#r&d — iPharmag

Illustration of a brain outline with a short RNA strand crossing a protective barrier

R&D

An RNA Drug Injected Under the Skin Cut Brain Tau by Up to 85% in an Early Alzheimer’s Trial

Arrowhead’s ARO-MAPT reached the brain from a simple injection — a first for an RNAi medicine — and lowered tau, the protein behind Alzheimer’s tangles, in healthy volunteers. Whether that helps patients is still unknown.

3 days ago · 4 min read

R&D

BioNTech’s mRNA Cancer Immunotherapy Misses Its First Goal in a Key Head and Neck Cancer Trial

Adding BNT113 to Keytruda did not delay the cancer’s progression at an interim check. The trial continues to see whether patients live longer — but the result is another reminder of how hard it is to improve on Keytruda.

Oct 13, 2026 · 4 min read

Illustration of a DNA strand being edited inside a liver cell with fat particles in a blood vessel

R&D

A One-Time CRISPR Treatment Cut “Bad” Cholesterol in Half — and It Held for a Year

CRISPR Therapeutics’ CTX310 switches off the ANGPTL3 gene in the liver. One-year follow-up in a small first-in-human trial shows LDL and triglycerides stayed down with no serious treatment-related side effects.

Sep 28, 2026 · 4 min read