Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by the loss of motor neurons and a gradual decline in motor function. Robust preclinical models are essential for understanding disease progression and evaluating potential therapeutic interventions. Transpharmation provides ALS preclinical services centered on the well-established SOD1 G93A model, supporting longitudinal efficacy studies with behavioral and clinically relevant biochemical endpoints designed to generate decision-relevant data for drug development.

Detail of offering

Transpharmation has experience working with both mouse and rat SOD1 G93A transgenic models of ALS. These models develop a progressive neurological motor deficit that can be monitored over time, enabling the assessment of disease onset, progression, and treatment effects.

Our ALS studies are typically designed as longitudinal efficacy programs and can incorporate:

  • Daily clinical observations to monitor disease progression and general health status.
  • Motor function assessments to quantify neurological decline and treatment-related effects.
  • Flexible study designs tailored to therapeutic objectives and development stage.
  • Integration with Transpharmation’s wider behavioral, biomarker, EEG, and histology capabilities where appropriate.

The progressive nature of the SOD1 G93A model allows repeated measurements throughout the study, providing a framework for evaluating therapeutic efficacy against relevant functional endpoints. Experience across both mouse and rat models enables selection of the most appropriate study design based on scientific objectives and program requirements.

Coming soon: a TDP-43-based model. We are characterizing a TDP-43-based mouse model to expand our service offerings into another translational ALS mechanism to better address preclinically the multifactorial pathology present in ALS patients.

Platforms used

Have a program in mind?

Speak with our team to discuss your ALS program, study objectives, and preferred endpoints. We can help design a SOD1 G93A efficacy study aligned to your development strategy and update you with our progress on the TDP-43-based model.

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