Spanish biotech SpliceBio has raised $135 million in financing that will help it move a gene-editing therapy for an inherited retinal disease (IRD) through clinical testing.

西班牙生物技术公司SpliceBio已经筹集了1.35亿美元的资金，这将帮助其推动一种针对遗传性视网膜疾病（IRD）的基因编辑疗法通过临床试验。

SpliceBio's lead in-house pipeline project is in Stargardt disease, an IRD with no approved therapies that is caused by changes in a gene called ABCA4 and results in fatty material building up in the macula of the eye, damaging vision.

SpliceBio公司内部主导的管线项目针对的是Stargardt病，这是一种由ABCA4基因变异引起的视网膜色素变性（IRD），目前尚无获批疗法。该病会导致脂肪物质在眼底黄斑区堆积，从而损害视力。

The Barcelona-based company is currently running a phase 1/2 study of the Stargardt candidate, codenamed SB-007, which it describes as the first dual adeno-associated viral (AAV) gene therapy for the condition to be cleared by the FDA for human testing.

总部位于巴塞罗那的公司目前正在对代号为SB-007的斯塔加特病候选药物进行1/2期研究，该公司称其为首个获得FDA批准用于人体试验的双腺相关病毒（AAV）基因疗法。

SB-007 is designed to deliver the full-length ABCA4 protein, which is too big to be carried by other gene delivery vectors. To solve that problem, SpliceBio and collaborators at Princeton University in the US have developed a way to split the protein into two, packaging it in two separate AAV vectors for delivery, and then using cellular machinery to join the two segments together into the functional protein..

SB-007旨在递送全长ABCA4蛋白，该蛋白太大，无法被其他基因递送载体携带。为了解决这一问题，SpliceBio公司与美国普林斯顿大学的合作者开发了一种方法，将该蛋白分成两部分，分别包装在两个AAV载体中进行递送，然后利用细胞机制将这两部分连接起来，形成功能性蛋白。

The Series B was co-led by new investors EQT Life Sciences and Sanofi Ventures and backed by SpliceBio's existing partner Roche, New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Asabys Partners, and Novartis' venture capital unit.

B轮融资由新投资者EQT生命科学和赛诺菲风险投资公司共同领投，并得到SpliceBio现有合作伙伴罗氏、新企业联合公司、UCB风险投资、Ysios资本、Gilde医疗保健、Asabys合伙人和诺华风险投资部门的支持。

'This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology and beyond,' commented SpliceBio chief executive Miquel Vila-Perelló.

“这笔融资标志着SpliceBio的一个关键里程碑，因为我们正在推进SB-007针对斯塔加特病的临床开发，并继续扩展我们在眼科、神经科及其他领域的产品线，”SpliceBio首席执行官米格尔·维拉-佩雷略表示。

After SB-007, the company's pipeline consists of two other candidates for unidentified single-gene IRDs and a single-gene central nervous system disorder.

在SB-007之后，该公司的研发管线还包括另外两种针对未知单基因IRDs和一种单基因中枢神经系统疾病的候选药物。

The Spanish biotech has already attracted one partnership with a big pharma group after Roche's gene therapy unit, Spark Therapeutics, licensed access to its delivery platform in a

这家西班牙生物技术公司已经吸引了一家大型制药集团的合作，此前罗氏的基因治疗部门Spark Therapeutics授权使用其递送平台。

$126 million deal

1.26亿美元的交易

signed in 2023.

签署于2023年。

Under the terms, Spark – which was

根据条款，Spark公司——它被

acquired

获得

by Roche in a $4.8 billion takeover deal in 2019 and has already brought one gene therapy for an IRD to market – bagged exclusive rights to a therapy for an as-yet undisclosed IRD in return for upfront, opt-in and milestone payments.

2019年，罗氏以48亿美元的收购交易获得了这种基因疗法，并且已经将一种针对IRD的基因疗法推向市场——通过支付预付款、选择权费用和里程碑款项，获得了另一种尚未披露的IRD疗法的独家权利。

Since then, Spark has been hit by lacklustre sales of its

自那时以来，Spark 的销量一直不佳

Luxturna

勒克斯图尔纳

(voretigene neparvovec) therapy for Leber's congenital amaurosis, and Roche recently

（伏瑞特基因奈帕罗韦）用于治疗莱伯先天性黑蒙症，罗氏公司近期

revealed

揭示

(PDF) big job cuts at the unit, whilst still affirming its commitment to developing gene therapies.

(PDF) 该部门大幅裁员，同时仍承诺致力于开发基因疗法。

Commenting on the new financing, Carole Nuechterlein, head of Roche Venture Fund, said that she had been 'impressed by the team's strong execution, the momentum behind SB-007 in Stargardt disease, and the platform's potential to unlock a new class of genetic medicines.'

罗氏风险基金负责人卡罗尔·诺伊切特莱因在评论这笔新的融资时说，她对“团队的出色执行力、SB-007在斯塔加特病领域的强劲势头以及该平台解锁一类新型基因药物的潜力”印象深刻。