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基因疗法的最新进展

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发表于 2007-8-26 21:07 | 显示全部楼层 |阅读模式
Quark Pharmaceuticals Extends Research Agreement with State University of New York for Proprietary siRNA Compounds for Acute Hearing Loss
FREMONT, Calif., Aug. 20 /PRNewswire/ -- Quark Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on discovering and developing novel RNA interference-based therapeutics, announced today that it has expanded its relationship with the State University of New York at Buffalo, Center for Hearing & Deafness, which is the Company's primary site for the pre-clinical studies of its product candidate, AHLi-11, for the treatment of acute hearing loss. Quark initiated its collaboration with the State University of New York in 2005.

The current studies, led by Professor Richard Salvi, focus on the in-depth analysis of the effect of AHLi-11 and other molecules in preventing chemotherapy induced hearing loss. Based on these studies, Quark Pharmaceuticals expects to file an IND within 2007 for AHLi-11 for the prevention chemotherapy-induced hearing loss.

AHLi-11 is a siRNA-based drug that temporarily inhibits the expression of human gene p53. Cochlear hair cell apoptosis (cell death), a key factor in several of the more common causes of acute hearing loss, is believed to be induced by molecular mechanisms most likely associated with p53-dependent stress response. Inhibition of p53, therefore, is suggested as a potential modality for the prevention of ototoxic hearing loss, a common side effect of certain drugs including aminoglycoside antibiotics and cancer therapeutics such as cisplatin, as well as acoustic trauma.

Quark has demonstrated delivery of siRNA into target cells in rats and monkeys along with its persistence in the cochlear hair cells for at least 15 days. In preclinical animal studies, AHLi-11 appears to protect cochlear hair cells from apoptotic cell death induced by the chemotherapeutic agents, cisplatin and carboplatin, and by acoustic trauma.

Daniel Zurr, CEO of Quark Pharmaceuticals, said, "We are encouraged by the advancement of AHLi-11 for acute hearing loss and look forward to filing an IND by year-end for the prevention of chemotherapy-induced hearing loss. Up to a million people per year are treated with cisplatin in the US and Europe, and the risk of serious hearing impairment is particularly devastating in children. With this in mind, Quark will continue its efforts to advance AHLi- 11 in response to this major unmet medical need.

"We are also thrilled to expand our relationship with Professor Salvi and his colleagues at the State University of New York as it will enable us to deepen our understanding of the molecular processes underlying acute hearing loss and to examine several variables related to the impact of AHLi-11 as a curative measure. Our collaborations with leading academic institutions mark the ongoing development of our pipeline and reinforce Quark's expertise in the development of siRNA-based therapeutics that offer the potential to treat a wide range of disease targets."

About AHLi-11

AHLi-11 is a synthetic siRNA that is a temporary and reversible suppressor of p53, and contains the same active compound as AKIi-5, Quark's drug candidate for the prevention of acute renal failure. AHLi-11 is in development for the prevention of acute hearing loss, initially induced by the ototoxic cancer therapeutic agent cisplatin. AHLi-11 is based on Quark's proprietary, patented concept of temporary and reversible inhibition, for therapeutic purposes, of the expression of the transcription factor human p53, which is associated with DNA repair and apoptosis. In response to certain chemotherapeutic agents, such as cisplatin and carboplatin, molecular mechanisms, most likely associated with p53-dependent stress response, are suspected of triggering cochlear hair cell apoptosis. Temporary inhibition of p53 prevents apoptosis, allowing restoration of normal DNA and cellular integrity. The AHLi-11 active molecule was designed and patented by Quark. The Company has licenses for certain RNAi intellectual property from Alnylam and Silence Therapeutics

About Quark Pharmaceuticals, Inc.

Quark Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing novel therapeutics based on its proprietary gene discovery science and technology, with an initial focus on drug candidates that work through the natural mechanism in the cell known as RNA interference, or RNAi, for the treatment of diseases associated with oxidative stress. Quark believes that its proprietary target gene discovery platform, BiFAR(TM), combined with its ability to design and successfully deliver synthetic molecules of the new class of RNAi therapeutics known as small-interfering RNA, or siRNA, to specific organs in the body, enables the Company to rapidly develop drug candidates. Quark has two internally discovered and developed lead product candidates: RTP801i-14 in phase 1 clinical trial for the treatment of wet age-related macular degeneration, and AKIi-5 for the prevention of acute renal failure. The Company has licensed RTP801i-14 to Pfizer on an exclusive worldwide basis. Quark has, in addition, a product candidate portfolio of RNAi therapeutics based on novel targets and therapeutic concepts discovered using BiFAR(TM) and designed for the treatment of oxidative stress associated diseases of the inner ear, lungs and additional organs of the body.

Quark is headquartered in Fremont, California and operates research and development facilities in Boulder, Colorado and Ness-Ziona, Israel. Additional information is available at www.quarkpharma.com

Forward-looking statement

Various statements in this release concerning the Company's future expectations, plans and prospects, including its intention to publicly offer shares of its common stock, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to fluctuations in our stock price, as well as those risks more fully discussed in the "Certain Factors That May Affect Future Results" section of the Company's most recent Quarterly Report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward- looking statements represent the Company's views only as of today and should not be relied upon as representing its views as of any subsequent date. The Company does not assume any obligation to update any forward-looking statements.

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 楼主| 发表于 2007-8-26 21:15 | 显示全部楼层
这是关于基因方面的,是其中一个方面进入了临床应用.

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发表于 2007-8-26 21:16 | 显示全部楼层
林照袄,有没有关于毛细胞的资料?我很想了解这种情况啊
不幸选择了我,我选择了坚强。

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 楼主| 发表于 2007-8-26 21:27 | 显示全部楼层
这则信息是关于毛细胞的,它主要是通过抑制一种P53的基因,来达到促进毛细胞生产的目的.(它们研究表明,P53基因对毛细胞生产有抑制作用).至于具体效果,前面没有具体报道.

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发表于 2007-8-26 21:50 | 显示全部楼层
上面的全是英文的,我的英语水平有限了,你能给大家翻译吗?^_^
不幸选择了我,我选择了坚强。

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 楼主| 发表于 2007-8-26 21:54 | 显示全部楼层
呵呵,我英文水平也非常有限啊,所以没法翻译过来.只知道他们开发出一种叫做AHLi-11的药,现在跟纽约州立大学合作.

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发表于 2007-8-27 03:07 | 显示全部楼层
LZ能给个文章的出处吗?

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 楼主| 发表于 2007-8-27 14:23 | 显示全部楼层

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发表于 2007-8-27 14:59 | 显示全部楼层
能给大家翻译吗?

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厦门智声听力 您值得信赖的朋友

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发表于 2007-8-27 15:46 | 显示全部楼层
苏版找个论坛翻译员,可兼职翻译此类文章,但需要大家的赞助哟!!!   
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