Vertex may have misjudged the data that regulators will want to see before approving one of the company’s most prized pipeline drugs. On Wednesday, the Food and Drug Administration held a workshop on developing medicines for alpha-1 antitrypsin deficiency, a genetic disorder where lack of a protein called AAT causes
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 of
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, Kamada
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 associated
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 outcomes
24th April 2018 As previously disclosed, in July 2017, Kamada submitted a proposed Phase 3 study protocol for Inhaled AAT for treatment of AATD to the FDA. In response, in August 2017, the FDA issued a letter to the Company stating the agency’s continued concerns and questions regarding the safety
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.
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 liver
Alpha-1 Awareness is dedicated to contributing a minimum of 10% of its funds raised towards research to find a cure for this condition. In 2018, we went above and beyond this target, donating £25,000 to the patron of the Charity, Prof Lomas, Vice-Provost of Health at University College London (UCL).
22nd December 2018 September 20-21, 2019 The meeting is focused on therapy of the liver disease in α1-antitrypsin deficiency (AATD) and is addressed to clinicians and basic science researchers. The conference will provide a comprehensive overview and detailed lectures on cutting-edge translational and clinical research and will foster the exchange of