Watertown, Mass. — Arkuda Therapeutics, a biotechnology company applying novel insights at the intersection of lysosomal biology and neuronal health to develop medicines that change the trajectory of neurodegenerative disease, announced Arkuda co-founder and previous Senior Vice President, Head of Discovery, Duane Burnett, PhD, has been appointed Chief Technology Officer,...
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ARDSLEY, N.Y. – ARMGO Pharma Inc., a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs known as Rycals®, announced today the publication of the results from a Phase 1b study of its Rycal ARM210 (also known as S48168), for the treatment of Ryanodine Receptor 1 Related...
Devon Parks doesn’t just believe in miracles — she says she has proof they exist. Parks’ 3-year-old son, Isaiah, was born with the rare disease called DiGeorge Syndrome, but he has outlived his 2-year life expectancy. He recently was named 2009 Child of the Year by the Pennsylvania Elks Home...
PASADENA, Calif. – 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...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim 48-week liver biopsy results from the AROAAT2002 study, an open-label Phase 2 clinical study of ARO-AAT, the company’s second generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited (“Takeda”) as a treatment for the rare...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim results from the first two cohorts of AROHIF21001, a Phase 1b dose-finding clinical study of ARO-HIF2, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma (ccRCC). ARO-HIF2...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim results from the first two cohorts of AROHIF21001, a Phase 1b dose-finding clinical study of ARO-HIF2, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma (ccRCC). ARO-HIF2 is...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today notified regulatory agencies, institutional review boards, and investigators that effective immediately it is voluntarily pausing AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF),...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has dosed the first patients in the PALISADE study, a Phase 3 clinical study to evaluate the efficacy and safety of ARO-APOC3 in adults with familial chylomicronemia syndrome (FCS). ARO-APOC3 is the company’s investigational RNA interference (RNAi) therapeutic...
VALENCIA, Spain — ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1)....