4 patents
Utility
Compositions and Methods for Modulating Forkhead Box P3 (FOXP3) Gene Expression
13 Apr 23
The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a forkhead box P3 (FOXP3) gene by targeting a FOXP3 expression control region and methods of use thereof for treating a FOXP3 associated disorder, such as an autoimmune disease, e.g., IPEX syndrome.
Timsi Rao, Jodi Kennedy, Pankaj Mandal, Jeremiah D. Farelli
Filed: 6 Sep 22
Utility
Methods and Compositions for Modulating Frataxin Expression and Treating Friedrich's Ataxia
3 Nov 22
The present disclosure relates generally to methods and compositions for modulating frataxin (FXN) expression, e.g., to treat Friedreich ataxia (FRDA).
Serena Lunardi, Adam Walter Scheidegger, Jesse Jerome Smith, Jeremiah Dale Farelli, Jodi Michelle Kennedy
Filed: 23 Sep 20
Utility
COMPOSITIONS AND METHODS FOR MODULATING HEPATOCYTE NUCLEAR FACTOR 4-ALPHA (HNF4alpha) GENE EXPRESSION
3 Nov 22
The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4a) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.
Jesse Jerome Smith, Jodi Michelle Kennedy, Jeremiah D. Farelli, Kendrick Alan Goss, Adam Walter Scheidegger, Yoseph Kassa, Christian Wessel Cobaugh, Timsi Rao
Filed: 23 Sep 20
Utility
Compositions and Methods for Modulating Apolipoprotein B (Apob) Gene Expression
15 Sep 22
The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of an apolipoprotein B (APOB) gene by targeting an APOB expression control region and methods of use thereof for treating an APOB associated disorder, e.g., hypercholesterolemia.
Vishwesh Ashok Patil, Jeremiah D. Farelli, Rahul Karnik, Jesse Jerome Smith, Can Sarisozen, Adam Walter Scheidegger, Barbara Bennett
Filed: 22 Mar 22
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