US2021139911A1PendingUtilityA1

Antisense composition and method for treating muscle atrophy

Assignee: SAREPTA THERAPEUTICS INCPriority: Feb 9, 2005Filed: Mar 10, 2020Published: May 13, 2021
Est. expiryFeb 9, 2025(expired)· nominal 20-yr term from priority
C12N 2320/30C12N 15/1136C12N 2310/11C07H 21/00C12N 2310/3513C12N 2310/3145C12N 2310/3233A61K 47/645A61K 31/675Y10T436/143333C12N 2310/31A61P 21/00C12Q 1/6876C12Q 2600/158C07K 7/08
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Claims

Abstract

A method and compound for treating skeletal muscle mass deficiency in a human subject are disclosed. The composition is an oligomer of morpholino subunits and phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5′ exocyclic carbon of an adjacent subunit, contains between 10-40 nucleotide bases, has a base sequence effective to hybridize to an expression-sensitive region of processed or preprocessed human myostatin RNA transcript, identified, in its processed form, by SEQ ID NO:6, and is capable of uptake by target muscle cells in the subject. In practicing the method, the compound is administered in an amount and at a dosage schedule to produce an overall reduction in the level of serum myostatin measured in the patient, and preferably to bring the myostatin level within the a range determined for normal, healthy individuals.

Claims

exact text as granted — not AI-modified
1 . (canceled) 
     
     
         2 . An antisense oligomer consisting of a sequence selected from SEQ ID NOS: 1-5. 
     
     
         3 . A pharmaceutical composition comprising the antisense oligomer of  claim 2  and a pharmaceutically acceptable carrier.

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