Transpher A, an Open-Label, Multicenter, Single-Dose, Dose-Escalation, Phase 1/2 Clinical Trial of Gene Transfer of ABO-102 in Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA): Safety, Tolerability, Biopotency and Neurocognitive Data

被引：0

作者：

Flanigan, Kevin ^{[1
]}

Smith, Nicholas J. C. ^{[2
]}

Luz Couce, Maria ^{[3
]}

Truxal, Kristen V. ^{[1
]}

McBride, Kim L. ^{[1
]}

Jose de Castro, Maria ^{[3
]}

McNally, Kelly A. ^{[1
]}

Simmons, Tabatha R. ^{[1
]}

Cope, Krista L. ^{[1
]}

Pertini, Mark ^{[4
]}

Jaensch, Louise ^{[5
]}

Lopez, Luisa ^{[6
]}

Tajes, Maria ^{[3
]}

Oreiro, Maria T. ^{[3
]}

Chans, Roi ^{[3
]}

Fuller, Maria ^{[5
]}

Paneda, Astrid ^{[7
]}

Ruiz, Juan ^{[7
]}

机构：

[1] Nationwide Childrens Hosp, Columbus, OH USA

[2] Womens & Childrens Hosp, Dept Neurol & Clin Neurophysiol, Adelaide, SA, Australia

[3] Hosp Clin Univ, Santiago De Compostela, Spain

[4] Womens & Childrens Hosp, Adelaide, SA, Australia

[5] Womens & Childrens Hosp, Adelaide, SA, Australia

[6] Clin Neurita, Lugo, Spain

[7] Abeona Therapeut, Madrid, Spain

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：

524

引用

页码：231 / 231

页数：1

共 50 条

[1] Safety, tolerability, biopotency and neurocognitive data of ABO-102 in Transpher A, an open-label, multicenter, single-dose, dose-escalation, Phase 1/2 Clinical Trial in Sanfilippo Syndrome type A (Mucopolysaccharidosis IIIA
de Castro, M. J.
Flanigan, K. M.
Smith, N. J. C.
Couce, M. L.
Truxal, K. V.
McBride, K. L.
Simmons, T. R.
McNally, K. A.
Oreiro, M. T.
Kunkler, K. L.
Jaensch, L.
Fuller, M.
Ruiz, J.
HUMAN GENE THERAPY, 2019, 30 (11) : A15 - A15
[2] Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)
Flanigan, K.
Smith, N.
Couce, M.
Escolar, M.
Truxal, K.
McBride, K.
de Castro, M.
Fuller, M.
Paneda, A.
Ruiz, J.
ANNALS OF NEUROLOGY, 2021, 90 : S85 - S85
[3] Updated results of Transpher A: multicenter, single-dose, phase 1/2 clinical trial of ABO-102 for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
Flanigan, Kevin
Smith, Nicholas
Couce, Maria
Escolar, Maria
Truxal, Kristen
McBride, Kim
de Castro, Maria
Fuller, Maria
Paneda, Astrid
Ruiz, Juan
MOLECULAR GENETICS AND METABOLISM, 2021, 132 : S133 - S134
[4] Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)
Flanigan, Kevin M.
Smith, Nicholas J. C.
Couce, Maria L.
Escolar, Maria
Truxal, Kristen V.
McBride, Kim L.
de Castro, Maria J.
Fuller, Maria
Paneda, Astrid
Ruiz, Juan
MOLECULAR THERAPY, 2021, 29 (04) : 104 - 105
[5] Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)
Flanigan, Kevin M.
Smith, Nicholas J. C.
Luz Couce, Maria
Truxal, Kristen V.
McBride, Kim L.
Simmons, Tabatha
Jose de Castro, Maria
Cope, Krista
Teresa Oreiro, Maria
Jaensch, Louise
Fuller, Maria
Ruiz, Juan
MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S56 - S57
[6] Updated results of Transpher A, a multicenter, single-dose, Phase 1/2 clinical trial of ABO-102 investigational gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
Flanigan, K. M.
Smith, N. J. C.
Couce, M. L.
Escolar, M.
Truxal, K. V.
McBride, K. L.
de Castro, M. J.
Fuller, M.
Paneda, A.
Ruiz, J.
del Campo, A. B.
Grachev, I. D.
NEUROLOGY, 2022, 98 (18)
[7] Transpher B, an Open-Label, Multicenter, Single-Dose, Dose-Escalation, Phase 1/2 Clinical Trial of Gene Transfer of ABO-101 in Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)
de Castro, Maria J.
Flanigan, Kevin
Heron, Benedicte
Couce, Maria L.
Truxal, Kristen V.
McBride, Kim L.
Ravelli, Claudia
Simmons, Tabatha R.
McNally, Kelly A.
Rinaldi, Federica
Giraudat, Kim
Lopez, Luisa
Tajes, Maria
Oreiro, Maria T.
Chans, Roi
Fuller, Maria
del Campo, Ana B.
Ruiz, Juan
MOLECULAR THERAPY, 2020, 28 (04) : 232 - 233
[8] Updated Interim Results of Transpher A, a Multicenter, Single-Dose, Pivotal Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)
Flanigan, K. M.
Smith, N. J. C.
Couce, M. L.
Rajan, D. S.
Truxal, K. V.
McBride, K. L.
de Castro, M. J.
Fuller, M.
Taylor, J.
del Campo, A. B.
Grachev, I. D.
MOLECULAR THERAPY, 2022, 30 (04) : 221 - 221
[9] 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
[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)

← 1 2 3 4 5 →