Related coverage linked through entity extraction aliases.
Feb 28, 2026
Researchers developed a post-assembly crosslinking approach that improves the structural stability and endosomal escape efficiency of mRNA lipid nanoparticles, potentially expanding applications for RNA therapeutics and vaccines.
Feb 27, 2026
Research identifies orphan nuclear receptor SHP (NR0B2) as a novel regulator of osteoarthritis progression, demonstrating that it protects cartilage by inhibiting IKKβ/NF-κB signaling and reducing matrix-degrading enzyme expression in chondrocytes.
Feb 26, 2026
Orphan drug development faces structural uncertainty due to small patient populations and varying global definitions, with regulators balancing early access against evidence requirements through accelerated pathways and post-market monitoring.
Feb 24, 2026
The FDA reversed its decision to reject Moderna's mRNA influenza vaccine application, accepting it for review one week after an initial refusal-to-file that reportedly involved senior leaders overruling staff advice.
Feb 23, 2026
Benitec Biopharma will present interim clinical study results for BB-301 gene therapy in oculopharyngeal muscular dystrophy patients at the Muscular Dystrophy Association Conference on March 9, 2026, including 12-month and 24-month follow-up data.
Feb 20, 2026
Switzerland-based CRISPR Therapeutics stands to benefit from increased Casgevy sales following partner Vertex Pharmaceuticals' fourth-quarter results, with five additional gene-editing therapies in clinical trials targeting larger patient populations.
Feb 20, 2026
Former Novartis executive John Tsai has been appointed global head of R&D at Daiichi Sankyo, replacing Ken Takeshita effective April 1. Tsai brings over 25 years of leadership experience in drug development.
Feb 17, 2026
Ultragenyx Pharmaceutical confronts multiple shareholder class action lawsuits over Phase III trial disclosures while reporting positive UX111 gene therapy data and implementing a 10% workforce reduction.
Feb 14, 2026
Eli Lilly achieved positive outcomes for nearly all R&D key events in 2025 and acquired Orna Therapeutics for $2.4 billion to develop circular RNA therapies for autoimmune diseases.
Feb 13, 2026
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
Jan 29, 2026
The FDA placed clinical holds on Regenxbio's RGX-111 and RGX-121 gene therapies following a tumor case in an MPS I patient. The company's stock fell nearly 18% on the news.
Feb 13, 2026
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.
