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Lunch & Learn: The Pathway to an Approval

The last session provided us with the routes of administration for different gene therapies both approved and in clinical trials. During this free session, we'll dive further into the clinical pathway for getting a therapy through a clinical trial and into the approval phase, and we'll hear the perspective of a patient advocate on cost and access once a therapy is approved. Watch our July session for a better understanding on the preclinical processes prior to this session. 


12 – 12:05 p.m. 
Betsy Bogard, MS, Head of Program and Alliance Management, Ensoma 

Pathway to an Approval 
12:05 – 12:25 p.m. 
Speaker: Emily McGinnis, MPH, Chief Patient Officer and Head of Government Affairs at Taysha Gene Therapies 

Emily McGinnis will take us through the clinical phases of a trial, and what is needed for FDA approval. We will also explore the the differences in these processes depending on what country you live in, and strategies for streamlining the approval process around the world.  

The Patient Advocate Perspective: Cost and Access of Approved Therapies 
12:25 – 12:40 p.m. 
Speaker: Kenneth Hobby, President of Cure SMA  

Kenneth Hobby will discuss their experience at Cure SMA when a gene therapy was approved for spinal muscular atrophy. They will also give us an overview on current state of access and cost for approved therapies.  

Moderated Q&A  
12:40 p.m. – 1:00 p.m.  

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Last Updated: 01/30/2024

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