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Rare Disease Therapeutics Development Services

Therapeutics development covers the entire process from drug discovery to final approval for commercialization, each of which requires rigorous experimental verification and regulatory review. Our company provides a full range of services and complete experimental platforms to help you accelerate the drug therapeutics development in rare diseases.

Therapeutics Development for Rare Diseases

Although thousands of types of rare diseases have been identified, effective treatments for these excruciating, and even life-threatening, conditions are far from meeting the demand, which has resulted in a significant number of patients not receiving timely care. However, due to low incidence, high dispersion, and difficulty in diagnosis, the development of corresponding therapeutics still faces many challenges.

Rare Disease Therapeutics Development Services-1Fig.1 Opportunities and Challenges in developing orphan drugs to prevent, diagnose and treat rare diseases. (Bhattacharjee, S., and Nandi, S., 2018)

Enabling people with rare diseases to receive accurate diagnosis, care, and effective treatment in the shortest time possible is an aspirational goal for all medical researchers. The development of therapeutics for rare diseases means providing more hope for patients to alleviate their suffering.

  • Rare Disease Mechanism Discovery
    Rare diseases are often caused by defects in a single gene. Finding the causes of these defects is very complex and difficult, and requires combining knowledge from multiple fields such as genetics, molecular biology, biochemistry and pathology to reveal the etiology and mechanism of rare diseases.
  • Access to Diagnosis of Rare Disease
    The application of technologies such as genome sequencing and mutation analysis has greatly increased the efficiency of rare disease diagnostic research and development, improved the diagnostic path for rare diseases, and provided a basis for the development of rare disease therapies.
  • Therapeutic Development for Rare Disease
    Therapeutic methods such as small molecules, monoclonal antibodies, protein replacement therapies, oligonucleotides, and gene and cell therapies are accelerating the development of rare disease therapies and are expected to provide more practical treatment options for patients with rare diseases.

Our Services

With the rapid growth of rare disease therapeutics technology, our company has established a mature therapeutics development process system to provide you with an experienced and high-level team and the necessary resources to support the development of rare disease therapeutics.

Rare Disease Therapeutics Development Services-2

Rare Disease Target Identification

  • Mosaic Variation Identification
  • Mitochondrial Disorders Gene Identification
  • X-linked Rare Disease Gene Identification
  • Rare Autosomal Recessive Disease Gene Identification
  • Rare Autosomal Dominant Disease Gene Identification
Rare Disease Therapeutics Development Services-6
Rare Disease Therapeutics Development Services-6
Rare Disease Therapeutics Development Services-6

Our Advantages

  • Time-saving services with high efficiency
  • Professional and experienced multidisciplinary experts
  • All-round project designing and reporting
  • Numerous service cases and customer praise
  • Cost-effective compared to other companies
  • Superior data quality and fast turnaround

With the participation of multidisciplinary researchers with extensive project experience, our company is committed to providing one-stop development services to help you accelerate the identification and validation of therapeutics for rare diseases. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Bhattacharjee, S., and Nandi, S. "Rare genetic diseases with defects in DNA repair: opportunities and challenges in orphan drug development for targeted cancer therapy." Cancers 10.9 (2018): 298.

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

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