Potency of CAR T-Cell Therapy for Myeloma May Vary by Disease Stage

Source CancerNetwork CAR T cells are a more effective therapy if manufactured for patients with multiple myeloma prior to the onset of relapsed or refractory disease, according to study results1 published in Blood Advances. “CAR T cells directed against the B-cell maturation antigen (BCMA) have demonstrated impressive initial results, but available data suggest that most patients with initial responses

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Early data for Novartis gene therapy show improvements for older spinal muscular atrophy patients

Source MedCity News Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2. A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children. Basel,

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Understanding the Challenges of Reconnecting Neurons after Spinal Cord Injury

Source University of Miami Health System Neuroscientists have been struggling for years to understand why neurons in the brain and spinal cord have so much difficulty re-growing connections after injury caused by trauma or disease. An intercontinental collaboration between the University of Miami and Imperial College London neuroscientists and computer scientists provides new insight into

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Carnegie Mellon and Yale Universities Receive NIH Grant To Further innovative, synthetic nucleic acid-based gene editing technique

The peptide nucleic acid-based technique offers an alternative to CRISPR-Cas9 A research team from Carnegie Mellon University and Yale University will advance their innovative, synthetic nucleic acid-based gene editing technique under a new grant from the National Institutes of Health’s (NIH’s) Somatic Cell Genome Editing (SCGE) Program. “Our PNA technique offers a promising avenue for treating — and possibly curing —

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New Automation Solution to Accelerate Cell and Gene Therapy Manufacturing Announced

Terumo BCT’s latest cell therapy technology, the Finia® Fill and Finish System, is a first-of-its-kind device developed to help bring reproducibility and control to cell therapy manufacturing to get therapies to more patients who need them. Finia is a fully automated, modular, functionally closed system that creates the final formulation of cell and gene therapies

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Penn developed Gene Therapy for Duchenne Muscular Dystrophy Safely Preserves Muscle Function

Penn study shows delivering a “distant cousin” of a key protein prevented muscle damage without triggering an immune response in large animal models A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal

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Researchers identify molecular process that could accelerate recovery from nerve injuries

Twenty million Americans suffer from peripheral nerve injuries, which can be caused by traumas such as combat wounds and motorcycle crashes as well as medical disorders including diabetes. These injuries can have a devastating impact on quality of life, resulting in loss of sensation, motor function and long-lasting nerve pain. The body is capable of

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