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Treatment for McArdle Disease

A viral therapy for McArdle disease that utilises a potent, muscle-specific promoter to deliver muscle glycogen phosphorylase

Published: 27th January 2020
Treatment for McArdle Disease
Jensflorian, Wikimedia, CC BY-SA 3.0
  • An Australian provisional patent application #201902856 entitled, “McArdle Disease Therapy” was filed on 9th Aug 2019. The therapy is considered to be novel (DCC assessment) but nevertheless, given this is a rare disease it is likely that the FDA will allow for access to the government incentives plus seven years of exclusive rights to treat that rare disease (U.S. Orphan Drug Act).
  • In addition, this is likely to be eligible for the Biological Exclusivity period of 12 years in the US (Patient Protection and Affordable Care Act (Public Law 111-148) BLA) and EU provides 10 years exclusivity (8 years for data and 2 years market).
IP Status
  • Patent application submitted
  • Licensing
  • Development partner
  • Commercial partner