Preclinical Kidney Disease R&D Solutions

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All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

Polycystic Kidney Disease (PKD) Models

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Ace Therapeutics delivers advanced preclinical solutions to support drug development and mechanistic research for polycystic kidney disease (PKD). Our expertise lies in deploying rigorously validated PKD models that recapitulate human disease progression, enabling reliable evaluation of therapeutic candidates and disease-modifying pathways.

Understanding Polycystic Kidney Disease (PKD)

Polycystic kidney disease is a genetic disorder characterized by renal cyst formation, progressive nephron loss, and impaired renal function. With two primary forms—autosomal dominant PKD (ADPKD) and autosomal recessive PKD (ARPKD)—this condition poses unique challenges for preclinical research due to its slow progression and heterogeneous clinical manifestations. Our preclinical platforms address these challenges by offering tailored models and analytical workflows to study cystogenesis, fibrosis, and therapeutic interventions.

Integrated Preclinical Services for DKD Research

Our DKD platform combines disease modeling with advanced analytical workflows to deliver actionable insights:

Therapeutic Candidate Testing

Evaluate efficacy of small molecules, biologics, or gene therapies using multi-parametric endpoints:

Cyst volume quantification (MRI/ultrasound)
Renal function biomarkers (creatinine, BUN, urinary albumin)
Histopathological scoring of cystic index and fibrosis.

Mechanistic Studies

Investigate molecular pathways driving cyst expansion and renal deterioration:

Transcriptomic profiling of cyst-lining epithelia
Immunohistochemistry for proliferation (Ki-67), apoptosis (TUNEL), and signaling pathways (mTOR, cAMP).

Biomarker Development

Identify and validate surrogate markers for disease progression or treatment response:

Urinary exosome analysis (miRNA, protein cargo)
Serum metabolomics linked to cyst growth.

Validated PKD Models for Translational Research

Ace Therapeutics employs genetically engineered and spontaneous PKD models to mimic human pathophysiology.

Model	Application	Validation
Pkd1^flox/flox Conditional Knockout Mice	Ideal for studying cyst initiation and progression via tissue-specific gene inactivation.	Longitudinal monitoring of cyst growth (ultrasound/MRI), histopathology (H&E, trichrome), and renal function (serum creatinine, BUN).
Han:SPRD Rat (Autosomal Dominant PKD)	Models spontaneous ADPKD with progressive cyst development and interstitial fibrosis.	Quantification of cyst volume, fibrosis biomarkers (α-SMA, collagen I), and glomerular filtration rate (GFR).
Pkd2^-/- Zebrafish	High-throughput screening for modulators of cyst formation and renal tubule dilation.	Live imaging of pronephric cysts, gene expression profiling (e.g., pax2a, wt1a).

Model Validation and Quality Assurance

Our PKD models undergo stringent validation to ensure translational relevance and reproducibility:

Longitudinal Disease Monitoring

Serial imaging (MRI, ultrasound) to track cyst growth rates.
Functional assessments (GFR, proteinuria) aligned with human disease stages.

Multi-Modal Histopathology

Digital pathology workflows for cyst quantification and fibrosis scoring.
Advanced staining techniques (PAS, Sirius Red) to assess extracellular matrix remodeling.

Molecular Pathway Analysis

qPCR/Western blot for disease-relevant targets (e.g., PC1, PC2, CFTR).
Single-cell RNA sequencing to map epithelial heterogeneity in cystic lesions.

Advantages of Ace Therapeutics' PKD Platform

Customizable protocols aligned with client compound profiles
Integrated data deliverables combining raw datasets, statistical analysis and interpretive reports
Dedicated project management for transparent timelines and milestone tracking
Access to multidisciplinary expertise across nephrology, imaging and bioanalysis teams

Frequently Asked Questions (FAQs) About PKD Model

How do I choose between murine and rat PKD models?

Murine models (e.g., Pkd1^flox/flox) allow genetic manipulation, while Han:SPRD rats better replicate spontaneous ADPKD progression. We align model selection with your study goals.

Which PKD model is most suitable for chronic efficacy studies

Models like Han SPRD rats and PCK rats exhibit progressive cystic disease over months enabling long-term dosing and safety assessments.

Can comorbidities be incorporated into PKD studies

Yes. We can introduce hypertension or dietary modifications to mimic human disease complexity and assess compound performance under stress.

What imaging modalities are available

High-resolution ultrasound and MRI are standard. We also offer optical coherence tomography for superficial cyst analysis.

Do you support biomarker assay development

Our team develops and validates ELISA and multiplex panels for urinary and serum biomarkers linked to kidney injury and cystic progression.