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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製藥公司和OliPass公司宣佈建立戰略合作伙伴關係開發反義寡核苷酸治療藥物
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.

華盛頓,9月亞洲網加利福尼亞州聖何塞2022年8月29日電萬達製藥公司(納斯達克代碼:VNDA)和奧利帕斯公司(OliPass)(KOSDAQ:24460)今天宣佈,他們已經簽署了一項研究和開發合作協議,共同開發一套基於奧利帕斯公司專有的修飾多肽核酸的反義寡核苷酸分子。這一創新的合作伙伴關係利用了Vanda和OliPass各自的優勢,以支持基於ASO的精準醫學療法的發展,並可能為兩家公司創造引人注目的價值機會。

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.

合作的重點將是在疾病狀態下使用ASO編輯和修改基因表達,在這些疾病狀態下,基因的表達要麼發生改變,要麼表達的基因序列可以改變以達到治療的目的。OliPass獨特的Olipass多肽核酸(OPNA)技術為實現這些基因表達修飾提供了交付平臺。

"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."

萬達公司首席執行官兼董事會主席邁克爾·H·波爾梅羅普洛斯醫學博士、首席執行官兼董事會主席邁克爾·H·波爾梅羅普洛斯説:“我們很高興有一個合作伙伴來加強我們的反義寡核苷酸計劃平臺,該計劃旨在治療具有眾所周知的遺傳機制的疾病,對這些疾病有很高的醫療需求。Olipass的交付平臺承諾高效交付我們的ASO,以追求精確療法的發展。“

"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首席執行官Shin Chung博士説:“萬達與我們有共同的願景,即反義寡核苷酸可能為患者提供廣泛的治療選擇。”我們期待着共同努力,提高這些分子的治療效果,並在將它們帶入臨牀環境中發揮不可或缺的作用。“

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已經確定了兩個ASO靶點,它們已經在模擬兩個未披露的疾病靶點的細胞系中得到驗證,一個是罕見的孤兒,另一個適用於廣泛的免疫腫瘤學疾病。萬達與OliPass的合作伙伴關係是利用OliPass獨特的OPNA化學物質來增強現有的ASO,這是將這些臨牀前發現應用於體內和臨牀測試的下一步。

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被選擇性地用陽離子部分修飾,這些陽離子部分在保持與靶核酸非常高的結合親和力的同時,提高了肽核酸(PNA)的穩定性和細胞通透性。在動物模型中,已觀察到OPNAs的治療效果,劑量低至10 ng/kg,顯示出比現有ASO技術以前可實現的劑量低許多數量級的效果。此外,OPNAs具有與細胞核中的前-mRNA結合的能力,並能夠靶向以前無法治療的致病變體和疾病。

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