Vertex Pharmaceuticals Incorporated (VRTX)

業務介紹

頂點製藥公司是一家生物技術公司,從事開發和商業化治療囊性纖維化的療法。該公司銷售SYMDEKO/SYMKEVI、ORKAMBI和KALYDECO,用於治療具有囊性纖維化跨膜傳導調節基因特定突變的患者;以及TRIKAFTA,用於治療6歲及以上至少有一個F508del突變的囊性纖維化患者。其管線包括:用於治療AAT缺乏症的VX-864(處於2期臨床試驗階段)、用於治療APOL1介導的局灶性節段性腎小球硬化症(FSGS)和其他嚴重腎病的VX-147(處於2期臨床試驗階段)、用於治療1型糖尿病的VX-880(處於1/2期臨床試驗階段)、用於治療急性、神經性、肌肉骨骼疼痛的VX-548(處於2期臨床試驗階段),以及用於治療嚴重的鐮刀型細胞貧血和地中海貧血的CTX001(處於3期臨床試驗階段)。該公司主要在美國向專科藥房和專科分銷商銷售其產品,並在國際上向專科分銷商、零售連鎖店以及醫院和診所銷售。它與多家公司有合作關係,包括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.。Vertex Pharmaceuticals Incorporated成立於1989年,總部位於馬薩諸塞州波士頓。



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