Therapeutic or Preventive Procedure
Novartis Prepares to File Intrathecal Zolgensma for Older SMA Patients by Mid-2025
Zolgensma, intrathecal delivery, spinal muscular atrophy, gene therapy, older patients
Roche continues ADC dealmaking spree with $1B biobucks pact with Oxford BioTherapeutics
OBT, therapeutic autologous dendritic cells, Roche (company), Partnership, Oxford BioTherapeutics, Antibody-Drug Conjugates, biobucks, New
Sarepta Reports Death of DMD Patient Following Elevidys Gene Therapy Treatment
Sarepta, Elevidys, gene therapy, Duchenne muscular dystrophy, patient death, acute liver failure
Sofinnova Partners Launches Three New Biotechs with €165M Acceleration Fund
Sofinnova Partners, biotech acceleration fund, BioClec, Forth Therapeutics, Signadori Bio, venture capital, pharmaceutical backing
Arch-backed HC Bioscience closes doors after seeing early data for tRNA asset
Vision, hydrocortisone, Bioscience
Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment
Del-zota, Duchenne muscular dystrophy (DMD), RNA therapeutics, exon 44 skipping, dystrophin production, creatine kinase reduction, safety profile
Lonza’s Vision for the Future: A Fireside Chat with New CEO Wolfgang Wienand
Lonza, Wolfgang Wienand, CDMO, Pharmaceutical manufacturing, Biologics, Cell and gene therapy, Small molecules, Leadership transition, Industry outlook
AstraZeneca deepens cell therapy expansion with $1B EsoBiotec buyout
AstraZeneca, EsoBiotec, cell therapy, acquisition, ENaBL platform, in vivo cell engineering, oncology, autoimmune diseases
Orca’s cell therapy trounces traditional care in blood cancer phase 3, swimming into space’s ‘holy grail’
allogeneic hematopoietic stem cell transplant with purging, Leukemia, Myelocytic, Acute, Acute lymphocytic leukemia