TechCrunch News 10月28日 02:42
用AI和基因编辑技术攻克多囊肾病
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Demetri Maxim因家族遗传的多囊肾病(PKD)而投身科研,受到CRISPR技术逆转小鼠PKD的研究启发,他创立了生物科技公司Nephrogen。该公司利用AI和先进筛选技术,开发出一种能将基因编辑药物精准递送至肾脏病变细胞的特殊系统。该系统效率比FDA批准的现有载体高100倍。Nephrogen正寻求400万美元种子轮融资,计划于2027年启动临床试验,Maxim本人也将参与其中,以期彻底治愈PKD。

💡 基因技术带来希望:Demetri Maxim因家族遗传的多囊肾病(PKD)深受困扰,在了解到CRISPR技术能逆转小鼠PKD的研究后,他看到了治愈的可能性,并致力于将这一技术应用于人类治疗。

🚀 Nephrogen的创新递送系统:为解决药物递送至病变肾脏细胞的难题,Maxim创立Nephrogen,利用AI和先进筛选技术开发出一种高效的基因编辑药物递送系统,其效率是FDA批准的现有载体的100倍。

💰 融资与临床试验:Nephrogen正在进行400万美元的种子轮融资,并计划于2027年启动针对其递送机制和药物的临床试验。Maxim本人将积极参与临床研究,以期为自己和更多患者带来治愈的希望。

🏥 现实的挑战与紧迫性:PKD患者面临背痛、频繁就医的困扰,现有药物效果有限且副作用明显。Nephrogen的突破性进展,若能成功实现,将为患者提供彻底摆脱疾病的可能,具有重要的现实意义。

When Demetri Maxim was seven years old, his mother’s kidneys stopped working. She was put on dialysis, which meant that she had to go to the hospital four times a week to have her blood filtered by a machine since her own kidneys could no longer function independently.

Two years later, Maxim’s mother received a kidney transplant. Although the surgery was successful and allowed her to lead a relatively normal life, it wasn’t the end of his family’s struggle with kidney disease. It turned out that Maxim had inherited the disease, Polycystic Kidney Disease (PKD), from her.

About one in seven Americans has Chronic Kidney Disease (CKD), and about 10% of those CKD cases are due to a genetic condition. Maxim has been obsessed with finding a cure for himself and others ever since he was in high school.

Maxim’s “Aha!” moment occurred in 2021, when Nature Magazine published a study proving that PKD is reversible in mice using CRISPR technology. At the time, he was pursuing his graduate degree in computational biology at Stanford while simultaneously engaging in kidney research under his professor, Vivek Bhalla.

Although Maxim was convinced that gene therapy could reverse PKD, the biggest hurdle was creating a mechanism to deliver the drugs directly to the diseased cells.

To solve this critical challenge, he founded Nephrogen in 2022, a biotech startup that uses AI and advanced screening to develop a specialized delivery system for safely getting gene-editing medicines into the exact cells in the kidney. Nephrogen is one of the 20 finalists in Startup Battlefield, part of TechCrunch Disrupt 2025.

After three years of development, Maxim claims Nephrogen has succeeded in creating a delivery mechanism that is 100 times more efficient at transporting medicine to the kidney than the “vehicles” currently approved by the FDA.

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The next major step for Nephrogen is to advance its novel delivery mechanism, along with a drug the startup developed, into clinical studies, which Maxim anticipates will begin in 2027. To support this, the company is raising a $4 million seed round.

Maxim intends to participate in the clinical study himself, given the significant challenges he faces living with PKD.

“You get a lot of back pain. You have to go to the hospital a lot. You are on this drug that’s supposed to slow the progression, but it doesn’t really do anything. Just makes you pee all the time,” he said, adding that there is always a risk that his disease will progress to require dialysis.

This makes Nephrogen’s approach all the more critical, as its success could cure him of PKD entirely.

If you want to hear from Nephrogen firsthand, and see dozens of additional pitches, attend valuable workshops, and make the connections that drive business results, head here to learn more about this year’s Disrupt, held October 27 to 29 in San Francisco. 

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多囊肾病 PKD 基因编辑 CRISPR AI 生物科技 Nephrogen Polycystic Kidney Disease Gene Editing Biotech TechCrunch Disrupt
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