Orphan diseases, known as rare diseases, individually affect less than 1 in 2,000 people. Despite their rarity, they have a significant collective impact, affecting an estimated 300 million people worldwide. There are more than 7,000 types of rare diseases, most of which begin in childhood and primarily affect children.
Recognizing the critical need for specialized drug development, Scantox is dedicated to establishing a robust preclinical drug development platform for various types of diseases. Services range from target identification to safety and toxicology studies needed for IND approval. Our experienced, multidisciplinary team utilizes advanced tools to customize drug development processes for small molecules, biologics, and gene therapies, addressing rare diseases’ unique and complex challenges.
End-to-End Preclinical Services
At Scantox, we employ integrated approaches using advanced in vitro and in vivo tools to evaluate drugs for orphan diseases. These tools accelerate preclinical studies and support the transition of promising treatments into First-In-Human trials.
Advanced Cell-based assays
Our in vitro capabilities encompass a range of cell systems, such as isolated neurons and cell lines crucial for screening novel compounds targeting disease-specific pathologies. We also offer disease-relevant cell systems, including iPS cells, patient-derived tissues, and primary cell lines. These resources enable us to develop tailored models and design precise assays for specific diseases.
Robust Animal Models
We complement our in vitro work with extensive in vivo model development, a cornerstone of Scantox services. Customers can choose from various transgenic and induced animal models, such as those used to elicit neuroinflammation experimentally. Our rodent models and precise study setups are crucial in selecting promising drugs for orphan diseases.
We maintain a range of ready-to-go in vivo models and routinely develop or setup customized models in mice and rats to address emerging research needs.
Examples of ready-to-go models by indication within the rare disease field:
- Neurodegenerative diseases
- Lysosomal storage disorders
- Pompe Disease
- Gaucher Disease
- Sanfilippo Syndrome
- Niemann-Pick Disease
- Fabry Disease: Read this article for more detailed information
- Other rare genetic diseases
State-of-the-Art Facilities
Scantox maintains breeding colonies for some of the models at our research facility. Additionally, we collaborate closely with well-known breeders of transgenic and non-transgenic rodents. Non-transgenic littermates are available as control animals needed for proper study design. Our facilities allow evaluations under Biosafety Level 2. De-risking packages include standard non-GLP exploratory studies in mice and rats. Advanced regulatory studies adhering to GLP standards ensure the highest quality and regulatory compliance.
Study Design & Readouts
Scantox offers custom-tailored study designs for all disease models to meet specific drug development needs. We work closely with clients to select the most suitable host species based on the drug’s nature and determine the best sample analysis methods.
In addition to animal model- and disease-specific readouts, we can include pharmacokinetic and pharmacodynamic measurements, safety assessments, and investigations of mechanisms of action bymonitoring critical biomarkers with customized and established analytical methods. Our bioanalysis department can analyze samples when required.
Team – Specialized Experts
The orphan disease development team comprises dedicated professionals, including study directors with specialized expertise in rare diseases and skilled animal technicians.
Known for their speed and effective communication, the team provides flexible solutions tailored to each project. Our collaborative and rigorous quality-driven approach ensures effective navigation through the complex preclinical stages of development.