Biotech & Medicine
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FDA Approves Regeneron's Otarmeni as First Gene Therapy for Genetic Hearing Loss, with the Drugmaker Pledging Free Access in the U.S.
Regeneron's one-time intracochlear gene therapy for OTOF-related deafness cleared the FDA 61 days after BLA filing, the first gene therapy product approved under the Commissioner's National Priority Voucher pilot program.
AXS-05 Reaches FDA Decision Deadline With April 30 PDUFA Date, Poised to Become Second-Ever Approved Drug for Alzheimer's Agitation
Axsome Therapeutics' AXS-05 faces its FDA verdict on April 30 for Alzheimer's disease agitation, backed by four Phase 3 trials but complicated by one failed study.
CorTec's Brain Interchange Wins FDA Breakthrough Designation and TAP Acceptance, Becoming the First BCI Cleared for Stroke Motor Rehabilitation
CorTec's implantable Brain Interchange system received FDA Breakthrough Device Designation on April 8 and TAP acceptance on April 23, marking the first BCI worldwide designated for stroke motor rehabilitation.
Intellia Therapeutics to Release World's First In Vivo CRISPR Phase 3 Data on April 27
Intellia Therapeutics will report topline Phase 3 results for lonvoguran ziclumeran in hereditary angioedema on April 27, 2026 — the first Phase 3 readout for any in vivo CRISPR candidate.
Personalized mRNA Vaccine Keeps 87 Percent of Pancreatic Cancer Responders Alive at Six Years, AACR Data Show
Six-year follow-up from a 16-patient Phase 1 trial of autogene cevumeran shows unprecedented survival rates in one of oncology's deadliest cancers, now entering a global Phase 2 trial.
Dana-Farber's CAR-PRISM Trial Achieves 100% MRD-Negativity in High-Risk Smoldering Myeloma, Pushing CAR-T Into Pre-Cancer Territory
A single infusion of Johnson & Johnson's Carvykti rendered all 20 patients in a Dana-Farber phase 2 trial undetectable for residual disease, the first time CAR-T therapy has been tested in a precursor cancer setting.
Mount Sinai Team Identifies Recessive ReNU2 Syndrome as the Most Prevalent Recessive Neurodevelopmental Disorder Ever Found
Biallelic RNU2-2 variants cause a newly characterized neurodevelopmental disorder that researchers estimate accounts for roughly 10 percent of recessive NDD cases with a known genetic cause.
FDA Clears Denali's AVLAYAH, the First Brain-Penetrating Enzyme Therapy, Cracking Hunter Syndrome's Neurologic Frontier After Two Decades
Denali's tividenofusp alfa becomes the first FDA-approved biologic engineered to cross the blood-brain barrier, breaking a long rare-disease rejection streak and validating a transport-vehicle platform that could unlock CNS access for many more drugs.
Xenon's Azetukalner Cuts Seizure Frequency by 53% in Phase 3 Trial, Poised for First-in-Class FDA Submission
Phase 3 data presented at AAN 2026 show azetukalner nearly halved seizure frequency in treatment-resistant focal epilepsy, with 38% of long-term patients achieving over a year of seizure freedom.
Northwestern Engineers Print Artificial Neurons That Talk to Living Brain Cells
Researchers at Northwestern University have used aerosol jet printing to create flexible artificial neurons that generate biologically realistic signals and directly activate living neural tissue.
Kent MenB Outbreak Triggers Second-Dose Push for Nearly 12,000 Exposed People
UK health officials are widening the MenB response in Kent by offering second doses to people already given a first shot, after an outbreak that has left two people dead and 21 confirmed cases.
Philips Wins FDA Clearance for Verida, the First Detector-Based Spectral CT With AI Baked Into the Reconstruction Chain
The Dutch medtech giant cleared a 510(k) for a spectral CT that reconstructs 145 images per second using a deep learning engine, bringing always-on spectral imaging to US hospitals for the first time.