Preclinical Hematology 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.

Bone Marrow Failure and Aplastic Anemia Models

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Ace Therapeutics provides comprehensive preclinical services focused on hematological disorders, with advanced expertise in developing, validating, and applying in vivo models of bone marrow failure (BMF) and aplastic anemia (AA). Our platform enables robust evaluation of therapeutic candidates—from small molecules to cell therapies—accelerating your drug development pipeline.

Why Focus on Bone Marrow Failure and Aplastic Anemia?

Bone marrow failure syndromes, including aplastic anemia, involve defective hematopoiesis leading to peripheral cytopenias and elevated risks of infection, bleeding, and transformation to leukemia. Key challenges in developing therapies include

Complex pathogenesis: Immune-mediated destruction of hematopoietic stem cells (HSCs), genetic mutations (e.g., FANCA, TERC), or toxic exposures.
Limited preclinical models: Models must replicate human disease heterogeneity and treatment responses.
Need for multi-parameter validation: Efficacy assessment requires integrated hematologic, histopathologic, and functional endpoints.

In Vivo Models Offered

Our in vivo services are based on validated disease models that closely mimic the pathophysiological mechanisms of BMF and AA. These models are used for a range of investigational needs, including efficacy evaluation, hematologic recovery analysis, and immunological profiling.

Radiation-Induced Myelosuppression Model

Induction Method: Total body irradiation (TBI), typically 5-7 Gy
Disease Features: Depletion of hematopoietic stem and progenitor cells, pancytopenia
Applications: Hematopoietic recovery, radioprotectants, stem cell mobilization agents
Advantages: Controlled induction, reproducible severity, scalable model
Model Validation: Blood counts, bone marrow histology, flow cytometry for HSPCs

Chemotherapy-Induced Myelosuppression Model

Induction Method: Busulfan or 5-fluorouracil (5-FU) administration
Disease Features: Myeloablative cytopenia, bone marrow hypocellularity
Applications: Supportive care agents, hematopoietic stimulants, adjuvants to chemotherapy
Advantages: Mimics clinical cytotoxic effects, dose-adjustable
Model Validation: Hematological profiling, bone marrow smears, survival curves

Immune-Mediated Aplastic Anemia Model

Induction Method: Intraperitoneal injection of allogeneic lymphocytes from C57BL/6 donors
Disease Features: T cell-mediated bone marrow destruction, severe pancytopenia
Applications: Immunosuppressive therapies, T-cell modulation, bone marrow transplantation studies
Advantages: Mimics idiopathic aplastic anemia, immune pathophysiology included
Model Validation: Flow cytometry of immune subsets, ELISA for cytokines, BM cellularity assessment

Fanconi Anemia Model (Genetic)

Strain: Fancd2^−/− or Fanca^−/− knockout mice
Disease Features: Bone marrow aplasia, chromosomal instability, DNA repair defects
Applications: Gene therapy candidates, DNA repair pathway inhibitors, oxidative stress mitigation
Advantages: Genetic fidelity to human disease, long-term study design possible
Model Validation: Chromosomal breakage tests, HSPC functionality assays, response to crosslinking agents (e.g., MMC)

Comprehensive Service Workflow

Our end-to-end services cover model customization, therapeutic testing, and mechanistic analysis.

Service Module	Key Components & Methodologies
Study Design & Model Selection	We recommend models based on your compound's mechanism and regulatory goals. Example frameworks include Immunosuppressant screening: Immune-mediated AA model + cyclosporine control. HSC-targeted therapies: Irradiation model + humanized CD34+ cell engraftment.
Therapeutic Dosing & Monitoring	Routes: IV, IP, PO, or subcutaneous delivery. Longitudinal sampling: Peripheral blood counts, serum cytokines, toxicity markers.
Endpoint Analysis	Hematologic: Complete blood counts (Hb, platelet, ANC) Histopathologic: BM cellularity, fibrosis, apoptosis Functional: CFU assays, HSC repopulation capacity Mechanistic: T-cell clonality, oxidative stress, DNA damage
Data Interpretation & Reporting	Comparative statistics vs. controls/baselines. Biomarker correlation with efficacy.

Technical Advantages

Multi-Model Integration

Combine immune-mediated, genetic, and toxicologic models for comprehensive efficacy profiling.

Advanced Imaging & Flow Cytometry

Quantify HSC niches, vascularization, and immune infiltration.

Bone Marrow Niche Analysis

3D micro-CT for vascular network assessment in osteoarthritic models adapted for BM vasculature studies.

Why Choose Ace Therapeutics

Expertise in Hematology Models: Our scientific team specializes in the design and execution of hematopoietic-focused preclinical studies.
Flexible Study Design: We offer tailored study protocols to meet specific client goals and compound profiles.
Validated Readouts: All models are validated with multi-parameter endpoints to ensure reliable translational insight.
Integrated Services: In addition to in vivo testing, we offer ex vivo analyses, bioanalytical support, and molecular validation platforms.

Contact Ace Therapeutics to explore how our in vivo bone marrow failure models can support your hematologic drug development. We provide robust, reproducible, and translationally relevant data to help you make informed preclinical decisions.

Frequently Asked Questions About Genetic Hematological Disease Models

How do I select the right model for my candidate?

We match models to your drug's mechanism. For example

Immune inhibitors → Immune-mediated AA model.
Gene therapies → Genetic models (Fancd2^-/-).
Mitigating chemotherapy-induced BMF → Irradiation/benzene models.

What endpoints demonstrate therapeutic efficacy?

Key endpoints include improved survival, hematopoietic recovery (↑Hb/Plt/ANC), BM cellularity restoration, and reduced pathogenic T-cells. Functional assays (CFUs, transplants) validate HSC recovery.

Can you test cell therapies or gene-editing approaches?

Yes. We utilize humanized models (e.g., irradiated NSG mice + CD34+ cells) and support in vivo delivery of viral vectors (e.g., AAV) or cell products (e.g., CRISPR-edited HSCs).

What sample types are required for analysis?

We accept test articles in any formulation (small molecules, antibodies, cells). For histopathology, intact femurs/tibias are ideal.