Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis

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Therapeutic Candidate or Device AS-202, an antisense oligonucleotide Indication Amyotrophic Lateral Sclerosis Therapeutic Mechanism AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly…

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Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)

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Therapeutic Candidate or Device MZ-9138 is an AAV9 gene therapy Indication WWOX-related epileptic encephalopathy Therapeutic Mechanism MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and…

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IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome

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Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy…

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