Biotech & Medicine
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UK Clinical Trial Reforms Take Effect, Cutting Set-Up Times to 122 Days and Making Trial Registration a Legal Requirement for the First Time
The MHRA and HRA's largest regulatory overhaul in 20 years went live April 28, introducing notifiable trials, a 14-day automatic-approval modification route, and mandatory transparency for UK studies.
FDA Launches Real-Time Clinical Trials Pilot With AstraZeneca and Amgen, Aiming to End Years-Long Reporting Lags
The FDA announced two proof-of-concept real-time cancer trials and opened public comment on a broader pilot program, betting that cloud and AI-mediated data flow can compress drug review timelines.
Lilly Bets Up to $2.25 Billion on Profluent's AI-Designed Recombinases, Pushing Gene Editing Beyond CRISPR Into Kilobase-Scale Territory
Eli Lilly's deal with Bezos-backed Profluent funds AI-designed recombinases that aim to insert entire genes, going beyond CRISPR's localized cuts.
FDA Awards National Priority Vouchers to Three Psychedelic Programs, Compressing Review Times to One to Two Months
Compass Pathways, Usona Institute, and Otsuka-acquired Transcend Therapeutics have been selected for FDA's Commissioner's National Priority Voucher program for psilocybin and methylone, six days after President Trump's executive order on serious mental illness.
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.