Ipsen, Exicure to co-develop therapies for neurodegenerative diseases

共同开发神经退行性疾病疗法

2021-08-04 01:00:08 PHARMACEUTICAL

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Biopharmaceutical company Ipsen and Exicure have collaborated to research, develop, and commercialize novel Spherical Nucleic Acids as potential investigational therapies to treat Angelman syndrome and Huntington’s disease. Exicure’s SNAs are nanoscale constructs comprising synthetic nucleic acid sequences and provide distinct chemical and biochemical properties to oligonucleotides. Oligonucleotides are synthetic structures of nucleic acids used to modulate gene expression through a range of processes such as splice-modulation, inhibition, and gene activation. Ipsen stated that SNAs have shown that they boost the cell penetration properties, biodistribution and organ persistence properties of oligonucleotides in preclinical models. Under the terms of the exclusive collaboration agreement signed by the companies, Ipsen will secure exclusive options to license SNA-based therapeutics arising from two collaboration programmes, which are currently under discovery evaluation for Huntington’s disease and Angelman syndrome. Meanwhile, Exicure will carry out discovery and preclinical development of SNA-based therapies for Huntington’s and Angelman. Exicure CEO David Giljohann said: “We are thrilled to partner with Ipsen, a leading global company with significant expertise and commitment to developing treatments for patients with rare neurological diseases. “In collaboration with Ipsen, we have the opportunity to apply our technology to Huntington’s disease and Angelman syndrome, both indications requiring deep brain penetration and technological advances to reach previously hard-to-drug targets. “We believe our platform technology with its deep penetration and persistence of medicinal effect will allow Exicure and Ipsen to overcome challenges from first-generation oligonucleotides and bring new medicines to patients in need.” As per the terms of the deal, Exicure will receive an upfront payment of $20m in cash from Ipsen upon closing. If Ipsen exercises its option, the company will lead further development and commercialisation of the licensed products and pay Exicure up to $1bn in exercise fees and milestone payments.
生物制药公司Ipsen和Exicure合作研究、开发和商业化新型球形核酸,作为治疗Angelman综合征和亨廷顿病的潜在研究疗法。 EXICEURE的SNAs是由合成核酸序列组成的纳米级结构,提供了寡核苷酸独特的化学和生化性质。 寡核苷酸是人工合成的核酸结构,通过剪接调节、抑制和基因激活等一系列过程来调节基因表达。 Ipsen指出,SNAs已经表明,它们在临床前模型中增强了寡核苷酸的细胞穿透特性、生物分布和器官持久性。 根据两家公司签署的独家合作协议的条款,Ipsen将获得独家选择权,以许可由两个合作项目产生的基于SNA的治疗方法,这两个项目目前正在对亨廷顿病和安格曼综合征进行发现评估。 同时,Exicure将为亨廷顿和安杰尔曼进行基于SNA的治疗方法的发现和临床前开发。 Exicure首席执行官大卫·吉尔约翰(David Giljohann)表示:“我们很高兴与Ipsen合作,Ipsen是一家领先的全球公司,拥有丰富的专业知识,并致力于为罕见神经疾病患者开发治疗方法。 “与Ipsen合作,我们有机会将我们的技术应用于亨廷顿病和安格曼综合征,这两种适应症都需要深入大脑和技术进步来达到以前难以药物的目标。 “我们相信,我们的平台技术凭借其深入的渗透和持久的药物效果,将使Exicure和Ipsen克服来自第一代寡核苷酸的挑战,并为有需要的患者带来新的药物。” 根据交易条款,Exicure将在交易结束时从Ipsen获得2000万美元的现金预付款。 如果Ipsen行使其选择权,该公司将领导特许产品的进一步开发和商业化,并向Exicule支付高达10亿美元的行使费和里程碑付款。

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