|Report Code: PP10212||Published: September 2019||Pages: 133||Available format:|
|Therapeutic Area(s):||Oncology | Immunology | Metabolic | Respiratory | Infectious | Ophthalmology | Gastroenterology | Nephrology | Neurology | Urology||Report Type: Mechanism of Action Reports|
Mitochondria (singular: mitochondrion) are cytoplasmic organelles found in eukaryotic cells, such as plants, animals, fungi, and protists. The number of mitochondria present in a eukaryotic cell depend on the metabolic requirements of that cell; and ranges from a single large mitochondrion to thousands of the organelles. Mitochondria generate adenosine tri-phosphate (ATP) through oxidative phosphorylation, and also plays a key role in apoptosis., Mitochondrial therapeutic development is focused on diseases, which are caused by mutations in mitochondrial deoxyribonucleic acid (DNA) or in nuclear gene encoding mitochondrial protein.
Mitochondrial disorders are diagnosed by complete physical examination; neurological examination; metabolic examinations, including urine and blood tests; reviewing a patient’s family history; and cerebral spinal fluid test.
Currently, there is no therapy approved for the treatment of disease caused by mitochondrial dysfunction. However, several mitochondrial-based drugs are under clinical trials for treating a range of diseases. Stealth BioTherapeutics Inc.’s drug candidate, Elamipretide, has reached the late stage of clinical development.
Further, technological advancement is a major factor that supports the drug development process. Due to advancements in drug development technologies, many pharmaceutical companies are leveraging improved and advanced technologies for the development of mitochondrial-based therapeutics.
These technologies are helpful in the development of drugs either in the form of combination therapies or single-agent therapies. They allow development of specific targeted therapies with better drug delivery. For instance, CohBar’s technology platform is specifically designed to rapidly identify therapeutically relevant peptides encoded within the mitochondrial genome. This further helps in evaluating their biological activity and developing these peptides into novel mitochondrial-based therapeutics that have the potential to treat diseases with major unmet medical needs.
As of August 2019, the mitochondrial-based therapeutics pipeline comprises of 27 drugs in different stages of development.
MITOCHONDRIAL-BASED THERAPEUTICS PIPELINE AND DRUG CANDIDATES (2019)
Some of the key players involved in the development of mitochondrial-based drugs are Stealth BioTherapeutics Inc., CohBar Inc., and Astellas Pharma Inc.
Some highlights of the Mitochondrial-Based Therapeutics Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations and Other Developments report are as below: