SBS Day 2 - Session III-b: Disease Prevention in Lysosomal Diseases

Session III-b: Disease Prevention in Lysosomal Diseases
Drug development in Cell and Gene Therapy
Industry Perspective:
Tom Miller (Bayer)
Panel III-b Moderators
: Kanwaljit Singh, Krista Casazza (C-Path)
Panelists
: Elizabeth Hart (FDA), GavinImperato (FDA), Tom Miller (Bayer), Galina Nesterova (Thermo Fisher), Laurie Turner (NationalNiemann-Pick Disease Foundation), 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.
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