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Project Description

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

In Vivo Efficacy Evaluation of RMT-Targeting Biologics

Inquiry

The blood-brain barrier (BBB) restricts the passage of most molecules, particularly large or polar molecules such as biotherapeutics. Therefore, fewer biologics are in development for the central nervous system (CNS) disorders compared to other therapeutic indications. One solution that has been utilized for CNS delivery of biotherapeutics is receptor-mediated transcytosis (RMT), which takes advantage of receptors abundantly expressed on brain endothelial cells to facilitate transport across the BBB. Evaluating therapeutic efficacy in vivo is a critical step in the preclinical development of RMT-targeting biologics. By using disease-relevant animal models and measurable functional outcomes, these studies provide essential data to de-risk CNS targeting programs and support translational development.

Fig. 1 The impact of 13C3a and TBTI antibodies on brain Aβ levels in APPSL mice. (Do, *et al*., 2020)

Our Services

At Ace Therapeutics, we offer comprehensive in vivo services designed to evaluate the therapeutic efficacy of RMT-targeting biologics. Our platform integrates disease-relevant animal models, robust pharmacological assessment tools, and CNS-specific endpoints to support your preclinical development.

Available Animal Models

We provide a wide range of well-established and customized animal models, including:

Neurodegenerative disease models	Alzheimer's disease (e.g., APP/PS1, 5xFAD, Tauopathy models) Parkinson's disease (e.g., MPTP, 6-OHDA) Huntington's disease (e.g., R6/2, zQ175)
Stroke and cerebrovascular disease models	Transient MCAO, photothrombotic models, bilateral carotid artery stenosis (BCAS)
Other CNS models	Multiple sclerosis Epilepsy Spinal cord injury Neuronopathic lysosomal storage disease

Efficacy Readouts

We can provide multiple readouts to evaluate the therapeutic efficacy of RMT-targeting biologics.

Behavioral and functional tests	Cognitive testing: Morris water maze, Y-maze, novel object recognition test, etc. Motor function tests: rotarod test, grip strength test, open field test, etc. Neurological scores and survival analysis in disease progression models
Histopathological and immunohistochemical analysis	Analysis of pathological features (e.g., amyloid and tau pathology) Assessment of neurodegeneration, demyelination, gliosis, and microglial activation Immunostaining of disease-relevant markers in brain tissues(e.g., NeuN, Iba1, GFAP, MBP)
Biochemical and molecular readouts	Cerebrospinal fluid (CSF) and plasma sampling for translational discovery Quantification of target engagement and downstream biomarkers in brain tissue and biofluids (e.g., Aβ42, p-Tau, neurofilament light chain, pro-inflammatory cytokines) ELISA, Western blot, qPCR, and multiplex immunoassays for molecular validation

Our Advantages

CNS-focused expertise: Our team specializes in CNS disease modeling and has a deep understanding of the unique challenges associated with RMT-targeted biologics.
Customizable study design: We design studies based on clients' specific target, payload, and disease indication, with flexibility in endpoints and model selection.
Comprehensive endpoints: From behavioral and molecular assessments to imaging-based analyses, we provide multi-level evaluations to generate robust efficacy data for RMT-targeted biologics.
End-to-end support: From animal model selection and study design to data interpretation and reporting, we offer full-service support to accelerate the development of RMT-targeted biologics.

With Ace Therapeutics' in vivo efficacy evaluation services, we aim to provide clients with the critical data needed to develop their RMT-targeting biologics. Contact us today to learn more about our RMT-based brain drug delivery services.

Reference

Do, T. M., et al. (2020). Tetravalent bispecific tandem antibodies improve brain exposure and efficacy in an amyloid transgenic mouse model. Molecular Therapy Methods & Clinical Development, 19, 58-77.