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法国AAVLife融资1200万美元开发治疗弗里德希氏共济失调基因疗法

  1. AAVLife
  2. 基因疗法
  3. 弗里德希氏共济失调
  4. 腺病毒载体

来源:生物谷 2014-04-17 09:17

AAVLife公司从Versant Ventures等风投公司获得了总额1200万美元的投资用于开发一种治疗知名罕见病弗里德希氏共济失调的基因敲除疗法。

2014年4月16日讯 /生物谷BIOON/ --法国的生物技术公司AAVLife公司最近宣布公司已经从Versant Ventures等风投公司获得了总额1200万美元的投资用于开发一种治疗知名罕见病弗里德希氏共济失调的基因敲除疗法。这种疾病主要是由人体一种名为共济蛋白的基因突变而引起的,它会导致进行性心脏退化并最终导致死亡。而AAVLife公司提出的治疗方法则是通过利用腺病毒类病毒载体将正常的共济蛋白基因导入人体中修复基因突变从而治愈这种疾病。

目前这种来源于法国国家健康与医学研究中心的疗法已经在动物实验中证明有很光明的前景。公司计划在进一步进行临床前研究并开始设计人体试验。而AAVLife公司也为这一项目作出了足够的准备,包括力邀曾在葛兰素史克公司担任研发的Amber Salzman出任CEO等。

据估计目前在欧洲和美国共有2万人患有弗里德希氏共济失调。(生物谷Bioon.com)

详细英文报道:

Parisian biotech AAVLife has come out of stealth mode with a Versant Ventures-led $12 million A round, looking to advance a gene-deleting therapy that promises to treat the rare and deadly Friedreich's ataxia.

The disease, which gradually robs sufferers of motor function, is a result of a mutation in the frataxin gene that leads to progressive cardiac degradation and, often, death. Targeting only Friedreich's ataxia's effects on cardiac function, AAVLife's solution involves replacing the malfunctioning frataxin gene with a healthy one, delivered using a harmless adeno-associated virus (AAV), which explains the biotech's name.

The method, based on work from the French National Institute of Health and Medical Research (Inserm), has already demonstrated promise in a mouse study published in Nature Medicine, and AAVLife said it's in the midst of further preclinical work with plans to test its therapy on humans next year.

To get there, the biotech has recruited a cast of experts in gene therapy and AAV delivery, including CEO Amber Salzman, a former GlaxoSmithKline ($GSK) R&D exec who played a major role in bringing about the first gene-therapy trial for the rare adrenoleukodystrophy, the top target of IPO success story bluebird bio ($BLUE). Combined with the $12 million round, led by Versant and joined by the Inserm Transfert Initiative, AAVLife now has the cash and personnel it needs to potentially make a difference for the roughly 20,000 Friedreich's ataxia sufferers in the U.S. and Europe, Salzman said.

"Friedreich's ataxia may be a rare disorder, but it is an all too common feature of everyday life for patients and their families," she said in a statement. "We are moving with appropriate care but also urgency."

Meanwhile, maturing science and willing investors have made it a good time to be a gene therapy startup. In February, Third Rock Ventures staked $45 million on Cambridge, MA's Voyager Therapeutics with a plan to use AAV-delivered genes to treat central nervous system diseases, and the Wellcome Trust put up $20 million in January to support NightstaRx and its gene therapies for degenerative eye conditions.

 

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