Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

被引：0
|
作者：
George, Lindsey A.
Ragni, Margaret V.
Samelson-Jones, Ben J.
Cuker, Adam
Runoski, Alexa R.
Cole, Grace
Wright, Fraser
Chen, Yifeng
Hui, Daniel J.
Wachtel, Katie
Takefman, Daniel
Couto, Linda B.
Reape, Kathleen Z.
Carr, Marcus E.
Anguela, Xavier M.
High, Katherine A.
机构：
来源：
BLOOD | 2017年 / 130卷
关键词：
D O I：
暂无
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
604
引用
收藏
页数：2
相关论文
共 50 条
  • [31] Continued characterization of the immune response in a phase-1/2 clinical study of valoctocogene roxaparvovec, an AAV5 mediated gene therapy for hemophilia A
    Long, B.
    Kim, B.
    Wong, W. Y.
    Yang, K.
    Vettermann, C.
    Yu, K.
    Lau, K.
    Hardet, R.
    Kuranda, K.
    Veron, P.
    Mingozzi, F.
    Pierce, G. F.
    Schweighardt, B.
    HAEMOPHILIA, 2019, 25 : 59 - 59
  • [32] Design of a dose escalation phase 1/2 trial for a novel combined cell and oncolytic viral therapy
    Contag, Pamela R.
    CANCER RESEARCH, 2018, 78 (13)
  • [33] A PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE-ONSET OTC DEFICIENCY: INTERIM RESULTS FROM COHORT 1
    Harding, Cary O.
    Geberhiwot, Tarekegn
    Aldamiz-Echevarria, Luis
    Luz Couce, Maria
    Khan, Aneal
    Tan, Wen-Hann
    Crombez, Eric
    Diaz, George A.
    MOLECULAR GENETICS AND METABOLISM, 2019, 127 (03) : 240 - 240
  • [34] A PHASE 1/2 CLINICAL TRIAL FOR AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH LATE-ONSET OTC DEFICIENCY: INTERIM RESULTS FROM COHORT 1
    Harding, Cary O.
    Geberhiwot, Tarekegn
    Aldamiz-Echevarria, Luis
    Luz Couce, Maria
    Khan, Anal
    Tan, Wen-Hann
    Crombez, Eric
    Diaz, George A.
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) : 278 - 278
  • [35] Preliminary Efficacy Results from a Phase 1 Trial of Vector-Mediated Gene Transfer for Chronic Intractable Pain
    Fink, David
    Mata, Marina
    Wechuck, James
    Krisky, David
    Wolfe, Darren
    NEUROLOGY, 2011, 76 (09) : A412 - A412
  • [36] Hepatocyte Growth Factor Gene Therapy for Patients With Critical Limb Ischemia: Results of a Phase I Dose-Escalation Trial
    Henry, Timothy D.
    Goldman, JoAnne
    Wang, Yale L.
    Lips, Daniel L.
    Dulas, Daniel
    McMillan, William D.
    Duval, Sue
    Biggs, Thomas A.
    Hirsch, Alan T.
    JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2009, 53 (10) : A40 - A40
  • [37] Completion of Acclaim-1 dose escalation: Recommended Phase 2 dose of quaratusugene ozeplasmid gene therapy and Osimertinib
    Spira, Alexander I.
    Berz, David
    Jotte, Robert M.
    Pachipala, Krishna K.
    Berger, Mark S.
    MOLECULAR CANCER THERAPEUTICS, 2023, 22 (12)
  • [38] AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: interim results from Phase 1 and Phase 2 studies
    Thomas, M.
    Nicholls, K.
    Carnley, B.
    Trame, M. N.
    Mason, C.
    Huertas, P.
    Asatryan, A.
    Escolar, D. M.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A23 - A23
  • [39] PRELIMINARY RESULTS FROM A PHASE 1 DOSE ESCALATION TRIAL OF LIMITED FIELD HYPOFRACTIONATED THORACIC RADIOTHERAPY FOR LIMITED STAGE SMALL CELL LUNG CANCER
    Roa, W. H. Y.
    Chu, Q.
    Butts, C.
    Joy, A.
    Fenton, D.
    Smylie, M.
    Reiman, A.
    Yee, D.
    RADIOTHERAPY AND ONCOLOGY, 2008, 88 : S287 - S287
  • [40] Interim results of Transpher A, a multicentre, single-dose, phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
    Flanigan, Kevin M.
    Smith, Nicholas J. N.
    Couce, Maria L.
    Escolar, Maria
    Truxal, Kristen V.
    McBride, Kim L.
    de Castro, Maria J.
    Fuller, Maria
    Paneda, A.
    Ruiz, J.
    del Campo, A. B.
    Grachev, I.
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S43 - S43
12345