Ace Therapeutics announced the launch of its integrated platform of preclinical genetic kidney disease models designed to recapitulate human hereditary renal pathology and support robust translational research for drug developers worldwide.
NEW YORK, NY, March 28, 2026 /24-7PressRelease/ — Ace Therapeutics announced the launch of its integrated platform of preclinical genetic kidney disease models designed to recapitulate human hereditary renal pathology and support robust translational research for drug developers worldwide.
Genetic kidney diseases, caused by inherited pathogenic mutations, represent a high unmet medical need with progressive renal impairment and limited treatment options. To address this challenge, Ace Therapeutics has developed and validated a suite of genetically engineered animal models that capture the core molecular, histological, and functional features of inherited renal disorders, enabling reliable target validation, mechanistic investigation, and therapeutic efficacy assessment.
Ace Therapeutics’ preclinical genetic kidney disease models cover a broad spectrum of rare and prevalent hereditary nephropathies, including Alport Syndrome, FSGS, Fabry Disease, Polycystic Kidney Disease (PKD), and C3 Glomerulopathy, using well-characterized knockout and conditional knockout rodent strains. Each model undergoes rigorous quality assurance, including immunofluorescence staining, transmission electron microscopy, molecular expression analysis, and longitudinal renal function monitoring, to ensure consistency and clinical relevance.
Beyond standard models, Ace Therapeutics offers custom model generation using CRISPR/Cas9 technology to create tissue-specific or conditional knockout lines for novel targets, further expanding the utility of its platform for exploratory and translational studies. All studies adhere to strict institutional animal care guidelines, with transparent data packages including raw and processed results to support regulatory and publication readiness.
Additionally, Ace Therapeutics possesses end-to-end preclinical service capabilities, spanning therapeutic efficacy testing, mechanistic profiling, biomarker development, and advanced imaging analytics. These services support dose–response evaluations for small molecules, antisense oligonucleotides (ASOs), and gene therapies, as well as single-cell RNA sequencing, urinary exosome analysis, and MRI-based cyst volumetry for in-depth disease characterization. Notably, Ace Therapeutics enables specialized gene therapy testing in Fabry disease models, where the company employs Gla knockout mice to evaluate AAV vector tropism, biodistribution, and therapeutic efficacy by measuring globotriaosylceramide (Gb3) accumulation, renal fibrosis, and other disease-relevant endpoints.
With these genetic kidney disease models and expanded preclinical renal research services, Ace Therapeutics reinforces its position as a trusted partner for biotech and pharmaceutical companies advancing novel therapies for genetic kidney diseases, supporting the discovery and development of transformative treatments for patients globally.
Ace Therapeutics is a specialized provider of preclinical research solutions for nephrology, offering integrated in vivo and in vitro models for chronic kidney disease, polycystic kidney disease, acute kidney injury, diabetic kidney disease, immune-mediated nephropathies, and genetic kidney disorders. Ace Therapeutics supports end-to-end preclinical drug development, from target validation to efficacy testing, with a focus on translational relevance and scientific rigor.
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