Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy (GA), a complication of age-related macular degeneration (AMD), as it prepares to test it in a clinical trial.

赛诺菲已获得美国食品药品监督管理局（FDA）对其用于治疗地理性萎缩（GA）的基因疗法的快速审查，地理性萎缩是与年龄相关的黄斑变性（AMD）的一种并发症，该公司正准备在临床试验中对其进行测试。

The SAR446597 gene therapy could provide a one-off treatment for

SAR446597基因疗法可能提供一次性治疗

GA

通用算法

, a major cause of irreversible sight loss that affects millions of people around the world, and free patients from the need for injections into the eye every few weeks using current therapies.

，这是影响世界各地数百万人的不可逆视力丧失的主要原因，并且使用当前的治疗方法，患者每隔几周就需要接受眼部注射。

The newly awarded fast-track status is reserved for new drugs and vaccines that aim to treat or prevent serious conditions, for which there are few or no alternative therapies, and can speed up their development and review, getting them to patients more quickly.

新授予的快速通道资格是为治疗或预防严重疾病的新药和疫苗而设，这些疾病很少或没有其他替代疗法，可以加快它们的开发和审查，让患者更快获得药物。

According to Sanofi, SAR446597 delivers genetic material encoding two therapeutic antibody fragments that target and inhibit two components of the complement pathway, namely C1s in the classical pathway and factor Bb in the alternative pathway.

根据赛诺菲的说法，SAR446597 传递编码两种治疗性抗体片段的遗传物质，这两种抗体片段分别靶向并抑制补体通路中的两个成分，即经典通路中的 C1s 和替代通路中的因子 Bb。

The company said this dual approach delivers 'sustained complement suppression within the retinal microenvironment.' The complement immune system is thought to be over-activated in GA.

该公司表示，这种双重方法“在视网膜微环境中实现了持续的补体抑制。” 补体免疫系统被认为在GA中过度激活。

It is preparing to start a phase 1/2 trial of the gene therapy, its second in the retinal diseases area after SAR402663, another one-shot gene therapy for neovascular or 'wet' AMD which started a phase 1/2 study last November.

它正准备开始该基因疗法的 1/2 期试验，这是其在视网膜疾病领域的第二个疗法，之前 SAR402663 是另一种针对新生血管性或“湿性”AMD 的一次性基因疗法，于去年十一月开始了 1/2 期研究。

AMD is an acquired progressive degeneration of the retina that affects approximately 200 million people globally, said Sanofi. GA is an advanced form of dry AMD, which results when the central part of the retina starts to deteriorate, while wet AMD is caused by the growth of aberrant blood vessels in the retina..

赛诺菲表示，AMD 是一种后天性的视网膜进行性退化疾病，全球约有 2 亿人受其影响。GA 是干性 AMD 的晚期形式，发生在视网膜中央部分开始退化时，而湿性 AMD 则是由视网膜中异常血管的生长引起。

Sanofi was formerly working with UK biotech

赛诺菲之前与英国生物技术公司合作

Gyroscope Therapeutics

陀螺仪治疗公司

on gene therapies for AMD, taking an option on its lead candidate for GA – GT005 – in 2021 in a deal that included an upfront payment of $40 million.

关于AMD的基因疗法，在2021年的一项交易中获得了其GA主导候选药物GT005的选择权，该交易包括4000万美元的预付款。

That came to an end after Novartis

在诺华之后，那件事结束了

acquired

获得的

Gyroscope later that year in a deal valued at up to $1.5 billion, taking control of the programme, which was in a pair of phase 2 studies. Two years later, Novartis

同年晚些时候，陀螺仪公司以高达15亿美元的交易金额获得了该计划的控制权，当时该计划正处于两项二期研究之中。两年后，诺华公司接手。

abandoned

废弃的

GT005 – which delivered the gene coding for complement factor I (CFI) – after interim results from the studies suggested there was little chance of showing efficacy.

GT005——递送编码补体因子I（CFI）的基因——在研究的中期结果表明几乎无法显示疗效后。

Other companies developing gene therapies for GA include Johnson & Johnson, which licensed rights to a candidate from Hemera Biosciences in 2020 (JNJ-1887) that expresses a soluble form of CD59 and is now in phase 2 testing.

其他开发GA基因疗法的公司包括强生公司，该公司于2020年从Hemera Biosciences获得了候选药物（JNJ-1887）的授权，该药物表达CD59的可溶性形式，目前正在进行二期试验。