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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.
Vertex Pharmaceuticals Incorporated는 미국, 유럽 및 전 세계에서 생명공학 기업으로 운영됩니다. 이 회사는 낭포성 섬유증 (CF), 겸상 적혈구 빈혈증 (SCD), 수혈 의존성 베타 지중해 빈혈 (TDT), 급성 통증 등 전문 분야에 집중하여 중증 질환 환자들을 위한 혁신적인 의약품을 제공합니다. 이 회사는 최소 하나의 F508del 돌연변이를 가진 2세 이상 CF 환자를 위한 TRIKAFTA/KAFTRIO; 6세 이상 CF 환자 치료를 위한 ALYFTREK; 6세 이상 CF 환자 치료를 위한 SYMDEKO/SYMKEVI; 1세 이상 CF 환자를 위한 ORKAMBI; 그리고 이바카프토르 관련 CF를 앓는 생후 1개월 이상 환자 치료를 위한 KALYDECO를 시판하고 있습니다. 이 회사는 또한 SCD 및 TDT 치료를 위한 CASGEVY; 성인의 급성 통증 치료를 위한 JOURNAVX; CF의 근본 원인을 치료하도록 설계된 CFTR mRNA 치료제인 VX-522 (현재 1/2상 임상 시험 중); APOL1 매개 신장 질환 치료를 위한 inaxaplin (현재 단일 2상 임상 시험 중); 1형 당뇨병 치료를 위한 VX-264; 1형 근긴장성 이영양증 치료를 위한 VX-670; 그리고 상염색체 우성 다낭성 신장 질환 치료를 위한 저분자 교정제인 VX-407을 개발하고 있습니다. 이 회사는 주로 전문 약국 및 유통업체, 도매업체, 소매 약국, 병원 및 의원에 제품을 판매합니다. Vertex Pharmaceuticals Incorporated는 1989년에 설립되었으며, 매사추세츠주 보스턴에 본사를 두고 있습니다.