Tom3 schreef op 15 april 2021 13:37:
ASADENA, Calif.--(BUSINESS WIRE)--Apr. 15, 2021-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced ARO-DUX4 as Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform. ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). Pending abstract acceptance, Arrowhead intends to present preclinical data on ARO-DUX4 at the 28th Annual FSHD Society International Research Congress being held virtually on June 24-25, 2021. Arrowhead is currently conducting IND/CTA enabling toxicology studies and intends to file for regulatory clearance in the third quarter of 2021 to begin clinical studies of ARO-DUX4.
Chris Anzalone, Ph.D., president and chief executive officer at Arrowhead, said: “ARO-DUX4 represents further expansion of our pipeline into diseases of the skeletal muscle, which is the fourth cell type that we may potentially be able to target with our proprietary TRiMTM platform. In addition, DUX4 fits perfectly with our strategy not only to bring RNAi outside the liver, but also to select gene targets that we believe are clear causes of specific diseases and for which there is strong biologic and genetic validation. We believe our preclinical data, which we intend to present at the FSHD Society International Research Congress in June pending abstract acceptance, highly support progressing ARO-DUX4 into clinical studies. In our various animal models, ARO-DUX4 reduced DUX4 expression by greater than 70%, prevented body weight loss associated with tamoxifen-induced DUX4 expression, and prevented loss of muscle function.”www.spierziekten.nl/fileadmin/user_up...