The drug industry’s efforts to develop a treatment for a rare kidney condition known in shorthand as ADPKD have come in fits and starts.

制药行业为一种简称为ADPKD的罕见肾病开发治疗药物的努力断断续续。

People with ADPKD, or autosomal dominant polycystic kidney disease, can use an Otsuka drug,

患有常染色体显性多囊肾病 (ADPKD) 的人可以使用大冢制药的药物，

Jynarque

Jynarque

, that U.S. regulators cleared in 2018 to slow the decline in organ function that’s brought on by the condition.

，美国监管机构在2018年批准了该药物，以减缓由这种疾病引起的器官功能衰退。

They have few other good options, however. Sanofi was developing a drug, venglustat, around the same time as Jynarque’s approval, but

然而，他们几乎没有其他好的选择。赛诺菲当时正在开发一种药物——韦格鲁司他，时间与Jynarque的批准差不多，但是

stopped clinical testing

停止临床试验

in 2021 after negative study results.

2021年，在研究结果负面之后。

The disease stayed on some drugmaker’s radars. In April, Novartis agreed to pay

这种疾病仍然在一些制药商的关注范围内。今年四月，诺华公司同意支付

$800 million to acquire

8亿美元收购

Regulus Therapeutics and an experimental therapy, farabursen, that the Swiss pharmaceutical company

瑞格勒斯治疗公司和一种实验性疗法，法拉布森，这家瑞士制药公司

said

说

showed “promising clinical efficacy and safety.” And a new biotechnology company launching Thursday is trying its hand at developing another.

显示出“良好的临床疗效和安全性”。而一家新生物技术公司周四开始尝试开发另一种药物。

Called Renasant Bio, the company raised $54.5 million in seed financing from blue-chip investors 5AM Ventures, Atlas Venture, OrbiMed and Qiming Venture Partners USA.

名为Renasant Bio的公司从5AM Ventures、Atlas Venture、OrbiMed和Qiming Venture Partners USA等一流投资者那里筹集了5450万美元的种子资金。

Renasant is the brainchild of a pair of University of California, San Francisco professors, Jeremy Reiter and Markus Delling, who sent 5AM a cold email in 2022 with a pitch built from their research into the disease’s biology. It's now headed up by Emily Conley, a Stanford University scientist who previously led .

Renasant 是加州大学旧金山分校的两位教授杰里米·雷特和马库斯·德林的心血结晶，他们在2022年给5AM发送了一封冷邮件，其中包含了他们对这种疾病生物学研究的提案。该公司现在由斯坦福大学科学家艾米丽·康利领导，她之前曾负责领导 。

Federation Bio

联邦生物

, a gut microbiome biotech, and oversaw business development at the genetic testing company 23AndMe.

，一家肠道微生物组生物技术公司，并在基因检测公司23AndMe负责业务发展。

Renasant draws inspiration from drug development in cystic fibrosis. While cystic fibrosis is a disease of the lungs, both conditions are linked to malformed

Renasant 从囊性纤维化药物开发中汲取灵感。尽管囊性纤维化是一种肺部疾病，但两种情况都与畸形有关。

ion channels

离子通道

. As in cystic fibrosis, where drugs like Vertex Pharmaceuticals’ Trikafta work by correcting and boosting the activity of those channel proteins, Renasant believes it can develop “corrector” and “potentiator” treatments that restore and open up the channels relevant to ADPKD.

就像在囊性纤维化中，像Vertex Pharmaceuticals的Trikafta这样的药物通过纠正和增强那些通道蛋白的活性来发挥作用，Renasant相信它可以开发出“矫正剂”和“增强剂”治疗方法，恢复并打开与ADPKD相关的通道。

“What a corrector does is it helps that misfolded protein fold properly,” said Conley. “Once the protein gets into the right shape, then it can go where it needs to go, and then the potentiator holds the channel open.”

“校正剂的作用是帮助错误折叠的蛋白质正确折叠，”康利说。“一旦蛋白质变成正确的形状，它就可以去到需要去的地方，然后增效剂会将通道保持在开放状态。”

“We know what's broken,” Conley added. “If we can fix it with correctors and potentiators, then we could have this very dramatic effect on patient outcomes.”

“我们知道问题出在哪里，”康利补充道。“如果我们可以用矫正剂和增强剂来修复它，那么我们就可以对患者的治疗结果产生非常显著的影响。”

Renasant’s lead drug candidate, which is currently in preclinical testing, is a small molecule corrector the company hopes can work in patients with any of the wide range of mutations that cause ADPKD. While Renasant envisions this molecule working as a standalone treatment, it’s also developing a potentiator that could be used in tandem to prevent cysts from forming or growing in the kidney..

Renasant公司的主要候选药物目前正处于临床前测试阶段，这是一种小分子矫正剂，该公司希望它能够适用于由导致ADPKD的各种突变的患者。虽然Renasant设想该分子可作为独立治疗手段，他们也在开发一种增强剂，可以与之配合使用，以防止肾脏中囊肿的形成或生长。

The company plans to enter clinical testing in the next few years.

公司计划在未来几年内进入临床试验阶段。

Overseeing Renasant’s scientific work is Gus Gustafson, a veteran of large pharmaceutical firms like Johnson & Johnson and Merck & Co. Its board of directors is led by Natalie Holles, the former CEO of Third Harmonic Bio, and includes Charlotte McKee, chief medical officer of cystic fibrosis drugmaker Sionna Therapeutics, as well as venture investors..

负责监督Renasant科学工作的是曾在强生和默克等大型制药公司任职的古斯·古斯塔夫森。其董事会由Third Harmonic Bio前首席执行官娜塔莉·霍尔斯领导，成员包括囊性纤维化药物制造商Sionna Therapeutics的首席医学官夏洛特·麦基以及风险投资人。

“Renasant has assembled the right team, with years of research experience in polycystic disease that has informed the right scientific approach,” Deborah Palestrant, a partner at 5AM, which incubated the biotech, said in a statement.

“Renasant 组建了合适的团队，拥有多年多囊性疾病研究经验，为正确的科学方法提供了依据，” 孵化该生物技术公司的 5AM 合伙人 Deborah Palestrant 在一份声明中表示。

Renasant estimates that some 12 million people have ADPKD worldwide. In addition to Novartis and Regulus, Vertex is also developing a drug for the disease.

Renasant 估计全球约有 1200 万人患有 ADPKD。除了诺华和 Regulus，Vertex 也在开发针对该疾病的药物。