Interim Review of Safety, Tolerability, and Efficacy From a First-In-Human Phase 1/2a Clinical Study of ICM-203, an Intra-Articular, AAV Gene Therapy for Osteoarthritis

被引：0
|
作者：
Heald, Alison E. [1 ,2 ]
Solomon, Bogdan [3 ]
Page, Richard [4 ,5 ]
Ahn, Yoenhee [1 ]
Yum, Young Na [1 ]
Myung, Jayhyuk [1 ]
Collins, Jamie E. [6 ]
Guermazi, Ali [7 ]
Kim, Dae-Won [1 ,8 ]
机构：
[1] ICM Co LTD, Seoul, South Korea
[2] Univ Washington, Sch Med, Seattle, WA USA
[3] Royal Adelaide Hosp, Adelaide, SA 5000, Australia
[4] Univ Hosp Geelong, Barwon Hlth, Geelong, Vic, Australia
[5] Deakin Univ, Geelong, Vic, Australia
[6] Brigham & Womens Hosp, 75 Francis St, Boston, MA 02115 USA
[7] Boston Univ, Sch Med, Boston, MA 02118 USA
[8] Yonsei Univ, Seoul, South Korea
来源：
MOLECULAR THERAPY | 2024年 / 32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：
146
引用
收藏
页码：80 / 80
页数：1
相关论文
共 42 条
  • [21] Preliminary Results from the Phase 1/2 GuardOne Trial: A First-in-Human Study of Ex Vivo Lentiviral Gene Therapy (AVR-RD-02) in People with Gaucher Disease Type 1
    Jacobsen, L. K.
    Trame, M. N.
    Golipour, A.
    Pfeifer, R.
    Khan, A.
    Mason, C.
    MOLECULAR THERAPY, 2021, 29 (04) : 340 - 340
  • [22] Updated interim safety, biomarker, and efficacy data from Imagine-1: A phase 1/2 open-label, multicenter study to assess the safety, tolerability, and efficacy of a single dose, intra-cisterna magna (ICM) administration of PBGM01 in subjects with type I (early onset) and type IIA (late onset) infantile GM1 gangliosidosis (GM1)
    Jarnes, Jeanine R.
    Hastings, Caroline A.
    Ficicioglu, Can H.
    Day-Salvatore, Debra-Lynn
    Giugliani, Roberto
    Baruteau, Julien
    Whitley, Chester B.
    Inbar-Feigenberg, Michal
    Bernard, Genevieve
    Ezgu, Fatih S.
    Ni, Yan G.
    Miller, Michelle
    Gelb, Michael H.
    Nagilla, Pruthvi
    Johnstone, Rose
    Elsasser, Patricia
    Cunningham, Elizabeth
    Ballard, Victoria
    Haws, Thomas F.
    Forman, Mark S.
    Weinstein, David A.
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 68 - 68
  • [23] Gene Therapy for Crigler-Najjar Syndrome with AT342, a Liver-Targeted AAV8-UGT1A1 Vector - Preliminary Safety and Efficacy Results from a Phase 1/2 Study (VALENS)
    Prasad, Suyash
    Strauss, Kevin A.
    McKiernan, Patrick
    Mazariegos, George
    Ovchinsky, Nadia
    Dhawan, Anil
    Ranganathan, Sarangarajan
    Deheragoda, Maesha
    Lawlor, Michael W.
    Noursalehi, Mojtaba
    Kennedy, William P.
    MOLECULAR THERAPY, 2018, 26 (05) : 251 - 251
  • [24] SAFETY, CLINICAL AND IMAGING OUTCOMES OF A NOVEL, INTRA-ARTICULAR, INJECTABLE, WNT INHIBITOR (SM04690) IN THE TREATMENT OF OSTEOARTHRITIS OF THE KNEE: EXPLORATORY ANALYSIS OF RESULTS FROM A 24 WEEK, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 1 STUDY
    Yazici, Y.
    McAlindon, T. E.
    Fleischmann, R. M.
    Gibofsky, A.
    Lane, N. E.
    Kivitz, A. J.
    Majumdar, S.
    Strand, V.
    Swearingen, C. J.
    DiFrancesco, A.
    Tambiah, J.
    Hood, J.
    Hochberg, M. C.
    ANNALS OF THE RHEUMATIC DISEASES, 2016, 75 : 826 - 826
  • [25] Long-term, sustained efficacy and safety from a phase 1/2 clinical trial of an AAV8-mediated liver-directed gene therapy in adults with glycogen storage disease type Ia
    Pico, M. L. Couce
    Mitchell, J.
    Ahmad, A.
    Olmos, M.
    Derks, T. G.
    Riba-Wolman, R.
    Weinstein, D. A.
    Rodriguez-Buritica, D. F.
    Lee, C.
    Valayannopoulos, V.
    Crombez, E.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A28 - A28
  • [26] A phase 1/2a, multicenter, open-label, first-in-human study to assess the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of DB-1311 (a B7-H3-targeting ADC) in patients with advanced/metastatic solid tumors
    Cheng, Ying
    Lisberg, Aaron
    Lemech, Charlotte
    Abed, Afaf
    Chaudhry, Arvind
    Schmidt, Andrew
    Lu, Xin
    Zhu, Zhongyuan
    Gu, Wei
    Qiu, Yang
    Shi, Rong
    Zhang, Bin
    CANCER RESEARCH, 2024, 84 (07)
  • [27] The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease
    Jacobsen, Leslie
    Kerner, Julie A.
    Ciotti, Rachel
    Golipour, Azadeh
    Yang, Josie
    Pfeifer, Richard
    Khan, Aneal
    Mason, Chris
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S51 - S52
  • [28] Interim Safety, Biomarker, and Efficacy Data From Imagine-1: A Phase 1/2 Open-label, Multicenter Study to Assess the Safety, Tolerability, and Efficacy of a Single Dose, ICM Administration of PBGM01 in Subjects with Type I (Early Onset) and 10. Type IIa (Late Onset) Infantile GM1 Gangliosidosis (GM1)
    Weinstein, David A.
    Hastings, Caroline A.
    Day-Salvatore, Debra-Lynn
    Ficicioglu, Can
    Whitley, Chester B.
    Inbar-Feigenberg, Michal
    Bernard, Genevieve
    Giugliani, Roberto
    Baruteau, Julien
    Ezgu, Fatih S.
    Jarnes, Jeanine R.
    Ni, Yan G.
    Nagilla, Pruthvi
    Ballard, Victoria L.
    Haws, Thomas F.
    Gelb, Michael H.
    Forman, Mark S.
    MOLECULAR THERAPY, 2022, 30 (05) : 5 - 6
  • [29] Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia a
    Pasi, K. John
    Rangarajan, Savita
    Kim, Benjamin
    Lester, Will
    Perry, David
    Madan, Bella
    Tavakkoli, Fatemeh
    Yang, Ke
    Pierce, Glenn F.
    Wong, Wing Yen
    BLOOD, 2017, 130
  • [30] A phase 1/2a, multicenter, open-label, non-randomized first-in-human study to assess the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of DB-1310 (a HER3-targeting ADC) in patients with advanced/metastatic solid tumors
    Lisberg, Aaron
    Lu, Shun
    Starodub, Alexander
    Amin, Harshad
    Rotow, Julia
    Wu, Lin
    Spira, Alexander
    Hamilton, Erika
    Shen, Weidi
    Liu, Liming
    Zhu, Zhongyuan
    Gu, Wei
    Qiu, Yang
    Shi, Rong
    Liu, Shengxue
    CANCER RESEARCH, 2024, 84 (07)
12345