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

28th March 2019   Researchers from Technical University of Denmark have produced CHO or mammalian cell lines which can produce 1, 2 g/L recombinant Alpha-1-antitrypsin protein. This breakthrough research can lead to better treatment of Alpha-, a genetic disorder. Liver produces the protein Alpha-1-antitrypsin. After being secreted by the liverContinue Reading