Ionis Pharmaceuticals, Inc.는 상업화 단계의 생명공학 기업으로, 미국에서 RNA 표적 치료제를 제공합니다. 이 회사는 가족성 유미지방혈증 증후군(FCS) 및 급성 췌장염 성인의 중성지방 수치를 낮추는 TRYNGOLZA, 성인의 유전성 혈관부종 발작 예방을 위한 DAWNZERA, 성인의 유전성 트랜스티레틴 매개 아밀로이드증(ATTRv-PN)으로 인한 다발성 신경병증 치료제 WAINUA, 그리고 척수성 근위축증(SMA) 소아 및 성인 환자를 위한 SPINRAZA를 제공합니다. 또한, 근위축성 측삭
Ionis Pharmaceuticals, Inc., a commercial-stage biotechnology company, provides RNA-targeted medicines in the United States. The company offers TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS) and acute pancreatitis; DAWNZERA for prophylaxis to prevent attacks of hereditary angioedema in adults; WAINUA for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults; and SPINRAZA for pediatric and adult patients with spinal muscular atrophy (SMA). It also provides QALSODY for the treatment of Amyotrophic Lateral Sclerosis (ALS); TEGSEDI for the treatment of ATTRv-PN in adults; and WAYLIVRA for treatment for FCS and familial partial lipodystrophy. It also develops products under Phase 3 clinical trials, such as Olezarsen for patients with hypertriglyceridemia (SHTG) and cardiovascular disease (CVD); and Zilganerse, a potential treatment for people with genetically confirmed Alexander disease, as well as ION582 which is in Phase 3 clinical trial for the potential treatment of AS, a rare genetic neurological disease. In addition, the company develops Eplontersen to degrade mutant and wild-type TTR mRNA through binding to the TTR mRNA; Pelacarsen to inhibit the production of apolipoprotein(a) in the liver to offer a direct approach for reducing lipoprotein(a); Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus; Sefaxersen to reduce the production of complement factor B and lower activation of the alternative complement pathway; and Ulefnersen to reduce the production of the fused in sarcoma, as well as other mid-stage pipeline investigational medicines. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with GSK, AstraZeneca, Novartis, and Roche, as well as with Metagenomi. The company was incorporated in 1989 and is headquartered in Carlsbad, California.