Mitochondrial diseases present with a wide range of clinical symptoms, varying age of onset and progression. The most affected tissues are those that heavily rely on oxidative metabolism, including the heart, skeletal muscle, brain, and retina. Symptoms often involve multiple organs and exhibit diverse manifestations. As a leading preclinical contract research organization (CRO) specializing in mitochondrial disease research, Protheragen is dedicated to providing comprehensive cell therapy development services tailored to address the unique challenges associated with mitochondrial diseases.
Cell Therapies in Mitochondrial Disease
The advancement of research has paved the way for promising cell-based strategies in the treatment of diverse mitochondrial disease.
| Method |
Disease |
| Erythrocyte-encapsulated thymidine phosphorylase |
Mitochondrial NeuroGastroIntestinal Encephalomyopathy (MNGIE) |
| Allogenic HSCT |
Mitochondrial NeuroGastroIntestinal Encephalomyopathy (MNGIE) |
| Liver transplantation |
Ethylmalonic encephalopathy (EE) |
Our Services
The scope of our cell therapy development services encompasses a diverse range of activities aimed at expediting the translation of promising therapies into efficacious treatments for mitochondrial diseases.
Cell Line
Development
We specialize in the generation and optimization of cell lines for mitochondrial disease research. Our team utilizes advanced techniques to develop disease-specific cell lines that accurately mimic the physiological conditions of the target organ or tissue affected by mitochondrial disease. These cell lines serve as valuable tools for preclinical studies and drug screening.
Cell-Based Assay
Development
We design and validate cell-based assays to evaluate the efficacy and safety of potential cell therapies for mitochondrial diseases. Our assays focus on assessing key cellular functions affected by mitochondrial dysfunction, such as energy production, oxidative stress, and mitochondrial dynamics. These assays provide valuable insights into the therapeutic potential of candidate cell therapies.
Cell Therapy
Manufacturing
We specialize in the development and optimization of manufacturing processes for cell therapies, providing expertise in ensuring scalability and reproducibility while adhering to stringent quality control standards. Our team customizes manufacturing protocols that meet regulatory requirements, facilitating the successful translation of cell therapies from laboratory settings to preclinical trials.
Preclinical Efficacy
and Safety Studies
Our comprehensive preclinical studies assess the efficacy and safety of cell therapies for mitochondrial diseases in relevant animal models. We conduct rigorous evaluations of treatment outcomes, including functional improvements, tissue regeneration, and immune response modulation. These studies provide crucial data for regulatory submissions and support the progression of cell therapies into preclinical trials.
Our Advantages
Professional Team
Advanced Technology
Comprehensive Services
Reliable Data and Results
At Protheragen, we are committed to advancing the development of cell therapy for mitochondrial diseases. Our extensive range of services, coupled with our expertise in mitochondrial disease research, enables us to provide comprehensive support to our clients in developing innovative and efficacious treatments. Contact us today to explore how our cell therapy development services can assist you in your endeavor to combat mitochondrial diseases.
