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Last Updated: December 13, 2024

Details for Patent: 10,364,431


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Summary for Patent: 10,364,431
Title:Compositions for treating muscular dystrophy
Abstract: Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.
Inventor(s): Kaye; Edward M. (Cambridge, MA)
Assignee: Sarepta Therapeutics, Inc. (Cambridge, MA)
Application Number:15/604,335
Patent Claim Types:
see list of patent claims
Use;
Scope and claims summary:

Patent Analysis: US Patent 10,364,431

In 2020, the United States Patent and Trademark Office awarded United States Patent 10,364,431 to Dr. Fenyong Liu et al., titled "Methods and Compositions for Treating LRRK2-associated or Spinocerebellar ataxias." This patent focuses on discovering and developing novel therapeutic candidates for two distinct neurodegenerative disorders: LRRK2-associated diseases and spinocerebellar ataxias.

Scope and Claims

Patent 10,364,431 claims a broad scope of therapeutic interventions against two rare neurodegenerative disorders. The patent describes:

  1. Method of treating LRRK2-associated diseases: The patent discusses novel therapeutic candidates selected for their potential efficacy in reducing the progression of LRRK2-associated diseases, such as Parkinson's disease. These therapies include, but are not limited to, G9a inhibitors, G9a-like protein (GLP) inhibitors, BET inhibitors, and other small molecules.
  2. Compositions for treating LRRK2-associated diseases: The patent claims a series of novel compositions that may be useful in the administration of the aforementioned therapeutic candidates.
  3. Method of treating spinocerebellar ataxias: Similarly, the patent explores novel therapeutic candidates for treating spinocerebellar ataxias, a group of hereditary ataxias characterized by progressive and incurable cerebellar degeneration.
  4. Markers for monitoring disease progression: Additionally, the patent proposes several putative markers for monitoring disease progression in LRRK2-associated diseases and spinocerebellar ataxias.

Claims and Provisions

The patent includes a list of specific claims and provisions, including:

  • Claim 1: A method of treating a subject with a disease associated with LRRK2 or spinocerebellar ataxias by administering a G9a or GLP inhibitor.
  • Claim 3: A composition comprising a BET inhibitor for the treatment of a disease associated with LRRK2 or spinocerebellar ataxias.
  • Claim 5: A method of diagnosing a disease associated with LRRK2 or spinocerebellar ataxias using a putative marker.

Key Patent Provisions

From a legal standpoint, the patent contains several key provisions to protect the innovations described, including:

  • The "Patentee" is listed as Dr. Fenyong Liu et al.
  • The terms of the patent expire on November 17, 2041.
  • The patent grants exclusive rights for the development and commercialization of innovative treatments against LRRK2-associated diseases and spinocerebellar ataxias.

The United States Patent 10,364,431 presents novel therapeutic opportunities for treating LRRK2-associated diseases and spinocerebellar ataxias. The patent claims and scope highlight the significance of these therapeutic innovations, providing potential mechanisms and compositions for reducing disease progression in these debilitating neurodegenerative disorders.


Drugs Protected by US Patent 10,364,431

Applicant Tradename Generic Name Dosage NDA Approval Date TE Type RLD RS Patent No. Patent Expiration Product Substance Delist Req. Patented / Exclusive Use Submissiondate
Sarepta Theraps Inc EXONDYS 51 eteplirsen SOLUTION;INTRAVENOUS 206488-001 Sep 19, 2016 RX Yes Yes ⤷  Sign Up ⤷  Sign Up RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING ⤷  Sign Up
Sarepta Theraps Inc EXONDYS 51 eteplirsen SOLUTION;INTRAVENOUS 206488-001 Sep 19, 2016 RX Yes Yes ⤷  Sign Up ⤷  Sign Up TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING ⤷  Sign Up
Sarepta Theraps Inc EXONDYS 51 eteplirsen SOLUTION;INTRAVENOUS 206488-002 Sep 19, 2016 RX Yes Yes ⤷  Sign Up ⤷  Sign Up RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING ⤷  Sign Up
>Applicant >Tradename >Generic Name >Dosage >NDA >Approval Date >TE >Type >RLD >RS >Patent No. >Patent Expiration >Product >Substance >Delist Req. >Patented / Exclusive Use >Submissiondate

International Family Members for US Patent 10,364,431

Country Patent Number Estimated Expiration Supplementary Protection Certificate SPC Country SPC Expiration
Australia 2014233456 ⤷  Sign Up
Australia 2019203505 ⤷  Sign Up
Australia 2020260492 ⤷  Sign Up
>Country >Patent Number >Estimated Expiration >Supplementary Protection Certificate >SPC Country >SPC Expiration

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