Two First-Ever Rare Disease Approvals Landed the Same Day. Both Were Measured by Proxy.
Pasatru’s flare benefit is clinician-assessed only. GENGLYCOS is indicated to reduce cornstarch intake. Two firsts, both measured a step from the patient.
Cell & Gene Tx
A UC Irvine Team Names Five Mitochondrial Drug Classes for CAR-T Neurotoxicity and Says Nobody Is Testing Any of Them
Five mitochondrial drug classes named for CAR-T neurotoxicity, and the authors say none is under formal investigation. Every one is generic or preclinical.
Everyone’s New Pharma Lane Runs Through Heathrow
Two carriers built new pharma lanes in five weeks. Both end at Heathrow, an airport that lost a day to one transformer fire in March 2025.
MHRA Puts Microbiome Medicines Inside the Existing Framework While the EU Builds a Separate One
No bespoke regime is coming. Microbiome medicines meet the standard biological bar in the UK, while the EU builds a separate SoHO framework.
Replimune Won Accelerated Approval on 91 Evaluable Patients and a 24% Response Rate
CBER took a 24% response in 91 evaluable patients, one day after the same committee voted nine to three against Capricor. Measurement, not size.
An FDA Panel Voted 9-3 Against Capricor on Cardiomyopathy and Never Voted on the Endpoint That Passed
The ballot was the outcome. A drug’s strongest dataset sitting off the voting question is not a footnote, it is the whole result.
J&J Paid $785 Million for a Look at Something That Has Never Been in a Human
J&J committed $785 million including a $465 million equity stake, plus an option to acquire Sail Biomedicines for $2.58 billion, on a preclinical in vivo CAR-T platform.
Ikarovec Eyes $1B Deal to Move Eye Gene Therapy In-Office
UK biotech Ikarovec teams with VectorBuilder on eye gene delivery.
Biogen’s High-Dose Nusinersen Wins CHMP Backing for Enhanced SMA Treatment
Biogen has earned a positive CHMP opinion for its high-dose nusinersen regimen, supported by strong DEVOTE study results showing major motor improvements and reduced mortality risk in SMA patients.
NextGen Gene Editing Breakthroughs Redefine Personalized Care
The New Frontier of Gene Editing: From Precision Tools to Personalized Therapies A new generation of programmable gene editing tools is pushing biotechnology toward an era of truly personalized medicine one in which therapies are tailored not only to specific diseases but to individual genetic profiles. As highlighted in a recent npj Biomedical Innovations feature … Read more