IONIS PHARMACEUTICALS INC

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.

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