Cell And Gene Therapy: Still On A Learning Curve

In today's ACT Brief, we examine how AI enables sponsors to pressure-test protocols before enrollment, HHS's multi-initiative effort to accelerate trial design and execution, and FDA approval expanding heart disease therapy to adolescents.

In today's ACT Brief, we examine AI's role as trial design advisor with human leadership, how to govern autonomous agents in regulated operations, and real-world outcomes from switching to oral weight-loss therapy.

In today's ACT Brief, we examine how data drives honest enrollment assumptions, why RWE infrastructure lags ambition, and the disconnect between FDA rulemaking and enforcement on compounded therapies.

In today's ACT Brief, we examine what AI simulations surface in trial design, how RBQM delivers measurable financial returns, and three new FDA-approved treatments across hair loss, movement disorder, and bone disease.

In today's ACT Brief, we examine how AI is surfacing endpoint options beyond traditional design thinking, why sponsor oversight of outsourced work often fails on inspection despite being robust in practice, and a new combination drug for advanced kidney cancer.

In today's ACT Brief, we examine what patient-site relationships need beyond easier technology, how to build defensible rare disease evidence from scratch, and how real-world data and AI improve enrollment screening accuracy.

In today's ACT Brief, we examine why defensible trial data depends on traceability and platform ownership, what CGT site readiness requires as therapy moves to community care, and why data harmonization is AI's primary bottleneck.

In this episode of Beyond Compliance, Otis Johnson, PhD, MPA, founder and principal consultant at Vantix Operations, speaks with Cecilia Xi, PhD, VP of clinical and scientific affairs at Vivalink, about why defensible trial data depends on traceability, platform ownership, and device strategy long after a study ends.