JNT-517, a first-in-class SLC6A19 inhibitor, reduces plasma phenylalanine levels in subjects with phenylketonuria in a Phase 1/2 study

2024年1月30日 · JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe ...

2024年9月4日 · JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe ...

jnt-517是一款在研的治疗苯丙酮尿症（pku）的口服药物。2024年1月，jnana公司宣布jnt-517在治疗pku患者的1期临床试验中获得具有统计学意义的积极中期结果，该结果支持jnt-517在2025年上半年开始关键性3期临床试验。

2022年3月5日 · 日前，Jnana Therapeutics宣布，已经选择 JNT-517作为一种候选开发药物和潜在的“first-in-class”口服方法，以治疗苯丙酮尿症（PKU）。 这是一种罕见的遗传代谢性疾病。 该公司计划在今年晚些时候启动评估JNT-517的1期临床试验。 PKU由苯丙氨酸羟化酶（PAH）缺乏引起。 苯丙氨酸（Phe）是存在于所有含蛋白质食物中的一种氨基酸，而PAH是分解Phe所必需的酶。 当PAH缺乏或功能缺陷时，Phe将在血液中积累至异常高的毒性水平。 如果不及时治疗， …

2024年1月31日 · Jnana Therapeutics公司今天宣布，在研疗法JNT-517在治疗苯丙酮尿症（PKU）患者的1期临床试验中获得具有统计学意义的积极中期结果。JNT-517是一款潜在“first-in-class”小分子抑制剂，针对苯丙氨酸（Phe）转运蛋白SLC6A19。

JNT-517 is an orally active, selective SLC6A19 allosteric inhibitor with an IC 50 of 47 nM for human SLC6A19. JNT-517 can be used for the study of phenylketonuria (PKU).

2024年8月1日 · Jnana's lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease.

2024年8月2日 · JNT-517 is Jnana Therapeutics' first-in-class cryptic allosteric SLC6A19 inhibitor to treat phenylketonuria (PKU). This is a notable case study employing a novel lead generation approach with photoaffinity probes, leading to a clinical candidate for a historically challenging-to-drug target class.

2023年1月6日 · Jnana's wholly-owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of ...