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Ace Therapeutics : Your Trusted Preclinical Partner in Hematology Research

Ace Therapeutics is a contract research organization dedicated to advancing preclinical research in hematologic diseases. With a strong foundation in hematology and pharmacology, we deliver high-quality in vitro and in vivo services, customized biospecimen analysis, biomarker profiling, and comprehensive drug discovery solutions.

Our scientific approach is grounded in translational relevance and designed to support the complex challenges of blood disorder research. At Ace Therapeutics , we aim to be a reliable partner in your journey toward therapeutic innovation.

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Understanding Hematologic Diseases: Classification and Characteristics

Hematologic diseases encompass a diverse group of disorders affecting the blood, bone marrow, and lymphatic systems. These diseases can impact red blood cells, white blood cells, platelets, plasma proteins, and the hematopoietic stem cells responsible for their production.

Key Categories of Hematologic Disorders

Red Blood Cell Disorders

  • Iron Deficiency Anemia: Caused by insufficient iron, leading to reduced hemoglobin production and microcytic hypochromic anemia.
  • Megaloblastic Anemia: Due to vitamin B12 or folate deficiency, resulting in impaired DNA synthesis and large, immature red cells.
  • Hemolytic Anemia: Caused by premature destruction of red blood cells; can be intrinsic or extrinsic.
  • Sickle Cell Disease: A genetic disorder leading to abnormal hemoglobin S, causing sickling of red cells and vaso-occlusion.
  • Thalassemia: Inherited disorders of globin chain synthesis, leading to ineffective erythropoiesis and microcytic anemia.
  • Aplastic Anemia: Bone marrow failure causing pancytopenia due to loss of hematopoietic stem cells.

White Blood Cell Disorders

  • Leukocytosis: Elevated white blood cell count, often due to infection, inflammation, or stress.
  • Neutropenia: Abnormally low neutrophil count, increasing susceptibility to infection.

Platelet and Coagulation Disorders

  • Immune Thrombocytopenia (ITP): Autoimmune destruction of platelets, leading to increased bleeding risk.
  • Thrombotic Thrombocytopenic Purpura (TTP): Life-threatening disorder with platelet aggregation in small vessels, due to ADAMTS13 deficiency.
  • Hemophilia A/B: X-linked recessive disorders due to deficiency of coagulation factor VIII (A) or IX (B).
  • Von Willebrand Disease: Most common inherited bleeding disorder, caused by deficiency or dysfunction of von Willebrand factor.
  • Disseminated Intravascular Coagulation (DIC): Acquired condition with widespread clotting and bleeding due to systemic activation of coagulation.

Comprehensive In vivo Models for Hematological Disease Research

At Ace Therapeutics , we offer a robust portfolio of validated animal models to support preclinical research in hematological disorders. Our models are designed to enable the in vivo evaluation of efficacy, toxicity, pharmacodynamics, and pharmacokinetics of investigational drugs targeting a wide range of blood diseases.

Genetic Hematological Disease Bone Marrow Failure and Aplastic Anemia Autoimmune Hematologic Disease Anemia Models Coagulation and Bleeding Disorder Myelodysplastic Syndromes Models Other Hematologic Models

Genetic Hematological Disease Models

Disease Model Type Preclinical Species
Sickle Cell Disease Transgenic HbS (e.g., SAD mice) Mouse
β-Thalassemia Hbb-th3/+ and Hbb-th3/th3 knockout models Mouse
α-Thalassemia Hba1/2 gene deletion models Mouse
Hereditary Spherocytosis Mutations in Ank1, Sptb, Epb42 Mouse
G6PD Deficiency G6PD knockout models Mouse
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Bone Marrow Failure and Aplastic Anemia Models

Model Type Induction Method Preclinical Species
Radiation/Drug-Induced Myelosuppression Total body irradiation, Cyclophosphamide Mouse, Rat
Immune-Mediated Aplastic Anemia Lymphocyte transfer models Mouse
Fanconi Anemia FANCA knockout models Mouse
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Autoimmune Hematologic Disease Models

Indication Model Platform Preclinical Species
Autoimmune Hemolytic Anemia Anti-RBC antibody injection Mouse
Immune Thrombocytopenia Anti-platelet antibody induction Mouse, Rat
SLE-associated Hematologic Manifestations NZB/NZW F1 or MRL/lpr lupus models Mouse
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Anemia Models (Non-Genetic)

Indication Induction Method Preclinical Species
Iron Deficiency Anemia Iron-deficient diet, Deferoxamine Mouse, Rat
Vitamin B12 / Folate Deficiency Dietary induction Mouse, Rat
Anemia of Chronic Disease Inflammation-induced (e.g., tumor models) Mouse
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Coagulation and Bleeding Disorder Models

Hemophilia Models

Indication Model Platform Preclinical Species
Hemophilia A Factor VIII knockout (F8−/−) Mouse, Dog
Hemophilia B Factor IX knockout (F9−/−) Mouse, Dog

Thrombosis and Coagulation Models

Indication Induction Method Preclinical Species
Venous Thrombosis Ferric chloride injury, IVC ligation Mouse, Rat
Arterial Thrombosis Laser injury, Ferric chloride Mouse
Disseminated Intravascular Coagulation LPS or snake venom induction Mouse, Rat
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Myelodysplastic Syndromes (MDS) and Related Models

Model Platform Preclinical Species
NUP98-HOXD13 transgenic model Mouse
Tet2/Dnmt3a mutant model Mouse
Human MDS xenografts (PDX) NSG Mouse
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Other Hematologic Models

Indication Model Platform Preclinical Species
Polycythemia Vera JAK2-V617F mutation models Mouse
Leukocytosis/Thrombocytosis GM-CSF, TPO, or EPO overexpression Mouse
Radiation-Induced Hematopoietic Damage Whole-body or targeted irradiation Mouse, Rat, NHP
Learn More

*Note: Ace Therapeutics provides model-based research services exclusively through internal execution—no live model exportation is offered.

Unmatched Needs? Our Models Fill the Gap!

Our experts specialize in developing disease-relevant models that replicate key features of human hematologic disorders — from anemia to myelofibrosis.
Whether optimizing established platforms or creating de novo systems (CRISPR/Cas9, patient-derived xenografts, humanized models), we translate your research targets into mechanistically driven results.

Reach Out for Tailored Preclinical Support

In vitro Hematology Modeling Services

Revolutionizing preclinical research with human-relevant, customizable systems for hematological drug development.

3D Hematopoietic Culture Systems iPSC-Derived Hematopoietic Disease Models Primary Human Hematopoietic Cells Mechanistic & Predictive Readouts Hematology & Vascular Interaction Analytical & Molecular Profiling

Next-Generation 3D Hematopoietic Culture Systems

We overcome the limitations of traditional 2D assays by offering biomimetic 3D platforms that recapitulate human hematopoietic niches.

  • Tissue-relevant extracellular matrices: Collagen, bioactive hydrogels.
  • Physiological gradients: Hypoxia, nutrient diffusion, vascular-like networks.
  • Complex co-culture models: Incorporating osteoblasts, adipocytes, endothelial cells, and patient-derived HSPCs.
  • Predictive assessment of drug efficacy, toxicity, and mechanism of action in disease-specific microenvironments.
  • Testing of niche-targeted therapies for rare hematological disorders.
  • Evaluation of CAR-T cell interactions in a dynamic 3D immune-oncology context.

iPSC-Derived Hematopoietic Disease Models

We offer patient-specific induced pluripotent stem cell (iPSC) platforms for modeling genetic blood disorders and age-related hematopoietic decline.

  • Generation of iPSCs from patient fibroblasts or blood cells.
  • CRISPR-edited isogenic lines for mechanistic or gene therapy studies.
  • Differentiation into erythroid, myeloid, and megakaryocytic lineages.
  • High-throughput drug screening tailored to individual genetic backgrounds.
  • Evaluation of clonal hematopoiesis, aging pathologies (e.g., aplastic anemia).
  • Personalized modeling of inherited blood diseases and gene editing validation.

Functional Assays Using Primary Human Hematopoietic Cells

Our primary human HSPC platforms offer clinically relevant, ex vivo systems for functional analysis of hematopoiesis and drug toxicity.

  • Bone marrow, mobilized peripheral blood, and cord blood (fresh or cryopreserved).
  • Disease-state CD34+ cells (e.g., myelofibrosis, MDS) for targeted drug testing.
  • Colony-Forming Unit (CFU) Assays: Quantify progenitor activity.
  • LTC-IC Assays: Assess long-term stem cell functionality.
  • Autologous co-culture systems to predict patient-specific responses.
  • Evaluation of T cell-mediated bone marrow failure mechanisms.

Mechanistic & Predictive Readouts: Functional + Omics Integration

To provide multi-dimensional insights, we combine functional readouts with high-resolution molecular profiling.

  • Single-Cell RNA Sequencing: Trace lineage commitment, capture transcriptomic drug responses.
  • Live-Cell Imaging: Track proliferation, apoptosis, and mitochondrial stress in real-time.
  • Metabolic Profiling: Assess metabolic shifts under hypoxia or drug exposure.
  • Cytokine & Chemokine Multiplexing: Quantify key signaling markers (e.g., SDF-1, TPO).

Specialized Hematology & Vascular Interaction Assays

Our platform includes specialized bioassays to model hematologic function and blood-endothelial interactions.

  • Erythropoiesis Assays from CD34+ progenitors
  • Platelet Aggregation & Function Testing for anti-thrombotic studies
  • Endothelial-Blood Cell Interaction Assays simulating inflammation and hemostasis
  • Hemoglobin Polymerization Inhibition Tests for hemoglobinopathies

Analytical & Molecular Profiling Platforms

We support all assay systems with robust molecular analysis technologies.

  • Flow Cytometry for lineage-specific and functional markers
  • ELISA for cytokines and plasma protein quantification
  • qPCR for targeted gene expression analysis
  • Morphological Imaging via cytospin and microscopy
  • Coagulation Assays for comprehensive hematology profiling

Customized Biospecimen Collection and Analysis

We provide tailored biospecimen services to support biomarker discovery and therapeutic validation.

  • Peripheral blood, bone marrow, plasma, serum, and spleen tissues
  • Longitudinal sample collection in disease models
  • Multiparametric analysis (hematologic, biochemical, molecular)
  • Biobanking with full traceability and metadata capture
  • RNA, DNA, and protein extraction optimized for hematologic samples

Hematological Disease Biomarker Analysis

Biomarkers are critical indicators that provide insights into disease pathogenesis, progression, and response to therapeutic interventions. Their comprehensive analysis in preclinical research is vital for understanding drug effects and guiding development. Ace Therapeutics offers a comprehensive bioanalysis platform for assessing a wide range of hematological disease biomarkers, including:

Disease Category Pathogenic Mechanism Biomarker Name(s) Research Utility
Anemia Iron Metabolism Hemoglobin, HCT, MCV, Ferritin, Serum Iron, TIBC Assess iron status, RBC indices
Bone Marrow Failure Soluble CD25, miR-150-5p, miR-146b-5p, miR-1 SAA diagnosis, severity indicator
Hemolysis Reticulocytes, LDH, Bilirubin, Haptoglobin, DAT, Cold Agglutinins Hemolysis detection, autoimmune involvement
Hemophilia Coagulation Factor Deficiency aPTT, PT, TT, FVIII, FIX activity Factor deficiency, clotting function
Arthropathy CTX-I/II, sCS846, VCAM-1, C2M, CRP Joint damage, inflammation
ITP Autoimmune Platelet Destruction Platelet count, Auto-Abs, BAFF, TGF-β1, miR-199a-5p Immune-mediated thrombocytopenia
Sickle Cell Disease Hemolysis HbF, LDH, Bilirubin, Free Heme, Retics Hemolysis monitoring, disease modifier
Endothelial Dysfunction VWF, E-selectin, MAC-1, CRP, Cytokines Vascular inflammation
Severity/Complications WBC, TCD velocity, NT-proBNP, Microalbuminuria Predict stroke, PH, renal risk
Thalassemia Iron Overload Serum Ferritin, IGHG4, C1S, TF, HBA1, CLU Iron burden, novel markers
Endothelial Dysfunction ICAM-1, VCAM-1, VWF, EMPs, NO, ET-1 Vascular risk profiling
Von Willebrand Disease VWF Deficiency/Dysfunction VWF:Ag, RCo, Multimers, Propeptide, FVIII Subtype classification, VWF function

Comprehensive Drug Development Solutions for Hematological Disorders

Ace Therapeutics offers a comprehensive suite of preclinical drug development solutions for hematological disorders, designed to support clients from the earliest stages of discovery through to preclinical studies.

Target Identification & Validation Preclinical Efficacy Solutions Preclinical PK/PD Solutions Preclinical Safety & Toxicology Solutions Bioanalytical Services Drug-Type Specific Development Services

Target Identification & Validation

  • Use RNA-seq, scRNA-seq, and phosphoproteomics to map abnormal pathways in blood diseases (e.g., sickle cell anemia, hemophilia, thalassemia).
  • CRISPR/Cas9 screening in iPSC-derived erythroid and megakaryocyte cells to find druggable targets.
  • Validate targets using primary HSC cultures, platelet adhesion tests, and clot retraction models.
  • AI-based prioritization using multi-omic data, disease networks, and cross-species conservation.
  • Confirm mechanisms with DARTS, CETSA, and SPR assays for small molecules and biologics.

Preclinical Efficacy Solutions

Disease-Specific Efficacy Profiling

  • Test therapies in disease models (e.g., Factor VIII/IX knockout mice, β-globin mutants)
  • Assess efficacy via hemoglobin stability, clotting factor activity, and RBC flexibility
  • Validate findings in both rodent and non-rodent models

Mechanistic Pharmacodynamic Analysis

  • Monitor platelet aggregation, thrombin generation, and RBC integrity using microfluidics and flow cytometry
  • Measure complement activity and Fc receptor-mediated clearance in autoimmune models
  • Track treatment effects via CBC, reticulocyte counts, and iron metabolism markers

Advanced Modality Support

  • Screen viral vector performance in iPSC-derived cells
  • Evaluate HSC engraftment, erythroid differentiation, and durability in vivo
  • Correlate PK/PD for antibody therapies targeting platelets or complement proteins

Preclinical PK/PD Solutions

Blood-Focused ADME Profiling

  • Assess oral, sublingual, and RBC-specific absorption for blood disorder drugs
  • Measure drug distribution in bone marrow, spleen, and RBCs via tracers or LC-MS/MS
  • Evaluate metabolism using CYP450 assays in liver and blood matrices

Exposure-Response Correlation

  • Link drug levels to outcomes (e.g., Factor VIII restoration, RBC sickling reduction)
  • Use AI to model PK/PD relationships and predict long-term effects
  • Determine optimal dosing for chronic anemias and anticoagulants

Translational Dose Regimen Design

  • Use animal data to estimate human doses for IVIG or iron chelators
  • Simulate steady-state levels in transfusion models
  • Adjust dosing for pediatric patients using juvenile animal models

Preclinical Safety & Toxicology Solutions

Hematologic-Specific Toxicology Profiling

  • Conduct dose-finding studies in disease models, focusing on bleeding and RBC stability
  • Compare PK and toxicology in rodents and dogs to track drug buildup in blood-rich tissues
  • Define safety margins using platelet counts, hemoglobin levels, and coagulation times

Hematologic Safety Pharmacology

  • Monitor cardiovascular risk with ECG and blood pressure in guinea pigs
  • Screen for CNS and respiratory side effects in rodents
  • Use TEG/ROTEM to detect clotting abnormalities

Immunotoxicity & Genotoxicity Profiling

  • Test complement activation (CH50, C5b-9) for biologics
  • Detect drug-induced anti-platelet antibodies via flow cytometry
  • Run genotoxicity assays (Ames, MLA, micronucleus) in hematopoietic cells

Target Organ Toxicity Identification

  • Examine bone marrow for cellularity and iron deposits
  • Evaluate spleen and liver weight, iron levels, and macrophage activation
  • Test endothelial safety using von Willebrand factor staining and permeability assays

Advanced Therapeutic Safety Support

  • Study vector distribution and shedding for gene therapies
  • Assess CRS risk and platelet clearance for biologics
  • Evaluate RBC membrane stability using fragility and Heinz body tests

Bioanalytical Services

Customized analytical solutions and biomarker profiling to support drug development in blood disorders.

  • Tailored assay development in plasma, whole blood, and bone marrow matrices
  • Use of hemolyzed, lipemic, or icteric samples to simulate conditions such as anemia and thalassemia
  • Evaluation of therapeutic effects via qPCR/NGS, flow cytometry, and single-cell genomics
  • PK/immunogenicity testing, target engagement assays, and monitoring of HSC-based therapies

Drug-Type Specific Development Services

Precision solutions for blood disorder therapies across modalities.

Small Molecules

  • Hematopoietic stem cell mobilization assays (CXCR4/SDF-1 axis modulation)
  • In vitro myelosuppression and cytotoxicity screening using human bone marrow cells
  • Red blood cell deformability and hemoglobin oxygen-binding assays
  • Iron chelation efficacy profiling in iron-overload anemia models
  • JAK/FLT3/BCR-ABL inhibitor activity in leukemic cell lines and xenograft models

Biologics

  • Monoclonal antibody cytotoxicity profiling (ADCC/CDC) in hematologic malignancy cell lines
  • Fc-engineered antibody half-life and biodistribution studies in rodent spleen/liver models
  • Thrombopoietin (TPO) and erythropoietin (EPO) mimetic assays in vitro and in vivo
  • Antibody-drug conjugate (ADC) payload release and target internalization kinetics in leukemia models

RNA-Based Therapies

  • siRNA-mediated gene silencing for hematologic oncogenes (e.g., BCL2, MYC)
  • Antisense oligonucleotide (ASO) efficacy in splicing disorders (e.g., β-thalassemia mutations)
  • Lipid nanoparticle (LNP) delivery optimization for marrow-targeted mRNA therapeutics
  • RNA stability and immune activation profiling in whole blood and PBMC cultures

Gene & Cell Therapies

  • Lentiviral/AAV vector transduction efficiency in CD34+ hematopoiietic stem cells
  • CRISPR/Cas9 editing validation in sickle cell disease and thalassemia models
  • Engraftment and multilineage differentiation in humanized bone marrow mouse models
  • In vitro off-target and insertional mutagenesis risk assessment for gene-modified cell products

Peptide-Based Agents

  • Thrombin receptor-activating peptide (TRAP) activity in platelet function assays
  • Protease-resistant erythropoietic peptide analogs for anemia correction
  • Fibrin-targeted peptides for thrombosis imaging and clot dissolution studies
  • Immune-modulatory peptides in autoimmune hemolytic anemia models

Targeted Drug Conjugates

  • CD33/CD123-targeted payload delivery in AML cell lines
  • Spleen-targeted ligand-drug conjugate distribution profiling
  • Antibody-peptide hybrid conjugates for enhanced marrow selectivity

Why Partner with Ace Therapeutics? Your Trusted Preclinical Contract Research Provider

Comprehensive Blood Disease Preclinical Expertise

  • Disease-Specific Focus: Specialization in hematologic disorders including hereditary anemias (e.g., sickle cell disease, thalassemia), coagulation disorders (e.g., hemophilia, von Willebrand disease), and immune-mediated conditions (e.g., autoimmune hemolytic anemia, thrombocytopenias).
  • End-to-End Solutions: Integrated workflows spanning target validation, mechanism-of-action studies, pharmacokinetic/pharmacodynamic (PK/PD) profiling, and safety pharmacology tailored to blood-specific therapeutics.

Frequently Asked Questions (FAQs)

What types of hematological diseases do you specialize in?

Ace Therapeutics focuses on a broad range of blood disorders. This includes various forms of anemia (e.g., iron deficiency anemia, aplastic anemia, hemolytic anemia), hemophilia (A and B), immune thrombocytopenia (ITP), sickle cell disease (SCD), thalassemia, and von Willebrand disease (VWD).

What kinds of preclinical models do you offer for hematology research?

A comprehensive suite of in vitro and in vivo models is offered. In vitro capabilities encompass advanced 2D and 3D cell culture systems, such as 3D bone marrow models and iPSC-derived hematopoietic cells. For in vivo studies, a range of species is utilized, including humanized mouse models and larger animal models (e.g., dogs, pigs, sheep, cynomolgus monkeys), which are carefully selected to best recapitulate specific human disease conditions.

How is the quality and translatability of preclinical data ensured?

Commitment to quality is paramount. Rigorous, scientifically defined protocols are employed for biospecimen collection and processing, aiming to ensure high-quality starting materials. Studies are designed with meticulous attention to detail, utilizing state-of-the-art equipment and advanced analytical techniques (e.g., multi-omics, high-throughput flow cytometry). The focus is on generating physiologically relevant data to enhance the translatability of preclinical findings to potential clinical outcomes.

Can drug development be supported from target identification through preclinical studies?

Yes, Ace Therapeutics provides end-to-end preclinical drug development solutions. Services span from initial target identification and validation to comprehensive efficacy, pharmacokinetics/pharmacodynamics (PK/PD), and safety/toxicology studies.

Are customized research solutions offered?

Absolutely. Ace Therapeutics prides itself on a collaborative partnership model. The scientific team works closely with each client to design tailored study protocols and solutions that precisely meet their unique research objectives and specific drug development needs.

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