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Vanda Pharmaceuticals And OliPass Announce Strategic Partnership To Develop Antisense Oligonucleotide Therapeutics

Vanda Pharmaceuticals And OliPass Announce Strategic Partnership To Develop Antisense Oligonucleotide Therapeutics

Vanda制药公司和OliPass公司宣布建立战略合作伙伴关系开发反义寡核苷酸治疗药物
Benzinga Real-time News ·  2022/09/29 16:31

WASHINGTON, Sept. 29, 2022 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (NASDAQ:VNDA) and OliPass Corporation (OliPass) (KOSDAQ: 24460) today announced that they have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on OliPass' proprietary modified peptide nucleic acids. This innovative partnership leverages the respective strengths of Vanda and OliPass to support the development of ASO-based precision medicine therapeutics and potentially create compelling value opportunities for both companies.

华盛顿,9月亚洲网加利福尼亚州圣何塞2022年8月29日电万达制药公司(纳斯达克代码:VNDA)和奥利帕斯公司(OliPass)(KOSDAQ:24460)今天宣布,他们已经签署了一项研究和开发合作协议,共同开发一套基于奥利帕斯公司专有的修饰多肽核酸的反义寡核苷酸分子。这一创新的合作伙伴关系利用了Vanda和OliPass各自的优势,以支持基于ASO的精准医学疗法的发展,并可能为两家公司创造引人注目的价值机会。

The collaboration will focus on editing and modifying gene expression using ASOs in disease states where the expression of genes is either altered or the sequence of the expressed genes can be altered for therapeutic benefit. OliPass' unique Olipass Peptide Nucleic Acids (OPNA) technology provides the delivery platform to enable these gene expression modifications.

合作的重点将是在疾病状态下使用ASO编辑和修改基因表达,在这些疾病状态下,基因的表达要么发生改变,要么表达的基因序列可以改变以达到治疗的目的。OliPass独特的Olipass多肽核酸(OPNA)技术为实现这些基因表达修饰提供了交付平台。

"We are excited to have a partner to enhance our antisense oligonucleotide program platform aimed at the treatment of disorders with well-understood genetic mechanisms and for which there is a high unmet medical need," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. "Olipass' delivery platform holds the promise of efficient delivery of our ASO's in pursuit of the development of precision therapeutics."

万达公司首席执行官兼董事会主席迈克尔·H·波尔梅罗普洛斯医学博士、首席执行官兼董事会主席迈克尔·H·波尔梅罗普洛斯说:“我们很高兴有一个合作伙伴来加强我们的反义寡核苷酸计划平台,该计划旨在治疗具有众所周知的遗传机制的疾病,对这些疾病有很高的医疗需求。Olipass的交付平台承诺高效交付我们的ASO,以追求精确疗法的发展。“

"Vanda shares a vision with us on the potential for anti-sense oligonucleotides to open up a broad range of therapeutic options to patients," said Dr. Shin Chung, CEO of OliPass. "We look forward to working together to enhance the therapeutic profile of these molecules and playing an integral role in bringing them into the clinical setting."

OliPass首席执行官Shin Chung博士说:“万达与我们有共同的愿景,即反义寡核苷酸可能为患者提供广泛的治疗选择。”我们期待着共同努力,提高这些分子的治疗效果,并在将它们带入临床环境中发挥不可或缺的作用。“

Vanda has already identified two ASO targets that have been validated in cell lines that model two undisclosed disease targets, one rare orphan and the other applicable to a broad set of immuno-oncological conditions. Vanda's partnership with OliPass to enhance the existing ASOs with OliPass' unique OPNA chemistry is the next step to take these preclinical findings to in vivo and clinical testing.

Vanda已经确定了两个ASO靶点,它们已经在模拟两个未披露的疾病靶点的细胞系中得到验证,一个是罕见的孤儿,另一个适用于广泛的免疫肿瘤学疾病。万达与OliPass的合作伙伴关系是利用OliPass独特的OPNA化学物质来增强现有的ASO,这是将这些临床前发现应用于体内和临床测试的下一步。

OPNAs are selectively modified with cationic moieties that enhance both stability and cell permeability of peptide nucleic acids (PNA) while maintaining very high binding affinities to target nucleic acids. Therapeutic efficacy for OPNAs has been observed with dosages as low as 10 ng/kg in animal models, showing effects at dosages many orders of magnitude lower than previously achievable with existing ASO technologies. Furthermore, OPNAs have the capacity to bind to pre-mRNA in nucleus and enable targeting pathogenic variants and conditions that were previously untreatable.

OPNAs被选择性地用阳离子部分修饰,这些阳离子部分在保持与靶核酸非常高的结合亲和力的同时,提高了肽核酸(PNA)的稳定性和细胞通透性。在动物模型中,已观察到OPNAs的治疗效果,剂量低至10 ng/kg,显示出比现有ASO技术以前可实现的剂量低许多数量级的效果。此外,OPNAs具有与细胞核中的前-mRNA结合的能力,并能够靶向以前无法治疗的致病变体和疾病。

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