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...
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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...
PHILADELPHIA, PA — Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it has completed enrollment in its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD). The trial is evaluating safety,...
PHILADELPHIA, PA — Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines in areas of high unmet need in rare muscle diseases, immunology, and oncology, today announced positive topline results from the Phase 1b portion of its study evaluating ABX1100, a novel investigational therapy for...
WALTHAM, Mass. — Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1),...
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),...