Orchard Therapeutics Sees Positive Results from Stem-Cell Gene Therapies
Orchard Therapeutics Sees Positive Results from Stem-Cell Gene Therapies
DJ Orchard Therapeutics Sees Positive Results from Stem-Cell Gene Therapies
By Chris Wack
Orchard Therapeutics PLC said it saw positive results from new data from several of its hematopoietic stem cell gene therapies in development for neurodegenerative disorders, including multiple clinical outcomes for OTL-203 in MPS-I.
The company on Tuesday said patients with the Hurler subtype of MPS-I suffer from a range of symptoms, including neurocognitive impairment and skeletal deformities that lead to impaired growth in the first decade of life.
Eight patients with MPS-IH have been treated with OTL-203 in a proof-of-concept study being conducted at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. As of November, follow-up in all patients has reached at least 12 months. Orchard said a request for parallel scientific advice from the U.S. Food and Drug Administration and European Medicines Agency has been accepted with the intention of seeking feedback in advance of initiating a registrational study for OTL-203 by year-end 2021.
Treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen, the company said. Anti-alpha-L-iduronidase antibodies present prior to gene therapy as a result of ERT weren't seen in any patient within two months following treatment.
Orchard said treatment demonstrated rapid and sustained metabolic correction with all patients achieving supra-physiological IDUA expression in dried blood spot samples at 12 months, a primary efficacy endpoint. Longitudinal growth that was within age-appropriate reference ranges was seen in all patients post-treatment, with follow-up ranging from 9 months to 2 years. Improved range of motion was shown in all patients compared to pre-treatment, with follow-up ranging from 9 months to 1.5 years, it said.
Orchard Therapeutics shares were up 12% to $8.11 in premarket trading.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
February 09, 2021 07:27 ET (12:27 GMT)
Copyright (c) 2021 Dow Jones & Company, Inc.
DJ Orchard Therapeutics Sees Positive Results from Stem-Cell Gene Therapies
DJ Orchard治疗公司看到干细胞基因治疗的积极结果
By Chris Wack
克里斯·瓦克(Chris Wack)著
Orchard Therapeutics PLC said it saw positive results from new data from several of its hematopoietic stem cell gene therapies in development for neurodegenerative disorders, including multiple clinical outcomes for OTL-203 in MPS-I.
Orchard Treeutics PLC表示，其正在开发的治疗神经退行性疾病的几种造血干细胞基因疗法的新数据显示出积极的结果，包括MPS-I中OTL-203的多种临床结果。
The company on Tuesday said patients with the Hurler subtype of MPS-I suffer from a range of symptoms, including neurocognitive impairment and skeletal deformities that lead to impaired growth in the first decade of life.
该公司周二表示，Hurler亚型MPS-I患者患有一系列症状，包括神经认知障碍和骨骼畸形，这些症状会导致出生后第一个十年的生长发育受损。
Eight patients with MPS-IH have been treated with OTL-203 in a proof-of-concept study being conducted at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. As of November, follow-up in all patients has reached at least 12 months. Orchard said a request for parallel scientific advice from the U.S. Food and Drug Administration and European Medicines Agency has been accepted with the intention of seeking feedback in advance of initiating a registrational study for OTL-203 by year-end 2021.
8名MPS-IH患者接受了OTL-203的概念验证研究，该研究在意大利米兰的San Raffaele Telethon基因治疗研究所进行。截至11月，所有患者的随访时间都达到了至少12个月。Orchard说，美国食品和药物管理局(FDA)和欧洲药品管理局(European Medicines Agency)提出的平行科学建议的请求已经被接受，目的是在2021年底之前启动OTL-203的注册研究之前征求反馈。
Treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen, the company said. Anti-alpha-L-iduronidase antibodies present prior to gene therapy as a result of ERT weren't seen in any patient within two months following treatment.
该公司表示，OTL-203的治疗总体上耐受性良好，安全性与所选的调理方案一致。在基因治疗前由于ERT而产生的抗α-L-艾杜糖醛酸酶抗体在治疗后的两个月内没有出现在任何患者身上。
Orchard said treatment demonstrated rapid and sustained metabolic correction with all patients achieving supra-physiological IDUA expression in dried blood spot samples at 12 months, a primary efficacy endpoint. Longitudinal growth that was within age-appropriate reference ranges was seen in all patients post-treatment, with follow-up ranging from 9 months to 2 years. Improved range of motion was shown in all patients compared to pre-treatment, with follow-up ranging from 9 months to 1.5 years, it said.
Orchard说，治疗显示出快速和持续的代谢纠正，所有患者在12个月后在干血斑样本中实现了超生理IDUA表达，这是一个主要的疗效终点。所有患者在治疗后的纵向生长都在年龄合适的参考范围内，随访时间从9个月到2年不等。报告称，与治疗前相比，所有患者的活动范围都有所改善，随访时间从9个月到1.5年不等。
Orchard Therapeutics shares were up 12% to $8.11 in premarket trading.
Orchard Treeutics股价在盘前交易中上涨12%，至8.11美元。
Write to Chris Wack at chris.wack@wsj.com
写信给克里斯·瓦克(chris.wack@wsj.com)
(END) Dow Jones Newswires
(完)道琼通讯社
February 09, 2021 07:27 ET (12:27 GMT)
2021年02月09日07：27(格林尼治标准时间12：27)
Copyright (c) 2021 Dow Jones & Company, Inc.
版权所有(C)2021年道琼斯公司
译文内容由第三方软件翻译。
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