SBS Day 2 - Session IV: Clinical Trial Design Considerations

Session IV: Clinical Trial Design Considerations
Panel IV-a Considerations for Clinical Trial Design Optimization in Alpha-1 Antitrypsin Deficiencyand Lysosomal Diseases
Moderators
: Gina Smith, Krista Casazza (C-Path)
Panelists
: Jon Hagstrom (Alpha-1 Foundation),Christine í Dali (Zevra), Gavin Imperato (FDA), Jonathan Jacoby (SOAR-NPC), Ed Marins (Takeda),Jeff Siegel (FDA), Yulia Yasinskaya (FDA)
This Critical Path Institute summit featured presentations and panel discussions with C-Path collaborators from industry, academia, global regulatory agencies, nurses, parents and patients. The workshop:
- Explored the unmet needs and potential solutions relevant to specific aspects of type-1 diabetes, alpha-1 antitrypsin deficiency, lysosomal diseases, and neonatal indications.
- Demonstrated the critical role data sharing plays in enhancing drug development for vulnerable patient populations with conditions that require interventions early in life.
- Reviewed the current landscape of regenerative therapies, identifying regulatory opportunities that could expedite the development of such groundbreaking products.
- Please check back in for recordings from our sessions, to be posted below.
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