로켓 파머슈티컬스 (RCKT) 주가 전망: 목표주가, 배당, 최신 뉴스

Rocket Pharmaceuticals, Inc. , together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing, manufacturing, and selling of genetic therapies for rare and devastating diseases in the United States.

It develops in vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage.

It also develops ex vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301).

It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red de Enfermedades Raras, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

Rocket Pharmaceuticals, Inc.는 자회사들과 함께 미국에서 희귀하고 치명적인 질환을 대상으로 유전자 치료제의 개발·제조·판매에 주력하는 임상 후기 단계의 바이오테크 기업입니다.

in vivo adeno-associated viral (AAV) 프로그램으로는 Danon disease (DD)를 표적하는 RP-A501(Phase 2), Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)를 표적하는 RP-A601(Phase 1), 그리고 BAG3 Dilated Cardiomyopathy (BAG3-DCM, 전임상) 등이 있습니다. Danon disease는 다기관 리소좀 관련 질환으로 심부전에 따른 조기 사망을 초래하고, PKP2-ACM은 진행성 심근 소실·우심실 확장·이형성·섬유지방성 대체 및 부정맥·돌연사 위험이 높은 유전성 심장질환이며, BAG3-DCM은 심장벽이 점진적으로 얇아지는 확장성 심근병증입니다.

ex vivo lentiviral (LV) 프로그램으로는 Leukocyte Adhesion Deficiency-I (LAD-I, RP-L201), Fanconi Anemia (FA, RP-L102), Pyruvate Kinase Deficiency (PKD, RP-L301) 등을 개발 중으로, 각각 면역 기능 이상, 골수 결함에 따른 혈구 생성 저하 또는 불량 혈구 생성, 만성 비구형 용혈성 빈혈을 표적합니다.

Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red de Enfermedades Raras, Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz, UCL Business PLC, The Regents of the University of California 및 Temple University와 라이선스 계약을 보유하고 있습니다. Rocket Pharmaceuticals, Inc.는 1999년 설립되었으며 본사는 Cranbury, New Jersey에 있습니다.

• 주소: 9 Cedarbrook Drive, Cranbury, NJ 08512, United States

• 전화번호: 609 659 8001

• 웹사이트: https://www.rocketpharma.com

• 섹터: Healthcare

• 산업: Biotechnology

• 직원 수: 202