SBS Day 2 - Panel IV-b Considerations for Clinical Trial Design Optimization in Neonates and T1D

Panel IV-b Considerations for Clinical Trial Design Optimization in Neonates and T1D
Moderators:
Kanwaljit Singh (C-Path), Joe Hedrick (C-Path),
Panelists
: A.J. Allen (I-ACT forChildren), Ralph Bax (EMA), John Concato (FDA), An Massaro (FDA), Victoria Niklas (Oak Hill Bio),Betsy Pilon (Hope for HIE), John Sharretts (FDA)
Closing Remarks
Klaus Romero, Kanwaljit Singh, and Joseph Hedrick (C-Path)
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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