LEXINGTON, Mass.–(BUSINESS WIRE)–Dec. 17, 2019– Dicerna™ Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the European Commission (EC) has granted orphan drug designation to Dicerna’s DCR-A1AT for the treatment of congenital alpha-1 antitrypsin (A1AT) deficiency based on a positive opinion from theContinue Reading

Rehovot, Israel, December 16, 2019 — Kamada Ltd. (NASDAQ & TASE: KMDA), a plasma-derived protein therapeutics company, announced today that the first patient has been randomized in Europe into its pivotal Phase 3 InnovAATe clinical trial evaluating the safety and efficacy of the Company’s proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy forContinue Reading

SAN DIEGO, September 4, 2019 /PRNewswire/ — Inhibrx, Inc. (Inhibrx), a clinical-stage biotechnology company focused on developing a broad pipeline of novel biologic therapeutic candidates, announced today the enrollment of the first patient in a Phase 1 clinical trial of INBRX-101 (NCT03815396). INBRX-101 is an Fc-fusion protein-based therapeutic candidate comprised ofContinue Reading

REHOVOT, Israel, Sept. 03, 2019 (GLOBE NEWSWIRE) — Kamada Ltd. (NASDAQ and TASE: KMDA), a plasma-derived protein therapeutics company, announced today the extension of its strategic supply agreement with Takeda for GLASSIA® [Alpha1-Proteinase Inhibitor (Human)].  Kamada will now continue to produce GLASSIA for Takeda through 2021.  Based on the extended agreement, KamadaContinue Reading

PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has dosed the first patient in SEQUOIA (AROAAT2001), a potentially pivotal Phase 2/3 clinical study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associatedContinue Reading

Rationale: Augmentation therapy with intravenous AAT (alpha-1 antitrypsin) is the only specific therapy for individuals with pulmonary disease from AAT deficiency (AATD). The recommended standard dose (SD; 60 mg/kg/wk) elevates AAT trough serum levels to around 50% of normal; however, outside of slowing emphysema progression, its effects in other clinical outcomesContinue Reading

Apr 12, 2019 PASADENA, Calif. –(BUSINESS WIRE)–Apr. 12, 2019– Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today presented preclinical data at The International Liver Congress™ 2019 (ILC), the annual meeting of the European Association for the Study of the Liver (EASL), demonstrating that sustained PASADENA, Calif.–(BUSINESS WIRE)–Apr. 12, 2019– Arrowhead Pharmaceuticals Inc.Continue Reading