In glaucoma, mitochondrial dysfunction is also an important physiopathological process. Oxidative stress triggers neuronal death in glaucoma, suggesting that mitochondria are involved in accelerating the loss of RGC as a major endogenous source of reactive oxygen species (ROS). Ace Therapeutics is dedicated to developing drug targets and gene therapies to improve mitochondrial function in glaucoma, providing meaningful solutions in the quest for improved gene therapy for glaucoma.
Mitochondrial Dysfunction in Glaucoma
Glaucoma is a degenerative disease. Validated risk factors for glaucoma include high intraocular pressure, aging, etc. All of these factors can disrupt oxygen supply and alter retinal function. In addition, mitochondrial dysfunction within the RGC can also be a risk factor. In malfunctioning mitochondria, oxidative stress causing RGC damage is irreversible.
Mitochondrial dysfunction is strongly associated with RGC death during glaucoma. This makes it an attractive therapeutic target. Gene therapy targeting mitochondria can modulate mitochondrial function, including mitochondrial membrane potential and membrane stability, redox signaling and oxidative stress, and mitochondrial quality control.
Fig. 1 Mitochondrial genetics and genetic therapy. (Ji M H, et al., 2021)
Solutions of Mitochondrial-Based Gene Therapy Development for Glaucoma
The mitochondria are a viable therapeutic target for glaucoma. Based on gene therapy, mitochondria-related targets can be targeted to achieve improvement or repair of the optic nerve. Therefore, we offer development services for mitochondrial-based gene therapy for glaucoma treatment, including development design, trial validation, and preclinical studies.
Table 1. Our specific solutions for mitochondrial-based gene therapies for glaucoma.
| Development Strategies | Our Solutions |
|---|---|
| Mitochondrial calcium | In retinal neurons, intracellular calpain activation triggers an apoptotic cascade. Calpain activation is associated with the formation and opening of mitochondrial calcium channels. Drugs targeting calcium channel blockers can modulate ROS production and calcium signaling, which are significantly regulated by mitochondria, and are beneficial for RGC survival and treatment of optic nerve degeneration in glaucoma. |
| Dynamin-related protein 1 (DRP1) | By inhibiting DRP1 activity, mitochondrial fission and cell death can be blocked in glaucoma. It also promotes RGC survival in the presence of elevated IOP. DRP1 inhibition rescues RGCs from mitochondrial fission-mediated oxidative stress in glaucoma. This is a novel approach to mitochondria-based gene therapy for glaucoma. |
Our Advantages
The use of mitochondria-based gene therapy for glaucoma is under continuous development and validation. With our assistance, you can proceed smoothly with your project. We help you solve problems in future studies, including,
- We can help you to focus your research on in vitro, in vivo studies. In the area of animal models, create animal models that are more similar to the human eye.
- We provide you with additional preclinical studies, validate the role in the pathophysiology of glaucoma and ensure the development of a promising therapeutic approach.
- In addition to drug development, this also includes the development of gene carriers.
Our drug development services include basic research, drug development, and preclinical studies in a one-stop shop. And we are committed to providing you with satisfactory results in the shortest possible time.
Ace Therapeutics' mitochondrial-based gene therapy drug discovery and development efforts aim to discover and launch new therapies for glaucoma. If you have an innovative idea, please do not hesitate to contact us.
References
- Cheung L, et al. Targeted Delivery of Mitochondrial Calcium Channel Regulators: The Future of Glaucoma Treatment? Frontiers in Neuroscience, 2017, 11:648.
- Ji M H, et al. The Present and Future of Mitochondrial-Based Therapeutics for Eye Disease. Translational Vision Science & Technology, 2021, 10(8):4.
- Daliri K, et al. Glaucoma, Stem Cells, and Gene Therapy: Where Are We Now? IJSC, 2017, 10:119-128.