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Rare Disease Symposium

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Genentech Hall (Byers Auditorium)

UCSF Mission Bay

600 16th Street

San Francisco, ca 94158

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Negotiating the Challenges of Therapeutics Discovery for Rare Diseases


The goals of the Rare Diseases Symposium at UCSF are:

  • To create a community of academic scientists, business leaders, regulatory experts and patient advocacy groups with a common interest to tackle rare diseases

  • To examine the key issues in development of treatments for rare diseases

  • To draw attention to and encourage research in origins and treatments of rare diseases

  • To showcase ground-breaking research in rare diseases at UCSF

  • To foster collaborations between academia, industry, and non-profit organizations


AGENDA

8:30-9:00 Registration/check-in


9:00-9:10

Welcome Cathy Tralau-Stewart, PhD, FRSC, Interim Director, UCSF Catalyst Program


9:10-9:40

Keynote: The Golden Age of Rare Disease Treatment Development

Emil Kakkis, MD, PhD, CEO and President, Ultragenyx


9:40-10:30

Sponsors Panel: How does Rare Disease Research fit into Company strategies/ approaches?

Moderator : Scott Clarke, Senior Vice President, BioMarin Pharmaceutical

  • Angelika Jahreis, MD,PhD, Group Medical Director, Roche/Genentech
  • Neil Kumar, PhD, CEO and Founder, BridgeBio
  • Gregory Fond, Director, Strategy & Business Development, Sanofi/Genzyme
  • Natalie Holles, SVP and COO, Audentes Theurapeutics


10:30-11:00 Break/Networking


11:00-11:20

Presentation: Orphan drug discovery using whole animal disease models

Ethan Perlstein, PhD, CEO, Perlara, PBC


11:20-12:10

Panel: Role of Disease Foundations

Moderator : Hank Mansbach, MD, Vice President, Ultragenyx

  • Mark Dant, President and CEO, MPS Society; Founder, Ryan Foundation
  • Ben Lenail, Founding Officer, ALD Connect
  • Kim Mooney, Associate Director for Patient Advocacy, Ultragenyx
  • Amer Haider, Founder, Growing Stronger
  • Dean Suhr, President and Founder, MLD Foundation


12:10-1:15 Lunch


1:15-1:45

Transformative Cellular Technologies in Rare Disease Research

Wendell Lim, PhD, Professor of Cellular & Molecular Pharmacology, UCSF


1:45-2:45

Development case-studies

  • Nadav Ahituv, PhD, Professor, School of Pharmacy, UCSF -- "CRISPRa as a Theraputic Haploinsufficiency"
  • Ophir Klein, MD, PhD, Professor, Depts of Orofacial Sciences & Pediatrics, UCSF -- "Medical Approaches to Structural Birth Defects: The First Step on a Long Road"
  • Joe Payne, Founder, President and CEO, Arcturus Therapeutics -- "A New Era of Nucleic Acid Therapeutics to Treat Orphan Diseases"


2:45-3:10 Break/Networking


3:10-3:30

Challenges in Rare Disease Development: Navigating the Regulatory Environment

Jeffrey Siegel, MD, Senior Group Head, Rheumatology and Rare Diseases, Genentech/Roche


3:30 -4:15

Panel: Challenges of rare disease development & clinical research in small patient populations

Moderator : Jeffrey Siegel, MD, Senior Group Head, Rheumatology and Rare Diseases, Genentech/Roche

  • Eric Green, MD, PhD, Head of Translational Research, Myokardia

  • Robert Nussbaum, MD, CMO, Invitae

  • Matthew Wilsey, President of Grace Science Foundation


4:15-5:00

Patient & Parent Perspectives

  • Karen Park, Parent, Principal, Bernstein Global Wealth Management
  • Mark Dant, Parent, President and CEO, MPS Society; Founder, Ryan Foundation


5:00-6:30pm Networking Reception

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Date and Time

Location

Genentech Hall (Byers Auditorium)

UCSF Mission Bay

600 16th Street

San Francisco, ca 94158

View Map

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