诺华与 Precision合作开发潜在的镰状细胞性贫血基因治疗

2022-06-23 MedSci原创 MedSci原创

Precision BioSciences 已与诺华达成独家全球体内基因编辑研发合作和许可协议。两家公司将致力于开发治​​疗镰状细胞病和β-地中海贫血等血红蛋白病的潜在疗法。

Precision BioSciences 已与诺华达成独家全球体内基因编辑研发合作和许可协议。两家公司将致力于开发治​​疗镰状细胞病和β-地中海贫血等血红蛋白病的潜在疗法。

Precision 将开发定制的 ARCUS 核酸酶并进行体外研究。诺华随后将处理所有后续的研究、开发、制造和商业化业务。诺华获得了定制 ARCUS 核酸酶的独家许可,并将向 Precision 支付 7500 万美元的预付款。届时,Precision 将有资格获得高达 14 亿美元的潜在里程碑付款,以及一些研究资金和可能的中个位数到低两位数的分层特许权使用费。

“我们很高兴与诺华合作,将 ARCUS 基因组编辑的精确性和多功能性与诺华的基因治疗专业知识和致力于开发一次性、潜在的变革性治疗难以治疗的遗传性血液疾病的承诺相结合,”迈克尔·阿莫罗索, Precision 首席执行官在一份声明中表示。 “这项合作将建立在 ARCUS 独特的基因插入能力的基础上,并说明其作为潜在的体内药物开发的优质基因组编辑平台的实用性。根据该协议,Precision 将单独或与世界一流的合作伙伴一起开展积极的体内基因编辑计划,用于在造血干细胞、肝脏、肌肉和中枢神经系统中进行靶向基因插入和基因缺失,展示 ARCUS 的独特多功能性。 ”

β-地中海贫血和镰状病基因疗法的机会是巨大的,一些公司正处于批准的风口浪尖。周二,CRISPR Therapeutics 报告了其为 CTX001 提交生物制品许可申请 (BLA) 的计划,CTX001 是一种治疗输血依赖性 β 地中海贫血和严重镰状细胞病的潜在疗法。 CTX001 (exa-cel) 是一种基于 CRISPR-Cas9 的基因编辑疗法。

6 月初,CRISPR 及其合作伙伴 Vertex Pharmaceuticals 报告了 exa-cel 在两种适应症中的阳性数据。首先,数据显示接受治疗的 44 名 TDT 患者中有 42 名在长达 37.2 个月内保持无输血状态。在 SCD 中,所有 31 例患者在治疗后均无复发性血管闭塞危象,持续时间长达 32.3 个月。

6 月 10 日,美国食品和药物管理局的细胞、组织和基因治疗咨询委员会对蓝鸟生物的两种慢病毒载体 (LVV) 基因治疗β-地中海贫血和脑肾上腺脑白质营养不良的基因疗法投了赞成票。

对于 betibeglogene autotemcel (beti-cel) 用于治疗β-地中海贫血,委员会以 13 票对 0 票赞成该疗法。 Beti-cel 是一种一次性基因疗法,适用于需要定期输血的 β-地中海贫血患者。后期试验表明,接受治疗的患者中有 89% 实现了输血独立。

对于蓝鸟用于脑肾上腺脑白质营养不良(CALD)的 LVV 基因疗法 elivaldogene autotemcel(eli-cel),咨询委员会以 15 票对 0 票赞成该疗法。 CALD是一种由ABCD1基因突变引起的严重神经系统疾病。它是罕见的、渐进的、X 连锁的,最终是致命的。该疗法使用 Lenti-D 慢病毒载体的离体转导将 ABCD1 基因的功能拷贝插入患者的造血干细胞。

Precision 的联合创始人兼首席科学官 Derek Jantz 博士说:“与目前正在开发的其他体外基因疗法相比,我们正在为镰状细胞病寻求的体内基因编辑方法可能具有许多显着优势。 也许最重要的是,它可以为治疗干细胞移植不是现实选择的地区的患者打开大门。我们相信 ARCUS 平台的独特特性,特别是其高效靶向基因插入的能力,使其成为该项目的理想选择,我们期待与诺华的合作伙伴合作,将这种新疗法带给患者。 ”

原始出处:

https://www.biospace.com/article/precision-partners-with-novartis-on-sickle-cell-disease-cure-in-1-4b-deal/?s=86

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    2022-06-23 ms5000000518166734

    学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习学习

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