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Arcturus500万美元用于支持RNAi技术以及纳米载药系统研究

  1. Arcturus
  2. RNAi
  3. 纳米载药系统

来源:生物谷 2013-10-29 09:30

2013年10月28日讯 /生物谷BIOON/ --位于圣迭戈的Arcturus公司本周投入了500万美元支持其刚刚从Marina生物技术公司获得的RNAi技术和公司自主研发的纳米载药系统。公司希望于明年公布其新型基因沉默药物。和其他许多致力于RNAi技术的公司一样,Arcturus的技术主要是围绕在促使疾病中的致病基因沉默而进行的。

2013年10月28日讯 /生物谷BIOON/ --位于圣迭戈的Arcturus公司本周投入了500万美元支持其刚刚从Marina生物技术公司获得的RNAi技术和公司自主研发的纳米载药系统。公司希望于明年公布其新型基因沉默药物。和其他许多致力于RNAi技术的公司一样,Arcturus的技术主要是围绕在促使疾病中的致病基因沉默而进行的。虽然公司相比于该领域其他公司尚显稚嫩,但公司CEO表示Arcturus拥有的碱基解码模型(UNA)具有十分广阔的应用前景。

Arcturus公司成立于2013年,是强生公司旗下一家子公司,公司于今年七月刚刚获得120万美元的启动资金。Arcturus公司希望于2014年获得自己的候选药物。(生物谷Bioon.com)

详细英文报道:

San Diego RNAi specialist Arcturus Therapeutics pulled in $5 million this week to support its RNAi tech, which it acquired earlier this year from Marina Biotech ($MRNA), and its own nanoparticle delivery platform. The company expects to unveil its novel gene-silencing drugs with a candidate early next year.

Like many RNAi companies, Arcturus' technology is based around the silencing of gene expressions for diseases, although Arcturus is at a much earlier stage than some of the field's leaders, including Alnylam ($ALNY), which aims to have 5 treatments in clinical trials by 2015. But, according to CEO Joseph Payne, Arcturus' patent for its unlocked nucleobase analog (UNA), acquired from Marina for an undisclosed amount, has claims that are very broad in scale, including up to 40 nucleotides and acylic monomers, microRNA, siRNA and RNAi trigger techs, among others.

"We believe that once we go public with this early next year, it will command attention within the RNAi community, and the fact that we were able to achieve such potency at multi-doses gives us an edge," Payne told FierceDrugDelivery. "In the many years I've been in this field, I've never seen anything like it."

The UNA technology from Marina works with Arcturus' own lipid nanoparticle delivery system and is intended for the orphan disease market, the company says. It expects the "best-in-class" delivery tech will help expand its pipeline and bring RNA drugs into the mainstream. Its endosomal lipid technology, which is designed to disrupt the endosomal function of the cell, brings small bits of RNA (sRNA) into the cytoplasm, where it can then act on the specific part of the genetic code for a disease.

Founded in 2013, the company works out of Janssen Labs, owned by Johnson & Johnson ($JNJ). It pulled in $1.3 million in June seed funding before acquiring Marina's patents in August. As for the most recent cash, the $5 million series A round from private investors in Canada, the U.S. and Japan gives Arcturus enough to keep going through 2014 and get the candidate into the clinic by 2015.

"The focus is our lead program," Payne said of the company's plans for the funding. "We want to nominate a clinical candidate in 2014 and, in the very near term, hire some top-tier talent, including in business development."

RNAi drugs make up a field that Global Industry Analysts projects to reach over $4 billion by 2017, and Arcturus has high hopes for its product: "We plan to lead the next wave of RNAi technology," Payne said.

 

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