NEW YORK, Jan. 22, 2021 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today that it has received a Notice of Allowance from the Japanese Patent Office (Japanese patent number 2016-512480) covering SLS-005 titled: "Treatment of Protein Aggregation Myopathic and Neurodegenerative Diseases by Parenteral Administration of Trehalose".
The allowed claims cover a pharmaceutical formulation comprised of trehalose for use in a method for treating or alleviating oculopharyngeal muscular dystrophy (OPMD) in a human subject. The allowed claims cover a method comprised of an intravenous administration of a therapeutically effective amount of a pharmaceutical formulation, with trehalose as the sole active ingredient, that complies with other parameters regarding the formulation characteristics and duration of intravenous administration, as described in the patent application.
Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells. In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.
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Seelos Therapeutics, Inc. (Nasdaq: SEEL)
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SOURCE Seelos Therapeutics, Inc.