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Lisocabtagene maraleucel, a chimeric antigen receptor (CAR) T cell (CAR-T) therapy, is the third gene therapy approved by the US Food and Drug Administration (FDA) for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma. [6] Lisocabtagene maraleucel was approved for medical use in the United States in February 2021 ...
The distribution of the therapeutic enzyme in the body (biodistribution) after these IV infusions is not uniform. [10] The enzyme in less available to certain areas in the body, like the bones, lungs, brain. For this reason, many symptoms of lysosomal storage diseases remain untreated by ERT, especially neurological symptoms. [10]
Wobenzym, a combination of proteolytic enzymes and the antioxidant rutin, works systemically by targeting various tissues and organs in the body.Wobenzym is targeted at modulating the immune response to restore a healthy balance between anti-inflammatory and pro-inflammatory cytokines. [1]
GDEPT is a suicide gene therapy in which the enzyme required for prodrug conversion is produced within the target cell, using a gene delivered to it by gene therapy. When an adequate differential exists between the targeted cell and endogenous tissue, non-toxic prodrug is administered and is subsequently converted into its toxic form within the ...
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Treatment of adults with advanced, unresectable or metastatic non-small cell lung cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy; and adults with advanced, unresectable or metastatic pancreatic adenocarcinoma harboring an NRG1 gene fusion with disease progression on or after prior ...
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