Development of Small Molecule Therapies for Rare Diseases
Small molecules are still in the mainstream of drug discovery. Our company is driven by research and innovation and focuses on small molecule drug discovery services for rare diseases. We provide comprehensive technical services to global pharmaceutical companies and research institutions to discover small molecules with good pharmacological and pharmacokinetic effects and low off-target effects.
Small Molecule Therapies for Rare Diseases
Small molecules are often a well-established and promising drug platform for a wide range of diseases, due to their multiple routes of administration, stability, controlled dosing, the scale of synthesis, and generally low cost of preparation. Small molecule drugs have made some progress in the field of rare diseases and have become the most established technology in the development of drugs for rare diseases. The discovery of small molecule drugs for rare diseases is mainly based on large-scale screening of model cell lines, followed by homolog optimization and optimization of absorption, distribution, metabolism, and other properties of the screened active compounds in order to obtain marketed drugs with better clinical effects.
In the treatment of rare diseases, small molecule drugs have clear advantages, such as broad targeting and ease of production. The huge scientific research base and the experience of clinical and regulatory experience with small molecules also facilitate the study of new molecules compared to other approaches. Furthermore, the potential of finding small molecule drugs with desirable efficacy through phenotypic screening, even when based on unknown novel mechanisms, may also be an advantage for rare diseases with unknown molecular etiology or multiple causative factors.
Fig. 1 Small molecule discovery and development for rare disease. (Schmidt D, et al., 2020)
Rare Disease Small Molecule Drug Development Services
Our company provides a complete one-stop service from small molecule screening, synthesis, drug analysis to formulation development to accelerate our customers' small molecule drug development for rare diseases. Our services include, but are not limited to:
- Small molecule drug discovery services
- Small molecule screening services
- Small molecule bioassay services
- Biomarker development services
Small molecules were able to affect their targets by covalent modification, inducement of alternative splicing, stop codon readthrough, allosteric activation, along with more a traditional approach, kinase inhibition. Our research team helps customers develop therapeutics for rare diseases based on the diversity of mechanisms of action employed by small molecules.
We offer high-throughput screening services to rapidly identify promising compounds.
We have established a variety of robust bioanalytical methods such as LC/MS (HPLC, UPLC, 2-D chromatography), HPLC/UV, and HPLC/fluorescence technologies to provide industry-leading small molecule bioanalytical services for drug development in rare diseases.
Our experts in biomarker identification and analytical validation provide customers with biomarker screening services and the information they need to advance their research.
Rare Disease Small Molecule Therapy Development Solutions
We are committed to providing our customers with comprehensive solutions for the development of small molecule therapies for rare diseases. Our solutions include, but are not limited to:
- Development of therapeutic small molecules based on cellular screens defined by potential mutations in the CFTR gene, which lead to defective protein production, trafficking, function, misfolding, or premature degradation, providing potential therapies for cystic fibrosis (CF) caused by different mutations.
- Development of small molecules that promote stop codon reading to provide possible therapies for rare diseases caused by mutations in specific genes, such as Duchenne muscular dystrophy (DMD) caused by stop codon mutations in the gene encoding dystrophin.
- Development of small molecule drugs that can be used to increase protein levels, providing potential therapies for rare diseases caused by inadequate protein production.
Our company's experienced scientists from a variety of disciplines and backgrounds provide significant intellectual and innovative input to offer rare disease small molecule drug development services to our customers. Our streamlined and integrated drug discovery process for identifying small molecules with suitable developmental properties has proven to be very successful for our partners. If you need our service and technical support, please contact us for more information.
Reference
- Schmidt, D.; Thompson, C. Case studies in rare disease small molecule discovery and development. Bioorganic & Medicinal Chemistry Letters, 2020, 30(21): 127462.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.