Abstract: Methods of treating hemophilia A in a subject with an F8 gene mutation, wherein the F8 gene is repaired and the resultant repaired gene, upon expression, confers improved coagulation functionality to the encoded FVIII protein of the subject compared to the non-repaired F8 gene. The invention also includes methods of inducing immune tolerance to a FVIII replacement product ((r)FVIII) in a subject having a FVIII deficiency, wherein the F8 gene mutation is repaired and the repaired gene, upon expression, provides for the induction of immune tolerance to an administered replacement FVIII protein product. The invention also includes isolated nucleic acids, vectors, recombinant viruses, cells, and pharmaceutical compositions to repair the F8 gene.
Type:
Application
Filed:
April 26, 2019
Publication date:
November 21, 2019
Applicants:
Haplomics, Inc., The Regents of the University of California, The United States Government as respresented by the Department of Veterans Affairs
Abstract: Methods of treating a subject having or at risk of developing a neurodegenerative disease or condition associated with demyelination, insufficient myelination, or underdevelopment of myelin sheath are described. The methods include administration of a therapeutically effective amount of sobetirome, or a pharmaceutically acceptable salt thereof.
Type:
Application
Filed:
February 13, 2019
Publication date:
June 13, 2019
Applicants:
Oregon Health & Science University, The United States Government as respresented by the Department of Veterans Affairs
Inventors:
Thomas S. Scanlan, Meredith Hartley, Andrew Placzek, Marco Righi, Dennis Bourdette, Gail Marracci, Priya Chaudhary
Abstract: Methods of treating a subject having or at risk of developing a neurodegenerative disease or condition associated with demyelination, insufficient myelination, or underdevelopment of myelin sheath are described. The methods include administration of a therapeutically effective amount of sobetirome, or a pharmaceutically acceptable salt thereof.
Type:
Application
Filed:
February 5, 2014
Publication date:
March 24, 2016
Applicants:
Oregon Health & Science University, The United States Government as respresented by the Department of Veterans Affairs
Inventors:
Thomas S. Scanlan, Meredith Hartley, Andrew Placzek, Marco Righi, Dennis Bourdette, Gail Marracci, Priya Chaudhary