11 patents
Utility
Frataxin-sensitive Markers for Monitoring Frataxin-replacement Therapy
9 Nov 23
The present disclosure is based, at least in part, on providing a set of markers, also referred to herein as FXN-sensitive genomic markers (or FSGMs), the respective expression levels of which are positively or negatively correlated to frataxin (FXN) levels in a cell.
Joan David BETTOUN, Matthew Garrett BAILE, Ruihua CHEN
Filed: 6 Apr 23
Utility
Tat Peptide Binding Proteins and Uses Thereof
2 Mar 23
The present invention encompasses TAT peptide binding proteins.
Xin XU, Erik Johan Wagner, Bonita Rup
Filed: 5 Feb 21
Utility
Use of Frataxin for Treating Leigh Syndrome, French Canadian Type
1 Dec 22
The present disclosure provides a method for treatment of a subject suffering from Leigh Syndrome French Canadian Type (LSFC), the method comprising administering to the subject a therapeutically effective amount of a frataxin (FXN) therapeutic compound.
Joan David Bettoun
Filed: 17 Jul 20
Utility
Methods of Quantifying Frataxin and Frataxin Fusion Proteins
1 Sep 22
The present disclosure provides methods for determining the amount of FXN or FXN fusion protein in a sample, e.g, a tissue sample, by using mass spectrometry.
Joan David Bettoun, Erik Johan Wagner, Xin Xu, Jean-Nicholas Mess, Amy Qiu Wang
Filed: 29 Jul 20
Utility
Pharmaceutical Composition Comprising Frataxin Fusion Protein and Methods of Use Thereof
23 Jun 22
Pharmaceutical compositions comprising a TAT-FXN fusion polypeptide are disclosed, as are methods of use of the pharmaceutical compositions to treat subjects diagnosed with Friedrich's Ataxia and/or hypertrophic cardiomyopathy.
Denis Boyle, Thomas Ransohoff
Filed: 13 Dec 21
Utility
Methods for Treating Myelin Associated Diseases and Mitochondria Associated Diseases
25 Nov 21
The present disclosure provides methods for treating or ameliorating a myelin associated disease or a mitochondria associated disease that comprising administering to a subject in need thereof a frataxin replacement therapeutic compound, e.g., a fusion protein comprising frataxin.
Joan David Bettoun
Filed: 30 Apr 21
Utility
Molecules for Organelle-specific Protein Delivery
18 Nov 21
The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia.
Joan David Bettoun, Rebecca Wissner
Filed: 26 Mar 21
Utility
Methods for Quantifying Frataxin Activity
27 May 21
The present disclosure provides methods, compositions and kits for measuring activity of a frataxin (FXN) protein, e.g., an FXN fusion protein.
Joan David Bettoun
Filed: 25 Nov 20
Utility
Pharmaceutical Compositions of METAP-2 Inhibitors
29 Jan 20
Disclosed herein, in part, are pharmaceutical compositions comprising a MetAp-2 inhibitor and a pharmaceutically acceptable excipient.
James E. Vath, Bryan Burkey
Filed: 11 Feb 18
Utility
Methods for Identifying METAP-2 Modulators
1 Jan 20
Disclosed herein, in part, are methods for identifying MetAP-2 inhibitors to address the treatment of obesity and related diseases as well as other ailments favorably responsive to MetAP-2 modulator treatment.
James E. Vath, Bryan Burkey
Filed: 11 Feb 18
Utility
Fumagillol Type Compounds and Methods of Making and Using Same
25 Dec 19
The disclosure provides fumagillol type compounds and their use in treating medical disorders, such as obesity.
Robert Zahler, James E. Vath
Filed: 22 Jan 19
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