Innovation in Rare Disease: Making Progress with Cell & Gene Therapies

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Jump to the Agenda for Each Day: Day One | Day Two | Day Three

 

This Rare Disease Day, Syneos Health continues the drive to raise awareness of rare diseases and their impact on patients’ lives – and to shine a light on the opportunities for cell and gene therapies in treating these disorders.

Join us for an inspiring program that brings together – over three days – patients and their families, legislators, industry experts, advocates and sponsors for presentations and panel discussions on education initiatives, recent advancements and the future promise of cell and gene medicine, and current patient experiences with these therapies.

 

Register for the Event

 

Day One | February 23rd, 2021 | 9am - 10:15am ET

All times are listed in ET

 

9:00am | Welcome & Opening Remarks

Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health

Tara J. Britt, Founder and President, Rare Disease Innovations Institute, Associate Chair, North Carolina Rare Disease Advisory Council 

 

9:10am | Keynote Address 

Congressman G. K. Butterfield, North Carolina’s First Congressional District and Congressional Co-Chair, Rare Disease Caucus.

 

9:20am | Overview of Cell & Gene Therapies

Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health

 

9:35am | Patient Perspectives & Discussion 

Hear from industry and family advocates from the Spinal Muscular Atrophy (SMA) community on their experience with gene therapies used to treat the disease.

Introduction by Meredith Huml, Communications Assistant, Disability Advocates of North Carolina

The Pantier Family, SMA Advocates

 

9:55am | Closing Remarks, Wrap up Day One & Day Two Introduction

Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health

Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council 

 

10:00 - 10:15am | Q & A

Open for Q & A and networking with organizers and panelists

 


Day Two | February 24th, 2021 | 9am - 10:15am ET

 

9:00am | Welcome & Opening Remarks

Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health

Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council 

 

9:05am | STRiDE State Rare Disease Education Forums

Morrie Ruffin, Executive Director, ARM Foundation

Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council

 

9:20am | Patient Perspectives & Discussion 

Hear from patients and patient advocates about their experience with Cell and Gene Medicine and rare disease.

Introduction by Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health

Patient Panelists:

Justina Williams (Sickle Cell)

Jenny Klein (MPS)

Kevin Schaefer (SMA)

Clayton Goldin (Mitochondrial Deficiency)

Charlene Cowell (Hemophilia)

Moderators:

Parvathy Krishnan, MS, RD, CNSC, LDN, Director, Patient Engagement, Rare Disease Innovations Institute

Keri McDonough, Lead, Advocacy and Patient Relations, Syneos Health

 

9:45am | Closing Remarks, Wrap up Day Two & Day Three Introduction

Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health

Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health

Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council 

 

9:50am | Q & A   

Open for Q & A and networking with organizers and panelists

 


Day Three | February 25th, 2021 | 9am - 10:15am ET

 

9:00am | Welcome & Opening Remarks

Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health

 

9:05am | Disease Specific Advancements in Cell and Gene Medicine

Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health

Dr. Maryna Kolochavina, Executive Director, Syneos One, Syneos Health

 

9:20am | Panel Discussion

Hear from experts in the field of retinitis pigmentosa (RP) on the current status and future of Cell and Gene Medicine.  

 

Dr. Cadmus Rich, Chief Medical Officer, Aura Biosciences

Parvathy Krishnan, MS, RD, CNSC, LDN, Director, Patient Engagement, Rare Disease Innovations Institute

Dr. Van K. Duesterberg, patient, Director of Automation Engineering, and Founder of Patient Led

Dr. Todd Durham, Vice President, Clinical & Outcomes Research, Foundation for Fighting Blindness

 

9:50am | Closing Remarks for the Workshop

Dr. Nick Kenny, Chief Scientific Officer, Syneos Health

Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council 

 

10:00 - 10:15am | Q & A

Open for Q & A and networking with organizers and panelists

 

Register for the Event

 

 

When 
Tue, Feb 23, 09:00 am to Thu, Feb 25, 10:15 am
Additional Details 

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