Vertex Pharmaceuticals Incorporated (VRTX)

業務介紹

Vertex Pharmaceuticals Incorporated是一家生物科技公司，致力於開發和商業化治療囊性纖維化病的療法。該公司將SYMDEKO/SYMKEVI、ORKAMBI和KALYDECO市場化，用於治療囊性纖維化病患者的囊性纖維化病膜通導調節因子基因中具有特定突變的患者；並將TRIKAFTA用於治療6歲或以上至少有一個F508del突變的囊性纖維化病患者。其產品管線還包括用於AAT缺乏症治療的VX-864，目前正在進行2期臨床試驗；用於治療APOL1介導的局灶性節段性腎小球硬化症（FSGS）和其他嚴重腎臟疾病的VX-147，目前正在進行2期臨床試驗；用於治療1型糖尿病的VX-880，目前正在進行1/2期臨床試驗；用於急性、神經病理性、肌骨骼疼痛治療的NaV1.8抑制劑VX-548，目前正在進行2期臨床試驗；以及用於治療嚴重SCD和TDT的CTX001，目前正在進行3期臨床試驗。該公司主要將其產品銷售給美國的專科藥房和專科分銷商，以及國際上的專科分銷商、零售連鎖店、醫院和診所。它與Affinia Therapeutics, Inc.、Arbor Biotechnologies, Inc.、CRISPR Therapeutics AG.、Kymera Therapeutics, Inc.、Mammoth Biosciences, Inc.、Moderna, Inc.、Obsidian Therapeutics, Inc.和Skyhawk Therapeutics, Inc.以及Ribometrix, Inc.、Genomics plc、Merck KGaA、Darmstadt, Germany和X-Chem, Inc.等公司合作。Vertex Pharmaceuticals Incorporated成立於1989年，總部位於麻薩諸塞州波士頓。

Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene; and TRIKAFTA for the treatment of patients with CF 6 years of age or older who have at least one F508del mutation. Its pipeline includes VX-864 for the treatment of AAT deficiency, which is in Phase 2 clinical trial; VX-147 for the treatment of APOL1-mediated focal segmental glomerulosclerosis, or FSGS, and other serious kidney diseases which is in Phase 2 clinical trial; VX- 880, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-548, a NaV1.8 inhibitor for treatments of acute, neuropathic, musculoskeletal pain which is in Phase 2 clinical trial; and CTX001 for the treatment severe SCD and TDT which is in Phase 3 clinical trial. The company sells its products primarily to specialty pharmacy and specialty distributors in the United States, as well as specialty distributors and retail chains, and hospitals and clinics internationally. It has collaborations with Affinia Therapeutics, Inc.; Arbor Biotechnologies, Inc.; CRISPR Therapeutics AG.; Kymera Therapeutics, Inc.; Mammoth Biosciences, Inc.; Moderna, Inc.; Obsidian Therapeutics, Inc.; and Skyhawk Therapeutics, Inc.; as well as Ribometrix, Inc.; Genomics plc; Merck KGaA; Darmstadt, Germany, and X-Chem, Inc. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.

公司網址

https://www.vrtx.com