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Broad State Participation in Medicaid Sickle Cell Gene Therapy Program
Approximately 33 states representing 84% of Medicaid beneficiaries have adopted a Centers for Medicare & Medicaid Services (CMS) program enabling pay-for-performance arrangements with gene therapy...
Genetic Alliance Expands Rare Disease Genomic Testing via $500K Grant
Genetic Alliance has secured a $500,000 grant from the Helmsley Charitable Trust to broaden its iHope Rare Disease program, which offers free whole-genome and exome sequencing for underserved...
ABL Diagnostics Partners for Molecular Assay Distribution in South Africa
ABL Diagnostics entered a commercialization agreement with Anatech Instruments to market its DeepChek and UltraGene molecular assay lines in South Africa. Anatech will offer DeepChek’s sequencing...
Ultragenyx Gene Therapy for Sanfilippo Syndrome Faces FDA Manufacturing Rejection
The FDA issued a complete response letter rejecting Ultragenyx’s gene therapy UX111 for Sanfilippo syndrome type IIIA, citing manufacturing process deficiencies identified during facility...
ABB AstraZeneca’s Phase III Win with Baxdrostat in Resistant Hypertension
AstraZeneca announced positive results from its BaxHTN phase III trial where baxdrostat, an aldosterone synthase inhibitor, achieved primary and secondary endpoints in patients with uncontrolled...
Hengrui and Kailera Advance Dual-Acting Obesity Drug with Chinese Phase III Success
Hengrui Pharmaceuticals and Kailera Therapeutics reported strong Phase III efficacy for their injectable GLP-1/GIP dual receptor agonist in China, showing approximately 18% mean weight loss over...
Waters to Acquire BD Biosciences and Diagnostics for $17.5 Billion
Waters Corp announced its $17.5 billion acquisition of Becton Dickinson’s Biosciences and Diagnostics business to expand into molecular testing and diagnostics. The deal nearly doubles Waters’...
Proteomics Consortium Releases Extensive Neurodegeneration Dataset
The Global Neurodegeneration Proteomics Consortium (GNPC) released one of the largest dementia-related proteomic datasets, including 250 million protein measures from over 35,000 biofluid samples...
FDA rejects Ultragenyx gene therapy: Manufacturing concerns stall Sanfilippo treatment
Ultragenyx Pharmaceutical encountered a setback when the FDA issued a complete response letter for its gene therapy UX111 targeting Sanfilippo syndrome type A, citing manufacturing process...
Hengrui/Kailera obesity drug posts near 18% weight loss in China trial; global trials planned
Hengrui Pharmaceuticals and startup Kailera Therapeutics reported phase 3 results from China positioning their once-weekly injectable dual GLP-1/GIP receptor agonist as a potent obesity treatment....
Sanofi’s $9.1B Blueprint Medicines acquisition boosts rare disease and oncology pipeline
Sanofi completed the $9.1 billion acquisition of Blueprint Medicines, gaining access to the approved KIT inhibitor Ayvakit for systemic mastocytosis and a robust pipeline in immunological diseases...
Waters to acquire BD’s biosciences and diagnostics unit in $17.5B deal
Waters Corporation announced plans to acquire Becton Dickinson's Biosciences and Diagnostic Solutions business for $17.5 billion, marking this year's largest life sciences deal. The acquisition...
AbbVie licenses Ichnos Glenmark trispecific antibody in $1.9B-plus multiple myeloma pact
AbbVie secured global rights to ISB 2001, a first-in-class trispecific T-cell engager targeting BCMA, CD38, and CD3 for multiple myeloma, via an exclusive license deal with Ichnos Glenmark...
FDA phasing out animal testing using AI and advanced human models to accelerate drug development
The FDA unveiled plans to reduce reliance on animal testing within 3-5 years for drug development, emphasizing the adoption of human-relevant methods such as organoids, organ-on-a-chip, and...
Global neurodegeneration proteomics consortium reveals biomarkers across diseases
An international consortium published one of the world’s largest proteomic datasets linking blood protein signatures with major neurodegenerative diseases including Alzheimer’s, Parkinson’s,...
Chimeric antigen receptor (CAR) therapies and tumor resistance: dual payload ADC advances
New strategies in antibody-drug conjugates (ADCs), including dual-payload ADCs delivering two distinct cytotoxic agents simultaneously, are gaining momentum to overcome tumor resistance due to...
Bird flu persists with ecological and zoonotic risks despite fading news coverage
The highly pathogenic H5N1 avian influenza continues circulating in wildlife and livestock, including surprising host jumps to marine mammals and dairy cattle, raising concerns about viral...
AbbVie challenges Missouri law regulating 340B contract pharmacies: Legal setback
AbbVie failed to secure a judicial injunction overturning Missouri's regulation of 340B contract pharmacy practices, facing a second legal defeat in weeks related to the federal 340B drug pricing...
FDA and NIH Push to End Animal Testing in Drug Development
The FDA has unveiled a roadmap aiming to phase out animal testing in drug development within three to five years, leveraging advanced human cell-based assays, organoid technologies, and AI-driven...
AbbVie Licenses Trispecific T-Cell Engager for Multiple Myeloma
AbbVie secured exclusive global rights to ISB 2001, a trispecific T-cell engager developed by Ichnos Glenmark Innovation targeting BCMA, CD38, and CD3 for relapsed/refractory multiple myeloma....