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肾脏疾病治疗方法开发商Purespring筹集1.05亿美元B轮融资,用于推进肾脏疾病的基因治疗

Purespring raises $105M to advance gene therapy for kidney disease

BioPharma Dive 等信源发布 2024-10-09 12:20

可切换为仅中文


Biotechnology company Purespring Therapeutics has raised about $105 million to fund its development of gene therapies for diseases of the kidney.

生物技术公司Purespring Therapeutics筹集了约1.05亿美元资金,用于开发肾脏疾病的基因疗法。

The Series B funding announced Wednesday was led by Sofinnova Partners and will support Purespring’s initiation of a Phase 1/2 study of its lead candidate for IgA nephropathy, or IgAN. Forbion, Glide Healthcare and founding investor Syncona Limited also participated in the round.

。Forbion、Glide Healthcare和创始投资者Syncona Limited也参加了这一轮。

Like many gene therapy developers, the company uses adeno-associated viruses, or AAVs, as the envelope for shuttling its gene therapies into the body. Established by Syncona in 2020, the company was formed to take AAV gene therapy into the kidneys, an organ that can be difficult to target.

像许多基因疗法开发人员一样,该公司使用腺相关病毒(AAV)作为将其基因疗法传递到体内的包膜。该公司由Syncona于2020年成立,旨在将AAV基因疗法引入肾脏,肾脏是一种难以靶向的器官。

Its choice of lead candidate, for IgAN, puts it in the competitive field, as a number of other drugmakers are working on new drugs — outside of gene therapy — for the rare kidney condition. Both Vertex Pharmaceuticals, and Novartis have acquired IgAN drug developers recently.

它选择了IgAN作为主要候选药物,使其进入了竞争领域,因为许多其他制药商正在开发除基因治疗之外的治疗罕见肾脏疾病的新药。Vertex Pharmaceuticals和诺华最近都收购了IgAN药物开发商。

IgAN results from the body producing too many antibodies that build up in the kidney, causing damage and eventually organ failure. While there are approved medications to treat disease symptoms, Purespring aims to target the condition’s roots. Its treatments targets a specialized cell called the podocyte, which it says are associated with approximately 60% of renal diseases..

IgAN是由于体内产生过多抗体而产生的,这些抗体在肾脏中积聚,导致损伤并最终导致器官衰竭。虽然有批准的药物可以治疗疾病症状,但Purespring的目标是针对疾病的根源。它的治疗靶向一种称为足细胞的特殊细胞,该细胞与大约60%的肾脏疾病有关。

Earlier this year, Purespring presented preclinical data for its AAV gene therapy at the 61st European Renal Association Congress. And in 2023, a paper published in the Science Translational Medicine showed the company’s gene therapy could successfully introduce DNA into podocytes in vivo.

今年早些时候,Purespring在第61届欧洲肾脏协会大会上介绍了其AAV基因治疗的临床前数据。2023年,发表在《科学转化医学》上的一篇论文显示,该公司的基因疗法可以成功地将DNA导入体内足细胞。

“Our novel treatment platform and deep understanding of kidney disease puts us in a position to stop, reverse and even cure kidney disease,” Purespring’s CEO Julian Hanak said in a statement. “The funds raised will allow us to bring our novel treatments to patients in the clinic.”

Purespring首席执行官朱利安·哈纳克(JulianHanak)在一份声明中表示:“我们新颖的治疗平台和对肾脏疾病的深刻理解使我们能够阻止、逆转甚至治愈肾脏疾病。”。“筹集的资金将使我们能够为诊所的患者带来新的治疗方法。”

The company will present IgAN data for the first time at the American Society of Nephrology’s Kidney Week meeting later this month.

该公司将于本月晚些时候在美国肾脏病学会肾脏周会议上首次提交IgAN数据。