The #FDA granted #dostarlimab Breakthrough Therapy status for DMMR/MSI-H cases. Clinical trials achieved a 100% pathological complete response in selected patients.
www.gsk.com/en-gb/media/...

Researchers at #CIMA have shown that blocking GDF-15 with visugromab restored immune response in resistant cancers. In trials, 19% of lung cancer & 18.5% of bladder cancer patients responded, achieving deep remissions after failing prior therapies. #Oncology
go.nature.com/3ORMltG

#CIMA scientists have cured lung cancer (metastatic and non metastatic) in 100% of mouse models by combining #DSTYK -targeted therapy with taxane-based chemotherapies like paclitaxel and docetaxel.
bit.ly/3ZoNrSx

FDA releases new draft guidance to fast-track drug approvals by up to 30%. This update focuses on enhancing criteria for breakthrough therapies and expanding eligibility for accelerated pathways, aiming to significantly reduce time-to-market for critical treatments. bit.ly/41nVMIR
#Pharma #FDA

New bioluminescent immunoassay detects anti-interferon-gamma antibodies with 100% accuracy (AUROC). Enhanced sensitivity vs ELISA and potential for better diagnosis in rare immunodeficiencies. Liquid-phase is a plus. #Immunology #Lumit
bit.ly/4gl3AiW

Led by #CIEMAT 's Biomedical Innovation Unit, a study demonstrates that gene therapy halted bone marrow failure in 62.5% of Fanconi anemia patients without the need for toxic conditioning, with safety and efficacy sustained over a 7-year follow-up👏🏻. #GeneTherapy #FanconiAnemia

GSK teams up with Muna Therapeutics in a $730M deal to combat Alzheimer's. Muna's MiND-MAP platform uses single-cell spatial multi-omics to tackle resilience mechanisms in neurodegeneration, including the TREM2 agonist program which aims to enhance microglial function and protect neurons.#Alzheimers

New findings from IRB show CPEB4 condensates disrupt synaptic processes by 40% in autism, offering new therapeutic targets #autism #CPEB4
go.nature.com/4fZhKq9

Lilly’s Zepbound (tirzepatide) outshines Novo’s Wegovy (semaglutide) in a head-to-head trial: 20.2% avg weight loss vs 13.7%. The obesity drug race heats up with results from #SURMOUNT5. #Obesity

Roche’s acquisition of Poseida spotlights a bold step in CAR-T therapy. Poseida’s off-the-shelf, donor-derived approach tackles scalability and safety with Cas-CLOVER gene editing, enabling multi-antigen targeting and reduced GvHD. #CellTherapy #CARTs

Novartis acquires the rights to PTC518, a splicing modifier targeting Huntington’s disease, in a $2.9B deal with PTC Thx. Early studies show it reduces mutant huntingtin protein levels effectively. pubmed.ncbi.nlm.nih.gov/39155237/
#HuntingtonsDisease #PharmaNews #DrugDevelopment
#SmallMolecules