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Cardiovascular drug development setback
Novartis’ pelacarsen failed to meet its primary goal in a pivotal Phase 3 outcomes study, as the company said the drug did not reduce cardiovascular death or emergencies such as heart attack or...
Oncology immunotherapy platform
Case Western Reserve University researchers unveiled a mechanistic blueprint for a next-generation cancer vaccine that pairs personalized mRNA payloads with engineered exosomes. The goal is to...
Cancer biology and microenvironment
A new study mapped the molecular fingerprint of hemorrhagic IDH-wildtype glioblastoma, suggesting that intratumoral bleeding is tied to identifiable genetic architecture rather than being a purely...
Alzheimer’s diagnostics under scrutiny
Researchers in a Journal of Molecular Medicine review argue that the FDA-cleared Lumipulse G plasma p-tau217/Aβ1−42 ratio has flaws as a blood-based Alzheimer’s diagnostic test. The paper by...
Gene therapy manufacturing partnership
NewBiologix and Synastra Biotechnology signed an agreement to develop a stable producer cell line for Synastra’s investigational Duchenne muscular dystrophy (DMD) gene therapy program. NewBiologix...
CAR T and cell therapy manufacturing innovation
Researchers from The Second Qilu Hospital of Shandong University reported a microfluidic manufacturing method to boost CAR-T cell transduction at low viral doses. The work targets a recurring...
Public health diagnostics and infectious disease surveillance
A surveillance study from Japan reports that sentinel surveillance missed an early wave of Mycoplasma pneumoniae, while universal PCR testing at a single hospital detected local epidemic activity....
Biotech financing and corporate moves
NeuShen Therapeutics secured more than $80 million in a Series B to advance three clinical-stage brain-health drug programs. The Shanghai-and-Boston biotech is led by a veteran transpacific drug...
Cancer risk biology and inherited predisposition
A rare case report described a metastatic pancreatic neuroendocrine tumor arising in the setting of Lynch syndrome, tracing an unusually complex genetic cancer history. The case, published in BMC...
FDA greenlights first disease-modifying therapy for Alexander disease
The FDA approved Ionis Pharmaceuticals’ antisense therapy zilganersen (Zanvastro) for Alexander disease in pediatric and adult patients, making it the first and only disease-modifying treatment...
Novartis/ Ionis Lp(a) therapy fails pivotal Phase 3
Novartis said its pelacarsen, the Ionis-partnered antisense therapy targeting lipoprotein(a) [Lp(a)], failed to reduce cardiovascular events in the Phase 3 HORIZON trial. The result is a setback...
CAR-T safety signals drive Novartis trial pause
Novartis paused immunology and neuroscience studies after reporting deaths of three patients who received its experimental CAR-T therapy, according to a company update discussed in industry...
Gene therapy manufacturing deal for Duchenne rAAV production
NewBiologix and Synastra Biotechnology struck an agreement to develop a stable producer cell line for Synastra’s investigational Duchenne muscular dystrophy (DMD) rAAV gene therapy program. Under...
Advancing CAR-T manufacturing throughput via low-dose microfluidics
Researchers from The Second Qilu Hospital of Shandong University reported a microfluidic method that boosts CD19-targeting CAR-T cell transduction under low viral-dose conditions. The study...
Clinical imaging path for PreserFlo MicroShunt success prediction
A Japanese study reported that anterior segment OCT imaging can predict one-year success following PreserFlo MicroShunt surgery. By linking imaging features to surgical outcomes, the work aims to...
New FDA-driven rare-disease pipeline milestone in oncology and beyond
Ionis’ Alexander disease approval highlighted in industry reporting signals a rare-disease “pipeline pillar” effect, where regulatory wins can translate into faster downstream development and...
AbbVie’s dual-acting myeloma drug posts Phase 3 win
AbbVie reported that its dual-acting multiple myeloma bispecific antibody etentamig met the main goal of a Phase 3 trial, improving survival without progression for patients who had progressed on...
FDA and regulatory shift for botanical drugs via RFI
The FDA issued a request for information aimed at accelerating development and review of botanical drug products in the U.S. The agency is seeking input on challenges sponsors face and potential...
rAAV and cell therapy startup financing to propel first clinical study
Superluminal Medicines secured an oversubscribed $60 million Series B round to drive its pipeline forward and support a first clinical study for endocrine and cardiometabolic targets. The...
FDA approval—rare neurodegeneration
FDA cleared Ionis Pharmaceuticals’ antisense therapy Zanvastro (zilganersen) for Alexander disease in pediatric and adult patients, marking the first and only disease-modifying treatment for the...