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 Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases

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Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and…

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