PKD1抑制剂(polycystin 1, transient receptor potential channel interacting inhibitors)、SHANK3调节剂(SH3 and multiple ankyrin repeat domains 3 modulators)、RNA干扰

治疗领域

遗传病与畸形泌尿生殖系统疾病其他疾病

在研适应症

常染色体显性多囊肾病Phelan-McDermid 综合征

非在研适应症-

原研机构

PYC Therapeutics Ltd.

在研机构

PYC Therapeutics Ltd.

非在研机构-

最高研发阶段临床1期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

关联

项与 PYC-003 相关的临床试验

NCT06714006 / Not yet recruiting临床1期

A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Intravenously Administered PYC-003, a Peptide-phosphorodiamidate Morpholino Oligonucleotide Conjugate, in Healthy Adult Participants and Adult Participants with Confirmed PKD1 Mutation-associated Autosomal Dominant Polycystic Kidney Disease

This is a Phase 1, First-in-Human study to evaluate the safety and tolerability of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD. In Phase A of this study, healthy adult participants will either receive a single dose of PYC-003 or a placebo. Phase B, healthy adult participant will not receive any intervention and will serve as a baseline. Phase C, adult participants with confirmed PKD1 mutation-associated ADPKD will receive a dose of PYC-003.

开始日期2025-04-01

申办/合作机构

PYC Therapeutics Ltd.

100 项与 PYC-003 相关的临床结果

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100 项与 PYC-003 相关的转化医学

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100 项与 PYC-003 相关的专利（医药）

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项与 PYC-003 相关的新闻（医药）

2023-11-13

·PRNewswire

PYC'S FOURTH DRUG CANDIDATE HAS DISEASE-MODIFYING POTENTIAL IN POLYCYSTIC KIDNEY DISEASE

PYC has developed a new drug candidate for the >5 million people worldwide[1] with Polycystic Kidney Disease (PKD) This drug candidate has demonstrated efficacy in human models derived from the kidneys of patients with end-stage renal failure due to PKD [2] PKD is a life-changing disease affecting 1 in every 1,000 people[3] Half of the patient population with PKD will require a kidney transplant by the age of 60 due to the absence of impactful treatment options in this disease[4] PKD represents an addressable market of >US$10 billion p.a. and is an area of major commercial interest for the drug development industry[5] Pre-clinical models suggest that addressing the underlying cause of PKD (the way that PYC's drug candidate works) could arrest the course of the disease in humans, enabling damaged kidneys to regenerate and restore function[6] PYC plans to accelerate this drug candidate into human trials in ~12 months – pursuing a potential high-velocity path through clinical trials to market[7] Investor Webinar Friday 17 November PYC will host an investor call at 9am AWST/12pm AEDST on Friday 17 November to discuss these results – investors can register for the call here: PERTH, Australia and SAN FRANCISCO, Nov. 13, 2023 /PRNewswire/ -- PYC Therapeutics today announces the results of a study conducted in human 3-dimensional models derived from patients with end-stage renal failure due to Autosomal Dominant Polycystic Kidney Disease (PKD). The results demonstrate that an investigational drug candidate designed by PYC (known as PYC-003) to address this disease at its root cause is effective. These 3D patient-derived cyst models represent the 'gold-standard' pre-clinical assay for evaluating drug candidates in this indication[8]. Continue Reading Figure 1 Figure 2 Figure 3 Figure 4