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UCLA, University College London Advancing Immune Disorder Gene Therapy Toward FDA Submission
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Researchers founded Rarity PBC as a public benefit corporation that will license the IP and develop operations to potentially market the gene therapy for ADA-SCID.
In particular, patients who had fewer than 10 annual vaso-occlusive events before the gene therapy were more likely to have none after treatment.
In a small validation set, the model was able to make accurate predictions about patient outcomes using routinely collected patient data.
'Fresh-In, Fresh-Out' Dual-Targeted CAR T-Cell Therapy Shows Promise in Phase II DLBCL Trial
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The non-cryopreserved CD19- and CD20-directed CAR T-cell therapy zamto-cel is manufactured in a closed-system device that could enable point-of-care treatment.
HER2 Biomarker Evolving as Enhertu Shows Activity in Breast Cancers With Ultralow Expression
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The new HER2-low category is exposing the limitations of IHC testing, challenging pathologists, and has oncologists revisiting patients' old test results.