Mitochondrial disease describes a diverse set of diseases that are caused according to their genetic and heterogeneous, all stemming from defects in mitochondrial oxidative phosphorylation. Enzyme replacement therapy (ERT) is a new therapeutic option for mitochondrial diseases, as it has the potential to restore the function of damaged mitochondria by specifically targeting enzyme deficiencies. Protheragen offers ERT development services for mitochondrial disease to advance research and development efforts in this critical area.
Our Services
ERT development service of our company addresses the needs of academic researchers, biotechnology companies, and pharmaceutical institutions involved in the design, optimizations, and preclinical assessments of therapeutics targeted at mitochondrial disorders. Our detailed knowledge and advanced infrastructure enable us to deal with a multitude of different fields and offer a comprehensive range of specialized services, thus supplying questions posed in the development of ERT for mitochondrial diseases.
Target Identification and Validation
The goal is to pinpoint enzyme targets linked to mitochondrial dysfunction using bioinformatics, genomics, and proteomics for validation, which would be performed in partnership with the clients.
Gene Cloning and Recombinant Protein Production
Have substantial experience relating to gene cloning, vector preparation, and expression of recombinant proteins in several expression systems such as mammals, bacteria, yeast, and insect cells.
Protein Purification and Characterization
The use of advanced purification techniques for the purification of recombinant enzymes to high levels of purity, stability, and biological activity for therapeutic use will be undertaken.
Formulation Development
Developed and optimized formulations to improve free energy of solvation and enhancement of the enzyme’s pharmacokinetics to ensure effective tissue targeting and delivery.
In Vitro Assays
In vitro, studies include a range of enzymatic assays, mitochondria function assays, cellular uptake as well as mechanism of action studies for ERT candidate to preclinically assess their safety and efficacy.
In Vivo Studies
The biological efficacy, pharmacokinetics, biodistribution, and safety of ERT candidates would be studied in appropriate animal models of mitochondrial disease for the preclinical studies.
Our Advantages
- Scientific Expertise
The wide-ranging knowledge of our scientists and researchers on preclinical drug development, enzyme therapeutics, as well as on mitochondrial biology enables effective guidance for clients throughout the entire ERT project cycle from planning to execution.
- Collaborative Partnership
Fosters productive working relations with clients to effectively identify and define their requirements and goals ensuring transparency, mutual respect, and effective communication at all phases of the partnership.
- Customized Solutions
Guarantees optimal results and desirable progression for the defined ERT development goals using custom ERT development solutions flexibly designed to meet each specific project scope and target requirements.
- Regulatory Compliance
Complies with quality and regulatory requirements for products and services to guarantee the safety, efficacy, and compliance of the ERT products while ensuring prompt granting of approval and conversion.
Protheragen provides development services for enzyme replacement therapy for mitochondrial disease to develop novel therapies for mitochondrial disorders. We harness scientific and technical know-how combined with partnerships, so clients accelerate ERT drug candidates' development. Please contact us to learn more about our services for ERT development for mitochondrial disease and how we can assist in your research and development activities.
