Emily Heffernan of Arcadis explains how facility scale, equipment, containment, and quality testing differ across ex vivo cell therapy, viral vector, and mRNA-LNP manufacturing.

Dr. Paz Vellanki discusses Bayesian methods, challenges in developing rare disease endpoints, and why sponsors should engage FDA early in clinical development.

The FDA grants accelerated approval to dostarlimab for rectal cancer, Lilly reports one-year remission data for pediatric ulcerative colitis, and Johnson & Johnson shares longer-term fistula remission results for Tremfya.

BMS's Dr Nathan Pennell outlines how biomarker testing barriers and trial diversity affect patient access to targeted oncology drugs.

In the phase 3 SHINE-2 trial, half of pediatric patients who responded to mirikizumab induction were in clinical remission at week 52. Lilly plans to file for a pediatric UC label expansion.

Roche will take ex-China development of Hansoh’s two preclinical ADCs and multiple programs against Roche-proposed targets once Hansoh completes global phase 1a trials. The deal includes $400 million upfront and more than $15 billion in potential milestones.

Opus Genetics reports sustained vision gains with gene therapy, the FDA approves Roche’s Enspryng for at-home treatment of thyroid eye disease, and Estuary Biotherapeutics launches with $103 million to develop gene therapies for polycystic kidney disease.

When large pharmaceutical companies deprioritize drug candidates, smaller developers may license or acquire them, but due diligence, deal structure, and trial planning determine whether a program can move forward.