A multitude of genetic disorders with manifestations come under the spectrum of mitochondrial diseases due to defective cellular energy production. Because of the multifactorial nature of mitochondrial dysfunction and the complexity of its therapy, pharmacological safety evaluation in drug development is crucial for these disorders. Protheragen provides fully integrated safety pharmacology services designed specifically for the needs of drug developers who focus on treating mitochondrial dysfunctions.
Our Services
Protheragen coordinates and curates tailored safety pharmacology studies across a diverse range of animal models for mitochondrial disease, facilitating a comprehensive approach to mitigate risks associated with compounds impacting multiple organ systems. This enables informed decision-making during early preclinical development.
Cardiovascular System Safety Pharmacology Service
Blood pressure, heart rate, electrocardiograms (ECGs), ventricular and pulmonary pressure, and echocardiography.
Respiratory System Safety Pharmacology Service
Respiratory rate, hemoglobin oxygen saturation, and tidal and minute volumes, in vitro electrophysiology.
Central Nervous System (CNS) Safety Pharmacology Service
Functional observation battery (FOB), modified Irwin screen, body temperature, and large animal neurological evaluations, quantitative motor performance, CNS electrophysiology (EEG), and higher order neurofunctional endpoints.
In Vitro Electrophysiology Safety Pharmacology Service
Complete cardiac ion channel assessments (e.g., Na+, Ca2+, K+ or hERG), the comprehensive in vitro proarrhythmia assay (CiPA) battery includes assessment of drug effects on multiple human cardiac currents using appropriate voltage protocols, in silico reconstruction of the human cardiac action potential, and evaluation of in vitro effects in human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs).
Renal/Urinary Safety Pharmacology Service
Serum creatinine, blood urea nitrogen (BUN), glomerular filtration rate (GFR), inulin or creatinine clearance, kidney injury molecule-1 (KIM-1), neutrophil gelatinase-associated lipocalin (NGAL), and urinary albumin.
Gastrointestinal Safety Pharmacology Service
Gastric emptying, intestinal transit time, colonic motility, gastrointestinal adverse effects, gastric mucosal integrity and barrier function.
Our Advantages
- Expertise
In our team, we have experienced scientists who specialize in mitochondrial biology, pharmacology, and drug development. We design and execute safety pharmacology studies tailored to the specific requirements of mitochondrial disease research.
- Customized Solutions
We understand that every drug development program is unique, so we focus on customization to solve specific goals and challenges of mitochondrial diseases. Engagement and collaborate closely for creating specialized assays or developing robust risk mitigation strategies to fight and protect against mitochondrial dysfunction.
- Efficiency
In developing therapies for quite complex and sensitive diseases such as mitochondrial dysfunctions, keeping high-efficiency standards is of utmost importance. To achieve this, we adopt modern instruments and approaches that ensure timely and dependable outcomes and uphold stringent safety standards.
- Collaborative Partnership
Maintain a close relationship and work alongside our clients at every stage of drug development as holistic partners to tackle therapies for mitochondrial diseases. We are in constant open communication concerning study design and data interpretation to ensure positive outcomes.
With the use of our knowledge, custom-built assays, and collaborative approach, we enable our clients to chart their way through the complexities surrounding the development of a drug in a safe and efficacious manner. For more details on how we can assist you in developing drug frameworks for mitochondrial disorders, do not hesitate to contact us today.
