Cell, Gene, and RNA Therapies are shifting the aim of drug development from managing symptoms to delivering durable, potentially curative therapies. But moving a construct from a promising preclinical milestone to a viable commercial asset—whether an ex vivo cell therapy, an in vivo viral vector, or an RNA...
Learn how evolving FDA programs, modern trial designs, and patient-centered practices are reshaping rare disease development.
The Cell and Gene Therapy (CGT) sector continues to progress toward a demonstrably execution-centric development paradigm, in which asset valuation is increasingly influenced by the capacity to integrate scientific innovation with operational precision, manufacturing robustness, and evidence strategies that withstand...
The cell and gene therapy (CGT) landscape has entered a period of unprecedented growth. Dozens of CGT products have already been approved globally, and more than 4,300 therapies—from AAV gene therapies to RNA-based and gene-modified cell therapies—are moving through development.i...
This webinar will bring together perspectives from a sponsor, CRO, clinical site, and institutional biosafety committee (IBC) expert to explore strategies for expanding and optimizing CGT trial sites.
In the rapidly evolving field of adeno-associated virus (AAV) gene therapy, patient advocacy groups (PAGs) have emerged as critical partners driving scientific, operational, and regulatory progress. Their influence extends far beyond awareness and fundraising—they help shape trial design, regulatory engagement,...