Design of a Phase 1/2 Study to Evaluate the Safety and Efficacy of an AAV9-Based Gene Therapy in Infants with Type 2 Gaucher Disease

被引：0
|
作者：
Youssef, Eriene [1 ]
Tamburri, Paul [1 ]
Beckerman, Yael [1 ]
Lowrey, Mark [1 ]
Mahoney, Erin [1 ]
McNamara, Ilan [1 ]
Sevigny, Jeffrey [1 ]
机构：
[1] Prevail Therapeut, New York, NY USA
来源：
MOLECULAR THERAPY | 2020年 / 28卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
623
引用
收藏
页码：276 / 277
页数：2
相关论文
共 50 条
  • [1] Phase 1/2 study update of an AAV9-based gene therapy for Gaucher disease type 2 (PROVIDE trial)
    Neuhaus, Sarah
    Lewis, Travis B.
    Tamburri, Paul A.
    Whitley, Chester B.
    Rajan, Deepa
    Gallagher, Renata C.
    Jones, Simon
    Donald, Aimee
    Escolar, Maria L.
    Beckerman, Yael
    Mahoney, Erin
    Hatch, Daniel A.
    Shaughnessy, Lee
    Sondergaard, Patricia
    Uspenskaya, Olga
    Sevigny, Jeffrey
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 93 - 93
  • [2] Design and outcome measures for a phase 1/2 dose-finding study to evaluate systemic administration of an AAV9-based gene therapy for peripheral manifestations of Gaucher disease
    Neuhaus, Sarah
    Tamburri, Paul A.
    Weaver, Michael A.
    Booth, Jeff
    Mahoney, Erin
    Shaughnessy, Lee
    Sondergaard, Patricia
    Hatch, Daniel A.
    Lewis, Travis B.
    Uspenskaya, Olga
    Sevigny, Jeffrey
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 94 - 94
  • [3] Safety of intracisternal administration of AAV9 based gene therapy: Case series of PR001 in infants with type 2 Gaucher disease
    Zibly, Zion
    Anikster, Yair
    Revel-Vilk, Shoshana
    Zimran, Ari
    Lowrey, Mark
    Youssef, Eriene A.
    Sevigny, Jeffery
    Pukenas, Bryan
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S116 - S116
  • [4] Design of Phase 1/2a Study of AAV9-Based Gene Therapy for Parkinson's Disease with Pathogenic GBA1 Mutations (PROPEL Trial)
    Uspenskaya, Olga
    Mahoney, Erin
    Verselis, Lynne
    Lowrey, Mark
    Velaga, Jenny
    Sevigny, Jeffrey
    NEUROLOGY, 2020, 94 (15)
  • [5] Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease
    Greenberg, Barry
    Taylor, Matthew
    Adler, Eric
    Colan, Steven
    Ricks, David
    Yarabe, Paul
    Battiprolu, Pavan
    Shah, Gaurav
    Patel, Kinnari
    Coggins, Matthew
    Carou-Keenan, Susanna
    Schwartz, Jonathan D.
    Rossano, Joseph W.
    NEW ENGLAND JOURNAL OF MEDICINE, 2024,
  • [6] Design of an AAV9-Based Gene Therapy Vector Able to Evade Neutralization by a Novel, Potent α-AAV9 Neutralizing Antibody
    Giles, April R.
    Govindasamy, Lakshmanan
    Somanathan, Suryanarayan
    Wilson, James M.
    MOLECULAR THERAPY, 2017, 25 (05) : 338 - 338
  • [7] Design of GALILEO-1, a phase 1/2 safety and efficacy study of FLT201 in adult patients with Gaucher disease type 1
    Hughes, Derralynn A.
    Barton, Sharon
    Chan, Jane
    Sherry, Nicole
    Long, Alison
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S57 - S58
  • [8] Full correction of neurologic and somatic lysosomal pathology in Mucopolysaccharidosis type IIIB by AAV9-based gene therapy
    Haurigot, Virginia
    Ribera, Albert
    Marco, Sara
    Garcia, Miguel
    Motas, Sandra
    Villacampa, Pilar
    Sanchez, Victor
    Maggioni, Luca
    Roca, Carles
    Leon, Xavier
    Molas, Maria
    Munoz, Sergio
    Ruberte, Jesus
    Mingozzi, Federico
    Moll, Xavier
    Anor, Sonia
    Bosch, Fatima
    HUMAN GENE THERAPY, 2014, 25 (11) : A37 - A37
  • [9] Development of an AAV-GBA Gene Therapy for the Treatment of Type 1 Gaucher Disease
    Cheng, Ting-Wen
    Souza, David
    Tabet, Ricardos
    Wright, Katherine
    Brodfuehrer, Joanne
    Keyes, Lisa
    Rao-Dayton, Sheila
    Lewis, Patricia
    Sievers, Annette
    McCarty, Douglas M.
    Somanathan, Suryanarayan
    Pan, Clark
    Bell, Robert D.
    MOLECULAR THERAPY, 2021, 29 (04) : 251 - 251
  • [10] Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102, an intravenous AAV9-based gene therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)
    Flanigan, K. M.
    Smith, N. J. C.
    Couce, M. L.
    Truxal, K. V.
    McBride, K. L.
    Simmons, T. R.
    de Castro, M. J.
    Cope, K. L.
    Oreiro, M. T.
    Jaensch, L.
    Fuller, M.
    Ruiz, J.
    NEUROLOGY, 2020, 94 (15)
12345