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治療公司看到幹細胞基因治療的積極結果
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，其正在開發的治療神經退行性疾病的幾種造血幹細胞基因療法的新數據顯示出積極的結果，包括MPS-I中OTL-203的多種臨牀結果。
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.
該公司週二表示，Hurler亞型MPS-I患者患有一系列症狀，包括神經認知障礙和骨骼畸形，這些症狀會導致出生後第一個十年的生長髮育受損。
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的概念驗證研究，該研究在意大利米蘭的San Raffaele Telethon基因治療研究所進行。截至11月，所有患者的隨訪時間都達到了至少12個月。Orchard説，美國食品和藥物管理局(FDA)和歐洲藥品管理局(European Medicines Agency)提出的平行科學建議的請求已經被接受，目的是在2021年底之前啟動OTL-203的註冊研究之前徵求反饋。
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.
該公司表示，OTL-203的治療總體上耐受性良好，安全性與所選的調理方案一致。在基因治療前由於ERT而產生的抗α-L-艾杜糖醛酸酶抗體在治療後的兩個月內沒有出現在任何患者身上。
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説，治療顯示出快速和持續的代謝糾正，所有患者在12個月後在幹血斑樣本中實現了超生理IDUA表達，這是一個主要的療效終點。所有患者在治療後的縱向生長都在年齡合適的參考範圍內，隨訪時間從9個月到2年不等。報告稱，與治療前相比，所有患者的活動範圍都有所改善，隨訪時間從9個月到1.5年不等。
Orchard Therapeutics shares were up 12% to $8.11 in premarket trading.
Orchard Treeutics股價在盤前交易中上漲12%，至8.11美元。
Write to Chris Wack at chris.wack@wsj.com
寫信給克里斯·瓦克(chris.wack@wsj.com)
(END) Dow Jones Newswires
(完)道瓊通訊社
February 09, 2021 07:27 ET (12:27 GMT)
2021年02月09日07：27(格林尼治標準時間12：27)
Copyright (c) 2021 Dow Jones & Company, Inc.
版權所有(C)2021年道瓊斯公司
譯文內容由第三人軟體翻譯。
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