Curated news and analysis on clinical trials, drug approvals, and medical research.
Scientists have developed two breakthrough approaches to dramatically enhance gene editing and mRNA therapy delivery: a simple amino acid supplement that increases CRISPR efficiency to nearly 90 percent, and a self-replicating CRISPR system that spreads between cells like a virus.
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
New market reports forecast significant expansion in CAR T-cell therapy, US pharmaceutical drug delivery, and healthcare nanotechnology sectors, driven by chronic disease prevalence and technological advances.
Brainstorm Cell Therapeutics announced a $1 million private placement at a premium to market price to support its upcoming Phase IIIb trial of NurOwn stem cell therapy for amyotrophic lateral sclerosis.
Researchers identify speech latency as a biomarker to enrich schizophrenia clinical trials, while a separate study finds clozapine more effective than alternatives for patients who fail initial antipsychotic treatment.
Pembrolizumab-based regimens demonstrated significant survival improvements in two separate trials: KEYNOTE-B15 for muscle-invasive bladder cancer and KEYNOTE-B96 for platinum-resistant ovarian cancer, with FDA approval granted for the ovarian cancer indication.
The global precision medicine market reached $82.1 billion in 2025 and is expected to grow to $179.4 billion by 2034. Researchers emphasize that precision nutrition interventions must demonstrate cost-effectiveness to deliver patient benefits.
New research from 18 countries shows COVID-19 vaccination during pregnancy, particularly with booster doses, significantly reduces the risk of preeclampsia and other serious complications for both mothers and babies.
The FDA is accelerating its review of daraxonrasib, a targeted therapy for pancreatic cancer, under a new pilot program that could reduce approval time from 10-12 months to one to two months.
Merck announced multiple data presentations at the 2026 ASCO GU Cancers Symposium, including results from trials evaluating pembrolizumab combinations in muscle-invasive bladder cancer and renal cell carcinoma.
The FDA issued reforms on January 11, 2026, loosening chemistry, manufacturing, and control requirements for cell and gene therapy products to advance innovation and accommodate the unique complexity of these treatments.
The global next generation drug conjugates market is projected to grow from $3.9 billion in 2026 to $10.9 billion by 2033 at 15.3% CAGR, while the radionuclide drug conjugate segment is forecast to reach $20.22 billion by 2032 at 9.6% CAGR.
Exploratory analysis from a phase 1/2 trial shows CD47 expression levels predict response to evorpacept plus zanidatamab in heavily pretreated HER2-positive metastatic breast cancer patients, supporting biomarker-driven patient selection.
The FDA has approved leucovorin for cerebral folate deficiency with FOLR1 gene variant, not for autism broadly. Agency officials cited insufficient data for broader autism efficacy despite previous hype. Prescriptions for the drug surged 71% among children following earlier promotional statements.
Sagimet Biosciences reported fourth quarter and full year 2025 financial results, announcing plans to initiate a Phase 2 trial of denifanstat and resmetirom combination in F4 MASH patients in the second half of 2026, with a 26-week biomarker readout expected in the first half of 2028.
Ascletis Pharma announced positive Phase II results for subcutaneous ASC30 showing 6.3% placebo-adjusted weight loss at 12 weeks, while advancing oral amylin receptor agonist ASC36 toward US FDA IND submission for obesity treatment.
The FDA has granted fast track designation to QRX003 for Netherton Syndrome and IBI3003 for relapsed or refractory multiple myeloma, while also accepting regulatory applications for other therapies addressing serious conditions with unmet medical needs.
Researchers have created a nasal vaccine platform that protects mice against flu, COVID-19, SARS, and bacterial infections by activating both innate and adaptive immune systems, offering a fundamentally different approach to disease prevention.
Researchers at the Regina Elena National Cancer Institute in Rome discovered how the hMENA protein enables non-small cell lung cancer to resist immunotherapy by regulating cancer-associated fibroblasts that create a protective tumor microenvironment.
Recent studies demonstrate zanubrutinib delivers higher response rates, longer treatment persistence, and lower healthcare costs compared to acalabrutinib and ibrutinib across multiple B-cell malignancies, with particularly strong benefits in high-risk populations.
