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As a leading provider of preclinical research services, Protheragen is dedicated to offering tailored diagnostic and therapeutic development solutions for rare movement and neurological disorders. We integrate biomarker discovery, target identification, disease modeling, and innovative drug development to address the complex medical needs of such conditions. Our mission is to accelerate the translation of scientific breakthroughs into targeted diagnostics and treatments, empowering researchers to fundamentally transform the management of rare diseases.

  • Rich Expertise and Experience
  • Cutting-Edge Technology Platforms
  • Excellent and Trusted Partner

Overview of Rare Movement and Neurological Disorders

Rare movement and neurological disorders represent one of the most intricate challenges in modern medicine. These conditions, including rare motor neuron diseases, rare musculoskeletal disorders, and rare neurological disorders, have low incidence rates. They are characterized by progressive motor impairments, declining cognitive abilities, and often early-onset symptoms, with complex and diverse underlying pathogenic mechanisms.

Genetic Mutations & Protein Dysregulation

Inherited or spontaneous mutations in genes critical for neuronal function, such as SOD1, C9ORF72, and SMN1, disrupt RNA metabolism, promote toxic protein aggregation (TDP-43, FUS), and impair protein homeostasis, ultimately leading to motor neuron degeneration and progressive functional decline.

Excitotoxicity & Oxidative Stress

Chronic overactivation of glutamate receptors triggers excessive calcium influx, overwhelming neuronal defenses. Coupled with mitochondrial dysfunction and reactive oxygen species (ROS) accumulation, this cascade accelerates motor neuron damage and cell death.

Neuroinflammation & Glial Cell Dysfunction

Activated microglia and astrocytes release pro-inflammatory cytokines (TNF-α, IL-6), creating a hostile microenvironment. Dysfunctional neuron-glia interactions further exacerbate neurodegeneration, forming a vicious cycle of inflammation and neuronal loss.

Axonal Transport Defects & Synaptic Failure

Motor neurons rely on efficient axonal transport to maintain synaptic connections with muscles. Impaired transport of mitochondria, vesicles, and other cargo disrupts neuromuscular junction (NMJ) maintenance. As axonal integrity deteriorates, muscles lose innervation, resulting in atrophy and progressive paralysis.

Overview of Rare Movement and Neurological Disorders

Challenges in Rare Movement and Neurological Disorder Management

The diagnosis and treatment of rare movement and neurological disorders face significant hurdles, including prolonged diagnostic delays due to symptom overlap with common conditions and limited access to specialized testing. Additionally, the lack of validated biomarkers, small patient populations for clinical trials, and insufficient disease-modifying therapies create barriers to effective care. High treatment costs and heterogeneous disease presentations further complicate personalized management, leaving many patients without targeted therapeutic options.

Diseases in Focus

To address key challenges in the field of rare movement and neurological diseases, Protheragen focuses on high-need disease areas where precision intervention can have a transformative impact. We prioritize diseases with clear molecular targets or high unmet need and provide customized diagnostic and therapeutic development services to promote effective management of these complex diseases.

Rare Motor Neuron Diseases

Rare Motor Neuron Diseases (MND)

Rare motor neuron diseases are characterized by the progressive degeneration of motor neurons, leading to muscle weakness, paralysis, and respiratory failure. These diseases often involve genetic mutations that disrupt neuronal function, protein homeostasis, and axonal transport.

Rare Musculoskeletal Diseases

Rare Musculoskeletal Diseases

Rare musculoskeletal diseases encompass a group of genetic and acquired disorders affecting muscles, bones, joints, and connective tissues, often presenting with progressive weakness, deformities, and mobility impairments.

Rare Neurological Diseases

Rare Neurological Diseases

Rare neurological diseases include a group of disorders that affect the central and peripheral nervous systems and are usually caused by genetic mutations or autoimmune dysfunction. These diseases often have complex symptoms, including movement impairments, cognitive decline or seizures.

One-stop Solution for Diagnostic & Therapeutic Development

At Protheragen, we provide comprehensive end-to-end solutions to expedite advancements in the realm of rare movement and neurological disorders. Backed by a team of seasoned experts and cutting-edge technology, we are steadfast in delivering holistic support for our clients’ research endeavors, seamlessly bridging the transition from early discovery to preclinical validation.

Diagnostic Development Services
Amino Acid Metabolism Defects
Disease Model Development Services
Preclinical Research Services
Clinical Trial Services

Featured Products for Cutting-Edge Research

To accelerate research into rare movement and neurological diseases, Protheragen offers a specialized portfolio of high-purity biologics. Our specialty products are validated and optimized while adhering to strict ethical and regulatory standards to replicate human disease pathology with unparalleled accuracy. All biologic products have strict temperature control and traceability protocols to maintain functionality and compliance.

Cell Lines

Protheragen's cell lines offer physiologically relevant in vitro models derived from patient samples or genetically engineered.

  • Neuronal Cell Lines
  • Glial Cell Lines
  • Brain Endothelial Cell Lines
  • Neural Stem Cell Lines
  • Neuromuscular Cell Lines

Tissue Samples

We provide well-characterized human tissue samples that are carefully preserved to ensure optimal biospecimen integrity for advanced research applications.

  • Brain Tissue Samples
  • Spinal Cord Tissue Samples
  • Bone Tissue Samples
  • Muscle Tissue Samples

Cerebrospinal Fluid (CSF)

Disease-specific CSF samples provided by Protheragen are rigorously quality controlled to ensure low microbial contamination. These samples are collected under IRB-approved protocols and can be used to reliably study CNS biomarkers.

  • Human CSF Biospecimens
  • Processed CSF Derivatives

Advanced Technology Platforms for Next-Generation Therapeutic Development

Protheragen provides one-stop R&D services for rare allergic diseases.

EndureCART™

EndureCART™ platform pioneers next-generation CAR-T cell therapies specifically engineered for rare neurological disorders, featuring enhanced blood-brain barrier penetration and CNS-specific targeting.

  • Brain Tumor Treatment: Our novel CAR constructs demonstrated superior tumor infiltration and significant size reduction in aggressive gliomas while maintaining an excellent safety profile.
  • Autoimmune Encephalitis Treatment: In autoimmune encephalitis, EndureCART™ delivers CSF-persistent, B-cell/plasma-cell-targeted CARs that directly clear pathogenic autoantibodies.

AAVLink™

AAVLink™ platform leverages engineered adeno-associated virus (AAV) vectors to enable precise and efficient gene delivery for rare neurological disorders, overcoming traditional challenges of off-target effects and immune clearance.

  • Gene Replacement: Deliver functional gene copies using cell-type-specific AAV vectors to restore protein expression in affected neurons and glia, addressing the root cause of monogenic disorders.
  • Gene Silencing: Enable CNS-targeted knockdown of toxic genes through optimized AAV-delivered shRNAs or antisense oligonucleotides.
Protheragen provides one-stop R&D services for rare allergic diseases.
Protheragen provides one-stop R&D services for rare allergic diseases.

GeniusAb™

GeniusAb™ is a cutting-edge platform specializing in the discovery and engineering of fully human single-domain antibodies (sdAbs).

  • Toxic Protein Clearance: sdAbs selectively bind and neutralize neurotoxic aggregates (e.g., tau, α-synuclein) with picomolar affinity, thereby enhancing clearance via microglial phagocytosis while sparing the native protein.
  • Receptor Modulation: Our allosteric sdAbs precisely regulate disease-relevant CNS receptors through conformation-specific targeting, restoring synaptic function without systemic side effects.

ImmuBridge™

ImmuBridge™ is an innovative platform specializing in the design of multispecific antibodies and next-generation T-cell engagers (TCEs) that precisely redirect T cells to tumors through customizable targeting architectures.

  • Glioblastoma & CNS Lymphoma Treatment: Multispecific TCEs with blood-brain barrier (BBB) permeability and hypoxia-responsive activation capabilities enable greater tumor selectivity while minimizing off-target neurotoxicity.
  • Pediatric Brain Tumor Treatment: For high-risk pediatric cancers like DIPG and medulloblastoma, ImmuBridge™ designs H3K27M- and B7-H3-targeted TCEs with enhanced CNS delivery.
Protheragen provides one-stop R&D services for rare allergic diseases.

Can’t find the service you’re looking for? Don’t worry!

Our team is equipped to provide you with customized research solutions tailored to your specific needs.

Contact Us for Personalized Support

Why Choose Us?

Deep Rare Disease Expertise

Cutting-Edge Technology

Standardized Service Workflow

End-to-End Solutions

FAQs

How do you ensure translational relevance in preclinical studies?

Protheragen specializes in connecting preclinical research with clinical applications by utilizing human-like model systems such as patient-derived iPSCs and humanized rodents. They focus on aligning their research with clinical goals by employing quantitative imaging, CSF biomarkers, and functional assessments.

Can you support IND-enabling studies for orphan drugs?

We provide end-to-end preclinical solutions tailored for orphan drug development, including toxicology studies, biodistribution analysis, and dose optimization designed specifically for CNS targeted therapies. Our team specializes in the unique challenges of rare neurological diseases and has extensive experience in preparing regulatory-ready packages.

What’s your turnaround time for in vivo efficacy studies?

Our standard timelines for in vivo efficacy studies typically range from 8-16 weeks, depending on the model complexity and study endpoints. The exact duration is project-specific and determined by factors such as disease progression rates, treatment protocols, and required readouts. We’ll provide a customized timeline after evaluating your study design.

Do you provide regulatory submission support?

Yes, we offer end-to-end regulatory support for preclinical programs, including study design consultation, GLP-compliant documentation, and preparation of IND-enabling data packages tailored to regulatory requirements. Our team assists with protocol development, final study reports, and strategic guidance to facilitate smooth regulatory transitions.

What biomarkers can you monitor in preclinical studies?

Protheragen's preclinical studies incorporate comprehensive biomarker profiling, including neurodegeneration markers (NfL, tau, GFAP), neuroinflammation indicators (IL-6, TNF-α, complement factors), and therapy-specific biomarkers (AAV genome copies, CAR-T persistence). We utilize CSF/serum analysis, imaging modalities, and digital pathology to quantify target engagement and treatment response. Custom panels can be developed to align with your mechanism of action.