HMI-203: Investigational gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome

被引：0

作者：

Patel, Kruti ^{[1
]}

Smith, Laura ^{[1
]}

Gingras, Jacinthe ^{[1
]}

Tzianabos, Alec ^{[1
]}

Schulman, Lindsay ^{[1
]}

Zhivich, Victor ^{[1
]}

Kivaa, Monicah ^{[1
]}

Faulkner, Deiby ^{[1
]}

Sengooba, Arnold ^{[1
]}

Seabrook, Tania ^{[1
]}

Behmoiras, Liana ^{[1
]}

Dollive, Serena ^{[1
]}

Avila, Nancy ^{[1
]}

Zhao, Jingyan ^{[1
]}

White, Yvonne ^{[1
]}

Newman, Jenn ^{[1
]}

Woodcock, Steve ^{[1
]}

Smith, Sean ^{[1
]}

Francone, Omar ^{[1
]}

Seymour, Albert ^{[1
]}

机构：

[1] Homol Med, Bedford, MA USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2021年 / 132卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2020.12.195

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

189

引用

页码：S82 / S82

页数：1

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[1] HMI-203: Gene therapy developmental candidate for mucopolysaccharidosis type II (MPS II), or Hunter syndrome
Patel, Kruti
Smith, Laura
Seabrook, Tania
Tzianabos, Alec
Schulman, Lindsay
Zhivivh, Victor
Kivaa, Monicah
Faulkner, Deiby
Sengooba, Arnold
Behmoiras, Liana
Newman, Jennifer
Dollive, Serena
Avila, Nancy
Zhao, Jingyan
White, Yvonne
Woodcock, Steve
Smith, Sean
Francone, Omar
Gingras, Jacinthe
Seymour, Albert
[J]. MOLECULAR GENETICS AND METABOLISM, 2021, 132 : S147 - S147
[2] Long-Term Expression of HMI-203: Investigational Gene Therapy Candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome
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Patel, Kruti
Gingras, Jacinthe
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Schulman, Lindsay
Zhivich, Victor
Kivaa, Monicah
Faulkner, Deiby
Sengooba, Arnold
Behmoiras, Liana
Seabrook, Tania
Dollive, Serena
Avila, Nancy
Zhao, Jingyan
White, Yvonne
Newman, Jenn
Woodcock, Steve
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Seymour, Albert
[J]. MOLECULAR THERAPY, 2021, 29 (04) : 247 - 247
[3] Summary of nonclinical data for gene therapy developmental candidate HMI-203 for mucopolysaccharidosis type II (MPS II, or Hunter syndrome)
Smith, Laura J.
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Avila, Nancy
Newman, Jennifer
Zhao, Jingyan
Woodcock, Steve
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Smith, Sean
Seymour, Albert
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[J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S113 - S114
[4] Clinical trial design for HMI-203 investigational gene therapy for mucopolysaccharidosis type II (MPS II) informed by cross-correction potential and KOL input
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[J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S46 - S46
[5] Blood-brain-barrier crossing leads to long term efficacy in the CNS of HMI-203: Gene therapy development candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome
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Tzianabos, A.
Seabrook, T.
Kivaa, M.
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Avila, N.
Zhao, J.
White, Y.
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Woodcock, S.
Smith, S.
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[J]. HUMAN GENE THERAPY, 2021, 32 (19-20) : A52 - A52
[6] Patient and physician perspectives inform clinical trial design for a single intravenous dose of HMI-203, a gene therapy candidate for adults with mucopolysaccharidosis type II (MPS II, Hunter syndrome)
Haroldson, Jeffrey
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[J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S54 - S55
[7] Investigational intrathecal (IT) enzyme replacement therapy for the severe form of Hunter syndrome (mucopolysaccharidosis type II, MPS II)
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[J]. MOLECULAR GENETICS AND METABOLISM, 2014, 111 (02) : S79 - S79
[8] Presentation and treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome)
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Yabe, Hiromasa
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Orii, Kenji E.
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[J]. EXPERT OPINION ON ORPHAN DRUGS, 2017, 5 (04): : 295 - 307
[9] Clinical benefit of enzyme replacement therapy (ERT) in mucopolysaccharidosis II (MPS II, Hunter syndrome)
Muenzer, J.
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[J]. JOURNAL OF INHERITED METABOLIC DISEASE, 2006, 29 : 28 - 28
[10] Clinical benefit of enzyme replacement therapy (ERT) in mucopolysaccharidosis II (MPS II, Hunter syndrome)
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Vellodi, A.
Martin, R.
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Puga, A.
Ulbrich, B.
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Wendt, S.
[J]. MOLECULAR GENETICS AND METABOLISM, 2007, 92 (04) : S18 - S18

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