Advanced Therapies Clinical Development

 

Advanced Therapies Clinical Development, Wednesday 24 April 2024

Adam Bloomfield, M.D., FAAP
Advanced Therapies Clinical Development
11:24

Chair's Remarks

Adam Bloomfield, M.D., FAAP, Vice President, Medical Affairs, Rare Diseases and Pediatrics, Premier Research
Adam Bloomfield, M.D., FAAP
Advanced Therapies Clinical Development
11:25

Unlocking Insights: How decentralized clinical trials and advanced therapies are revolutionizing pediatric rare disease research

  • This session will explore solutions to challenges in pediatric rare disease research, specifically in consideration of decentralized clinical trials (DCT) and cell and gene therapy development. You will learn:
  • The complexities of executing cell and gene therapy programs in pediatric populations
  • Key considerations for instituting a DCT approach for cell and gene therapy programs in a pediatric population
  • Strategies for seamless operationalization of DCTs
Adam Bloomfield, M.D., FAAP, Vice President, Medical Affairs, Rare Diseases and Pediatrics, Premier Research
Renata Gallagher, MD, PhD
Advanced Therapies Clinical Development
11:45

An honest discussion: The challenges of genotype interpretation and phenotype variation in rare disease

Renata Gallagher, MD, PhD, Medical Director, Parexel
Panel discussion
Advanced Therapies Clinical Development
12:05

Sustainability of N-of-1

Moderator: Dave Pearce, Chair, International Rare Diseases Research Consortium (IRDiRC)
P.J. BROOKS, Deputy Director of the NCATS Division of Rare Diseases Research Innovation, National Institutes of Health
Sarah Glass, Chief Operating Officer, n-Lorem Foundation
Larissa Lapteva, Associate Director, FDA
Linda Dell
Advanced Therapies Clinical Development
12:45

From science to solution: Navigating the clinical development journey

Linda Dell, VP, Program Leader, ReAlta Life Sciences, Inc.
Advanced Therapies Clinical Development
13:05

Optimizing patient recruitment by leveraging existing online communities: Success stories

Ethan Ash, Senior VP of Business Development, BioNews
Marcella Debidda, President, BioNews Clinical, BioNews Clinical
Adam Bloomfield, M.D., FAAP
Advanced Therapies Clinical Development
14:59

Chair's Remarks

Adam Bloomfield, M.D., FAAP, Vice President, Medical Affairs, Rare Diseases and Pediatrics, Premier Research
P.J. BROOKS
Advanced Therapies Clinical Development
15:00

Maximizing therapeutic impact: Exploring the potential of basket trials for concurrent treatment strategies

P.J. BROOKS, Deputy Director of the NCATS Division of Rare Diseases Research Innovation, National Institutes of Health
Panel discussion
Advanced Therapies Clinical Development
15:20

A new legislative era in the EU: How an integrated approach can help smaller pharma and biotech achieve a sustainable model to deliver equity of access in short timeframes

Ruth Rostron, Director of Program Management, Uniphar
Maurice Leonard, Medical Director, Uniphar
Laurence Mignon
Advanced Therapies Clinical Development
15:40

Treating the nano-rare: Redefining clinical management, one patient at a time

Laurence Mignon, Executive Director, Clinical Development, n-Lorem Foundation
Panel discussion
Advanced Therapies Clinical Development
16:00

Conducting clinical studies in rare diseases

Moderator: Marshall Summar, Chief Executive Officer, Uncommon Cures
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
Chuck Bucklar, Principal, Chair 5 Consulting, LLC
Andrew Lo, Charles E. and Susan T. Harris Professor, Massachusetts Institute of Technology (MIT)
last published: 24/Apr/24 15:45
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Advanced Therapies Clinical Development, Thursday 25 April 2024

last published: 24/Apr/24 15:45
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