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Drug Repurposing Service

Drug Repurposing Services for Rare Diseases

The process of repurposing drugs for new indications is a time- and cost-efficient approach with a higher success rate than developing new orphan drugs, thus significantly reducing the risk of drug development for rare diseases. Our company has an integrated technology platform to offer customers drug repositioning services and screening services of these compounds for new therapeutic purposes, accelerating the development of new therapies for rare diseases.

Drug Repurposing Screening for Rare Diseases

There are approximately 7,000 identified rare and neglected diseases, the vast majority of which lack effective treatments. The development of new treatments for rare diseases is a major challenge because the drug discovery process is often laborious, expensive, and prone to failure, it is particularly unappealing for rare diseases that affect only a small number of people and thus generate reduced profits. Drug repurposing has been proposed as an alternative strategy for the development of new therapies, particularly in the field of rare diseases. It offers lower risks, lower costs, and shorter timescales than the development of novel orphan drugs.

Drug repurposing, also known as drug repositioning or drug reprofiling, is the process of redeveloping compounds for use in different diseases. The strategy is based on the scientific principles that a single drug often interacts with multiple targets or pathways, and multiple drugs may act on the same target or pathway. With the Human Genome Project providing a wealth of genetic information on many rare diseases, including gene regulation, protein structure, and drug-target interactions, scientists have the opportunity to make breakthrough achievements in the field of drug repurposing.

Fig. 1 Graphic representation of drug repurposing.

Fig. 1 Graphic representation of drug repurposing. (Hua Y, et al., 2022)

Rare Disease Drug Repurposing Services

With extensive knowledge and advanced technologies in the areas of big data analysis, computer models, and high-throughput screenings, our company has the ability to help customers conduct more systematic, organized, and data-oriented searches for candidates. In addition, combined with the wealth of information obtained from multi-omics analysis of rare diseases and the available gene annotations of human and model organisms, we assist customers in examining newly identified rare disease-causing genetic defects to see whether they share the same pathways or biological processes as other diseases with approved treatment options, thereby accelerating drug repurposing for rare diseases. Our services include, but are not limited to:

  • Binding assays to identify target candidates for rare diseases
  • We provide our customers with the analysis of all possible binding targets of drug candidates by techniques such as chromatography and mass spectrometry.

  • Phenotyping screening for rare diseases
  • We provide our customers with screening services for target drug candidates through cell-based screening involving cell lines derived from human or induced pluripotent cell lines (iPSC), etc. Through high throughput screening (HTS) technology, we are able to help customers screen thousands of chemical drug libraries at once.

  • Artificial intelligence (AI) services
  • We apply genetic and molecular information on a wide range of complex rare diseases through machine learning (ML) algorithms to help our customers predict whether compounds are likely to be used in new indications.

By adding our team to your drug repurposing program, you will:

  • Give new meaning to drug candidates by quickly identifying new indications for a wide range of disease states.
  • Quickly find new markets by using our platform that integrates multiple assays across dozens of rare disease states.
  • Guide the repurposing of drug candidates through our personalized data analysis and interpretation.

Rare Disease Drug Repurposing Solutions

  • Pathway mapping/pathway-based drug repurposing for rare diseases
  • Target mechanism-based drug repurposing for rare diseases
  • Network strategy-based drug repurposing for rare diseases
  • Activity-based repurposing for rare diseases

Our company has a dedicated and highly educated team of researchers who understand rare diseases and the complexities behind drug repurposing. We offer drug repurposing services to our customers to help them identify potential treatments for rare diseases more quickly. If you are developing a therapy for a rare disease, please contact us to find out more about how we can help you make the most of your development program.


  • Hua, Y.; et al. Drug repositioning: Progress and challenges in drug discovery for various diseases. European journal of medicinal chemistry, 2022: 114239.

For Research Use Only.

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