01.08.26 -- Navigating Conflicting Raw Material Preferences With Your CDMO

Discover how one family's race against time led to the creation of a groundbreaking gene therapy for their daughter, just 14 months after her ultra-rare disease diagnosis.

A highly sensitive ddPCR assay targeting the VSV-g sequence allows for absolute quantitation and robust monitoring without product-specific qualification.

RNA sequence and guide modifications, delivery platform design, and dose optimization all improve gene editing outcomes, which offer a clearer path toward safer, more potent, and scalable mRNA-based therapeutics.

Scaling advanced therapies requires navigating extreme logistics and regulatory hurdles. Learn why moving to a flexible, outsourced model is essential for freeing capital and mitigating global risk.

Here, we highlight real-world applications of AI in gene therapy, providing case studies and addressing the challenges and prerequisites for successful implementation.

Managing timelines to IND is the top challenge in cell therapy, impacting funding, patent value, and patient access amid competitive, capital-constrained development environments.

Early, phase-appropriate CMC planning is critical for advanced therapy success. Proactively managing regulatory risks and applying Quality by Design principles secures a clear path to market.

Gain practical strategies and regulatory insight on viral clearance studies, including virus selection, performance benchmarks, case studies, and submission-ready data for biologic products.

Review the development of a high-strength synthetic gene promoter that enhances titers while maintaining exceptional product quality and expression stability, to support more efficient biomanufacturing.