Vertex Pharmaceuticals Incorporated는 미국, 유럽 및 국제적으로 생명공학 기업으로 운영됩니다. 이 회사는 낭포성 섬유증(CF), 겸상 적혈구 빈혈증(SCD), 수혈 의존성 베타 지중해빈혈(TDT) 및 급성 통증과 같은 전문 시장에 중점을 두고 중증 질환을 앓는 사람들을 위한 의약품을 제공합니다. 주요 제품으로는 2세 이상 CF 환자를 위한 TRIKAFTA/KAFTRIO, 6세 이상 CF 환자를 위한 ALYFTREK, SYMDEKO/SYMKEVI, 1세 이상 CF 환자를 위한 ORKAMBI, 1개월 이상 CF 환자를 위한 KALYDECO가 있습니다.
이 회사는 SCD 및 TDT 치료를 위한 CASGEVY, 성인의 급성 통증 치료를 위한 JOURNAVX, CF의 근본 원인을 치료하기 위한 CFTR mRNA 치료제 VX-522, APOL1 매개 신장 질환 치료를 위한 inaxaplin, 제1형 당뇨병 치료를 위한 VX-264 등을 개발하고 있습니다. 또한 희귀 내분비 질환 및 내분비 관련 종양을 위한 새로운 치료법의 발견, 개발 및 상업화에 중점을 둔 임상 단계 제약 회사로도 운영됩니다. 제품은 주로 전문 약국 및 유통업체, 도매업체, 소매 약국, 병원 및 클리닉에 판매됩니다.
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Vertex Pharmaceuticals Incorporated operates as a biotechnology company in the United States, Europe, and internationally. The company offers transformative medicines for people with serious diseases with a focus on specialty markets, such as cystic fibrosis (CF), sickle cell disease (SCD), transfusion dependent beta thalassemia (TDT), and acute pain. It markets TRIKAFTA/KAFTRIO for people with CF with at least one F508del mutation for 2 years of age and older; ALYFTREK for the treatment for people with CF 6 years of age and older; SYMDEKO/SYMKEVI for treatment of patients with CF 6 years of age and older; ORKAMBI for CF patients 1 year or older; and KALYDECO for the treatment of patients with 1 month or older who have CF with ivacaftor. The company also develops CASGEVY for the treatment of SCD and TDT; JOURNAVX for the treatment of acute pain in adults; VX-522, a CFTR mRNA therapeutic designed to treat the underlying cause of CF, which is in Phase 1/2 clinical trial; inaxaplin for the treatment of APOL1-mediated kidney disease, which is in single Phase 2 trial; VX-264 for treating Type 1 Diabetes; VX-670 for the treatment of myotonic dystrophy type 1; and VX-407, a small molecule corrector for the treatment of autosomal dominant polycystic kidney disease. In addition, the company also operates as a clinical-stage pharmaceutical company, that focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company sells its products primarily to specialty pharmacy and distributors, wholesalers, retail pharmacies, hospitals, and clinics. Vertex Pharmaceuticals Incorporated has a strategic collaboration with AbCellera Biologics Inc. to research, develop, manufacture, and commercialize multispecific T-cell engagers (TCEs) for autoimmune diseases and other conditions. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.
