SBS Day 2 - INC current perspective: Conducting gene therapy clinical trials

INC current perspective: Conducting gene therapy clinical trials in neonatal and early pediatric age, including newborn screening
Jim Wilson (Penn)
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.
Contact [e-mail] if you have any questions.
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