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Small Molecule Drug Development Platforms

Small Molecule Drug Development Platforms-1

Small molecule drugs are increasingly used in rare diseases. Our company launched a small molecule drug development platform, which can provide comprehensive services from small molecule drug discovery to preclinical studies for rare diseases. We have rich experience helping global pharmaceutical companies and research institutions offer small molecule drug development.

Small Molecule Drugs for Rare Diseases

Small molecule drugs are the most common drugs and are molecularly defined chemical entities of low molecular weight ranging from 0.1 to 1.0 kDa. With the increasing prevalence of rare diseases year by year, the total number of patients with rare diseases worldwide is nearly 400 million. Therefore, drug discovery and innovative therapies for rare diseases are urgent. There are clear opportunities for the development of small-molecule drugs for rare diseases.

Small molecule drugs are organic compounds that affect molecular pathways by targeting essential proteins that are low in molecular weight and easily penetrate cells. The drug development process of small molecule drugs includes identification and validation of initial targets, screening of lead compounds, method development and modeling, pharmacokinetic characterization, preclinical safety assessment, selection of preclinical drug candidates, and clinical studies.

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Fig.1 Small molecule discovery and development for rare disease. (Schmidt D, et al., 2020)

With the development of pharmaceutical technology, there are various types of drugs and innovative therapies for rare diseases. Small molecule drugs are very attractive for the treatment of rare diseases.

Our Platforms for Small Molecule Drug Development

Our company employs talented and highly trained scientists who focus on all aspects of preclinical research of small molecule drugs. Combined with our unique platform, we can provide comprehensive, high-quality, personalized small molecule R&D services to global partners.

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PROTAC Development Platform

The development of PROTAC bifunctional small molecules for targeted protein degradation is becoming a new therapeutic method for rare diseases. Our company has a complete PROTAC development platform and is committed to providing customers with comprehensive PROTAC development solutions. Our rich experience and professional knowledge will help you more quickly achieve the research and development of rare disease drugs.

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Molecular Glue Degraders Development Platform

Our company has an experienced team of experts who can provide you with complete rare disease therapy and drug development solutions. At the same time, we have rich experience in the field of protein degradation. Our molecular glue degraders development platform can provide you with professional and comprehensive services for rare disease research.

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SMDC Development Platform

Small molecule-drug conjugates (SMDCs) have shown great potential as a new class of targeted drugs for the therapy of rare diseases. Our company is driven by research and innovation, and has extensive experience and technology in drug repurposing to accelerate drug discovery for rare diseases. We have launched a new SMDC one-stop platform to provide you with more professional services.

Capabilities of Our company

  • Synthesis of various compounds
  • Isolation and purification of compounds
  • Complete chemical technology platform
  • Customizable experimental services
  • One-stop platform with an experienced technical team

Project Workflow

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With a research team with extensive expertise in small molecule development as well as an advanced platform, our company is confident to provide customers with small molecule development services for rare diseases. We customize personalized solutions and analysis processes according to your different scientific research needs and track the progress of projects in real-time to accelerate your scientific discovery. If you are interested in our platform, please contact us for more details.


  • Schmidt, D.; Thompson, C. Case studies in rare disease small molecule discovery and development. Bioorganic & Medicinal Chemistry Letters, 2020, 30(21): 127462.

For Research Use Only.

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