EP4299750A2 — Compositions and methods for treating ornithine transcarbamylase deficiency
Assigned to Arcturus Therapeutics Inc · Expires 2024-01-03 · 2y expired
What this patent protects
The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC …
USPTO Abstract
The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC deficiency wherein upon administration of the mRNA to the patient, the protein of the disclosure is expressed in the patient in therapeutically effective amounts to treat OTC deficiency. The present disclosure also provides codon optimized mRNA sequences encoding wild type human OTC comprising a 5' UTR derived from a gene expressed by Arabidopsis thaliana for use in treating OTC deficiency in a patient.
Drugs covered by this patent
- Onpattro (PATISIRAN SODIUM) · Alnylam Pharmaceuticals
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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