Protein Misfolding Inhibitor Development
Protein misfolding inhibitors are medications that prevent or alleviate protein misfolding. With our pioneering advancements in rare neurological diseases due to protein misfolding, Protheragen stands at the forefront of protein misfolding inhibitor development. We value your partnership and will provide unwavering assistance tailored precisely to meet your research needs.
Overview of Protein Misfolding Inhibitors for Rare Neurological Diseases
Many neurodegenerative disorders such as Alzheimer's disease (AD), Parkinson's disease (PD), and certain forms of amyotrophic lateral sclerosis (ALS) arise due to the fundamental pathological process of protein misfolding. These diseases appear to be associated with the inability of certain proteins to achieve their native three-dimensional structures, resulting in either a loss of function or gain of toxic properties. Protein misfolding inhibitors are targeted therapeutics intended to relieve or reverse damage done by proteins misfolding.
Fig.1 Protein misfolding inhibitors can block protein misfolding and prevent further damage. (Cheng B, et al., 2013)Mechanisms of Action of Protein Misfolding Inhibitors
Protein misfolding inhibitors function by preventing, correcting, or clearing misfolded proteins through diverse mechanisms. Stabilization of native structures of proteins, molecular chaperoning, and enhancement of clearance pathways along with correct folding and interference with aggregation of misfolded proteins are some of the functions these inhibitors can perform. Protein misfolding inhibitors strive to restore protein homeostasis by intervening prior to the pathological cascade.
Fig.2 Mechanisms involved in protein misfolding & therapeutic targets. (Sweeney P, et al., 2017)
Development of Protein Misfolding Inhibitors for Rare Neurological Diseases
| Drug Names | Indications | Targets | Mechanism of Action | Phase |
| Arimoclomol | ALS, Niemann-Pick type C | HSP70/90 | Chaperone-mediated protein refolding | Phase III |
| Trofinetide | Rett syndrome | IGF-1 receptors | Modulates protein synthesis and folding | Approved |
| Troriluzole | Spinocerebellar ataxia | SLC1A2 | Stabilizes neuronal proteostasis | Phase III |
| ANX005 | Guillain-Barré syndrome | Complement protein C1q | Antibody-mediated protein homeostasis | Phase II |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
Protheragen focuses on providing customized solutions for rare neurological disorders through leveraging novel research on misfolded proteins. With robust preclinical studies and intricate disease models, we strive to provide solutions that approach the mechanisms of protein misfolding to enable new treatments in this area of great difficulty.
Protein Misfolding Inhibitor Devlopment Services
Workflow of Protein Misfolding Inhibitor Development
Target Identification & Validation
Drug Screening and Optimization
In Vitro Characterization
In Vivo Efficacy Studies
Safety & DMPK Profiling
At Protheragen, we are pioneering next-generation therapeutics targeting rare neurological disorders driven by protein misfolding. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
- Cheng B, Gong H, Xiao H, et al. Inhibiting toxic aggregation of amyloidogenic proteins: a therapeutic strategy for protein misfolding diseases[J]. Biochimica et Biophysica Acta (BBA)-General Subjects, 2013, 1830(10): 4860-4871.
- Sweeney P, Park H, Baumann M, et al. Protein misfolding in neurodegenerative diseases: implications and strategies[J]. Translational neurodegeneration, 2017, 6: 1-13.