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New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: AGTC, Dyne, Genenta, Luye, Metagenomi, Otonomy, Regenxbio, Tessa, Ultragenyx.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: 4D, Affinia, Arcellx, Generation, Homology, Rocket, Sio Gene, Uniqure.
