果园治疗亮点最近的成就,到2019年战略重点为全球领先的基因治疗

乌节标志

  • 准备MLD,ADA-SCID三导计划并且是监管文件,在未来三年
  • 最近,在X-CGD宣布临床验证的概念演示平台的改造潜力
  • 推进前期管线与潜在临床用于TDBT和MPS-IIIA临床试验申请证明 - - 概念
  • 在财务状况稳健,进入2019以$ 340M的现金和投资

美国波士顿和英国伦敦,2019年1月7,/美通社B3C / -金宝搏官网mg果园治疗(NASDAQ:ORTX),一家领先的商业阶段的生物制药公司致力于将患者的生命有严重和危及生命通过创新的基因治疗罕见疾病,今天在第37届年度总结最近的成就,并在其参加联合2019战略重点J.P. Morgan Healthcare Conference in San Francisco. Mark Rothera, president and chief executive officer, will present a business overview outlining the company’s progress as a global leader in gene therapy on Wednesday, January 9, 2019 at 3:00 p.m. PT that will be webcast at ir.orchard-tx.com.

“2018是用于果园一个重要的一年,我们的收购和整合葛兰素史克的罕见疾病的成功,标志着离体基因治疗产品组合,我们的制造能力,我们的首次公开发行完成初始缩放”罗瑟拉先生说。“2019将继续公司的发展在基因治疗的领先者,拥有多个临床里程碑支持三台监管文件,在未来三年,越来越多的制造能力。我们有一个大胆的设想,并且非常的道路上提供有转换的患者少见,威胁生命的疾病的生活与世界各地的单次治疗的潜在基因疗法“。

2019战略重点

神经代谢障碍

  • 释放20例二,三年的随访数据来自OTL-200为异染性脑白质退化症的新配方registrational试验(MLD)
  • 在前三患者释放从冷冻保存的制剂的临床试验OTL-200的MLD数据移入
  • 提交临床试验应用(CTA)对于OTL-201黏多糖贮积症临床试验的类型IIIA(MPS-IIIA)和支持起始

原发性免疫缺陷

  • 在20例患者解除从OTL-101的腺苷脱氨酶严重联合免疫缺陷的新配方registrational试验为期两年的随访数据(ADA-SCID)
  • 从OTL-101在ADA-SCID冷冻保存的制剂的临床试验释放10例移植数据
  • 8例释放从OTL-103的维斯科特 - 奥尔德里奇综合征的新配方registrational试验的3年随访数据(WAS)
  • 发起用于冷冻保存的制剂的临床试验OTL-103在WAS
  • 设计与X连锁慢性肉芽肿病(X-CGD),其最近取得证据的概念临床(链接到充分的释放搞上registrational试验监管的OTL-102这里

血红蛋白病

  • 报告在输血依赖性β-地中海贫血的临床验证的概念,数据OLT-300(TDBT)

主要成就2018

输油管扩展和进步

  • 完成葛兰素史克的罕见疾病的战略收购和随后的整合离体基因治疗产品组合,包括Strimvelis®,患者ADA-SCID批准在欧盟唯一的治疗方法,随着临床MLD方案,WAS和TDBT
  • 完成预生物制品许可申请(BLA)和程序仍有望在2020年美国一个BLA申报在与美国食品和药物管理局(FDA)的OTL-101 ADA-SCID CMC专题会议,
  • 在X-CGD实现概念的临床证明对OTL-102,证明12个月后患者中性粒细胞功能持续水平
  • 得到的稀土儿科疾病型号从FDA批准用于OTL-200用于治疗MLD和OTL-201为MPS-IIIA的治疗
  • 从欧洲药品管理局(EMA)的OTL-300获得重点药品(PRIME)指定为TDBT治疗

企业与制造业发展

  • 从C系列融资和承销IPO,融资总收益约3.75亿$ 2018
  • 位于加州弗里蒙特,租用一个生产基地,开辟了马萨诸塞州波士顿的公司办公室。制造工厂将增强公司的能力开发和交付离体慢病毒载体和基因校正的造血干细胞对宽范围在全球范围内的罕见疾病和将补充伙伴集体,将巩固发射的前三个方案的现有网络。(链接到完全释放这里

现金指导
该公司截至2018拥有约3.4亿$的现金和投资。该公司预计,其现金,现金等价物和有价证券为2018 12月31日,将会使公司的当前预期运营开支和资本开支要求提供资金到2020年下半年。

介绍在37届摩根大通保健大会

乌节将在下午3点网络直播从第37届摩根大通保健大会在旧金山周三,2019年1月9日其公司演示PT。演示文稿的网上直播将是下在orchard-tx.com该公司网站的投资者与媒体节“新闻事件”可用。该网络广播的重播将在该网站果园呈现以下上进行归档。

关于果园
果园治疗是一个完全集成的商业化阶段的生物制药公司,致力于转化的患者的生命通过创新的基因治疗严重和危及生命的罕见疾病。

乌节路的自体组合离体基因疗法包括Strimvelis®,第一自体离体腺苷批准欧洲药品管理局基因治疗脱氨酶重症联合免疫缺陷(ADA-SCID)。对于神经代谢紊乱的附加程序,原发性免疫缺陷和血红蛋白病包括用于异染性脑白质营养不良(MLD),ADA-SCID和威斯科特 - 奥尔德里奇综合征(WAS),为X连锁慢性肉芽肿病(X-CGD)和临床方案三个高级registrational研究输血依赖性β-地中海贫血(TDBT),以及一个广泛的临床前管道。

果园目前在英国和美国,包括伦敦,旧金山和波士顿设有办事处。

前瞻性声明
This press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s expectations regarding the timing of regulatory submissions for approval of its product candidates, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, the likelihood of approval of such product candidates by the applicable regulatory authorities, and Orchard’s guidance that its existing cash, cash equivalents and marketable securities as of December 31, 2018 will enable the company to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2020. These statements are neither promises nor guarantees but are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the delay of any of Orchard’s regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchard’s product candidates, the receipt of restricted marketing approvals, or delays in Orchard’s ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. For additional disclosure regarding these and other risks faced by Orchard, see the disclosure contained in Orchard’s public filings with the Securities and Exchange Commission, including in the final prospectus related to Orchard’s initial public offering filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act of 1933, as amended, as well as subsequent filings and reports filed by Orchard with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of publication of this document. Orchard undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

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