Innovations in Single-Use Bioprocessing Components

Inhaled EXO-CD24 kept 85% of patients with ARDS free of respiratory failure at day 7 vs 33.3% with placebo in a 90-patient phase 2b trial.

Nuravax will test AV-1959R in a 160-adult phase 2 trial to see whether the amyloid vaccine can prevent Alzheimer disease before symptoms begin.

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.

Guselkumab (Tremfya) sustained perianal fistula healing through 1 year in Crohn disease, with remission climbing to 35% in one FUZION dosing arm.

Interim phase 1/2 data presented at Retina in Rio 2026 showed sustained visual improvements with Opus Genetics’ AAV8 gene therapy OPGx-LCA5 and improved retinal sensitivity with OPGx-BEST1 in inherited retinal diseases.

Nathan Edwards of Arya Consulting moderates a discussion with Elisabeth Gardiner of Tevard Biosciences and Donny Xu of the RTW Institute about rare disease treatment development and navigating the FDA’s Rare Pediatric Disease Priority Review Voucher Program.

Mirikizumab (Omvoh) achieved comprehensive disease control in 27.7% of UC patients and deep remission in 20.4% with Crohn disease at 1 year.

As ArsenalBio, TScan, Bristol Myers Squibb, and others abandon ex vivo programs for in vivo engineering, early clinical data and platform differences point to both the promise and the unresolved safety, manufacturing, and regulatory questions of generating CAR-T cells inside the body.