Connecticut Rare Disease Forum

Join us for industry-led discussions on innovation in rare disease diagnostics and treatment, and how we can accelerate patient access to these groundbreaking advancements.

• Registration: 9:00am – 9:30am
• Program: 9:30am – 12:00pm

AGENDA:

Opening Comments & Keynote

• Introduction to Keynote: Craig Miller, PhD, Senior Principal Scientist, Research Project Leader, Boehringer Ingelheim
• Keynote Speaker: Tim Yu, MD, PhD, Attending Physician, Division of Genetics and Genomics, Boston Children’s Hospital; Assistant Professor, Harvard Medical School

Panel One: Innovation in Therapeutic Development – from Genetic Science to Patient Impact

• Moderator: Toby Bannon, JD, Partner, Wiggin & Danna
• Mike Castagna, PharmD, MBA, Chief Executive Officer, MannKind
• James Gregory, PhD, Director, Neuroscience, Arvinas
• Yong-Hui Jiang, MD, PhD, Chief of Medical Genetics at Yale School of Medicine and Yale New Haven Hospital, Director of Yale NORD Center of Excellence
• Jeanne Magram, PhD, Chief Scientific Officer, Quarry Thera
• Jonathan Whitton, PhD, Vice President, Global Program Head of Genetic Medicines, Regeneron

Panel Two: Policy and Regulation: Shaping the Future of Research and Treatment

• Moderator: Cat Lutz, PhD, MBA, Vice President of The Jackson Laboratory Rare Disease Translation Center
• P.J. Brooks, PhD, Acting Director, Office of Rare Disease Research, NCATS, NIH
• Andrew Steinsapir, MBA, Director of Gene Therapy, Deerfield Discovery; Acting Chief Technology Officer, Apertura Gene Therapy
• Sonia Vallabh, PhD, JD, Director, Prion Therapeutic Science, Broad Institute
• Walter Voegtli, PhD, Executive Director, Discovery Research, Alexion, AstraZeneca Rare Disease

Thank you to our generous sponsors:

Diamond Sponsors:     

Video Sponsor: 

Table Sponsors:                          

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