It appears Shire's had interest in Friedreich's Ataxia for awhile now.
Jun 15, 2012 - Shire Human Genetic Therapies, Inc.
The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.
Patent History Patent number: 9260495 Type: Grant Filed: Jun 15, 2012 Date of Patent: Feb 16, 2016 Patent Publication Number: 20140135275 Assignee: Shire Human Genetic Therapies, Inc. (Lexington, MA) Inventors: Dennis Keefe (Wilmington, MA), Michael Concino (Bolton, MA), Michael Heartlein (Boxborough, MA), Serene Josiah (Cambridge, MA), Bettina Strack-Logue (Somerville, MA) Primary Examiner: Alexander Kim Application Number: 14/127,074
patents.justia.com/patent/9260495
I believe most of the inventors are still with Shire.
My posts are solely my opinion unless presented with (or directed to) factual information. All investors are strongly encouraged by myself to do their own due diligence before making an investment decision.