US 11,793,887 B2
Gene therapy for treating peroxisomal disorders
Nancy Braverman, Montreal (CA); Catherine Argyriou, Montreal (CA); Joseph Hacia, Pasadena, CA (US); Jean Bennett, Philadelphia, PA (US); Junwei Sun, Philadelphia, PA (US); Ji Yun Song, Lower Gwynedd, PA (US); and Devin McDougald, Philadelphia, PA (US)
Assigned to The Trustees of the University of Pennsylvania, Philadelphia, PA (US); University of Southern California, Los Angeles, CA (US); and The Research Institute of McGill University Health Centre, Montreal (CA)
Appl. No. 16/615,941
Filed by The Trustees of the University of Pennsylvania, Philadelphia, PA (US); University of Southern California, Los Angles, CA (US); and The Research Institute of the McGill University Health Centre, Montreal (CA)
PCT Filed May 31, 2018, PCT No. PCT/CA2018/050642
§ 371(c)(1), (2) Date Nov. 22, 2019,
PCT Pub. No. WO2018/218359, PCT Pub. Date Dec. 6, 2018.
Claims priority of provisional application 62/513,156, filed on May 31, 2017.
Prior Publication US 2020/0093937 A1, Mar. 26, 2020
Int. Cl. A61K 48/00 (2006.01); C12N 15/79 (2006.01); C07H 21/04 (2006.01); A61K 35/761 (2015.01); C12N 9/14 (2006.01); C12N 15/86 (2006.01); C12N 15/864 (2006.01)
CPC A61K 48/005 (2013.01) [A61K 35/761 (2013.01); C12N 9/14 (2013.01); C12N 15/86 (2013.01); C07H 21/04 (2013.01); C12N 15/8645 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01)] 21 Claims
 
1. A method of treating a deficiency in peroxisome biogenesis factor 1 (PEX1) in a subject in need thereof, comprising directly administering to an eye of the subject a recombinant adeno-associated virus (rAAV) comprising an AAV capsid having packaged therein a vector genome comprising a nucleic acid sequence that encodes human PEX1, thereby improving visual function in the subject.