PhD
Takeda Pharmaceuticals
9:55 AM

Antisense Modulation of RNA Processing for Rare-Disease Therapy

Speaker

Adrian Krainer, PhD
Cold Spring Harbor Laboratory
10:15 AM

Senior Scientist Winner Q&A

Speaker

Adrian Krainer, PhD
Cold Spring Harbor Laboratory
10:25 AM

Break

Session 3: Novel Approaches to Treating Rare Diseases

10:30 AM

Session Introduction

Speaker

Sarah Sheikh, MRCP
Takeda Pharmaceuticals
10:35 AM

Exon Skipping Therapy for Duchenne and Other Rare Diseases

Speaker

Annemieke Aartsma-Rus, PhD
Leiden University Medical Center
10:55 AM

Designer DNA Drug Therapy for Neurodegenerative Disease

Speaker

Don Cleveland, MD, PhD
University of California, San Diego
11:15 AM

Session 3 Q&A

11:25 AM

Break

Session 4: Keynote Presentation

11:30 AM

Session/Keynote Introduction

Speaker

Norman Barton, MD, PhD
Takeda Pharmaceuticals
11:35 AM

Genetic and Therapeutic Approaches to Tackle Neurodevelopmental Disorders

Speaker

Huda Zoghbi, MD
Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital
12:05 PM

Session 4 Q&A

Session 5: Lunchtime Panel Discussion

12:15 PM

The Challenges of Research in Rare Diseases

Speakers

Brad Margus, Moderator
Co-founder and CEO, Cerevance
Graciana Diez-Roux, PhD
CSO, Telethon Institute of Genetics and Medicine
David Fajgenbaum, MD
University of Pennsylvania
Anne Heatherington, PhD
Takeda Pharmaceuticals
12:55 PM

Closing Remarks

Speaker

Brooke Grindlinger, PhD
New York Academy of Sciences
1:00 PM

Symposium Adjourns

2020 Innovators in Science Award Poster Session

1:00 PM

Poster Session Begins

1:45 PM

Poster Session Adjourns

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