The most promising RP treatments in next 10 years

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  • čas přidán 12. 09. 2024
  • In the coming 8 to 10 years, we can expect more and more RP treatments to enter the market and provide curative solutions. Some of them are promising and closed to get FDA approval.
    1. One of the most anticipated technologies is CRISPR-Cas9, which has revolutionized the world by making gene editing simpler than ever. However, the use of gene therapy will likely be limited to a small number of patients with specific mutations.
    2. Developing a gene therapy is expensive, making it unfeasible to develop one for each mutation, especially for extremely rare mutations. To overcome this challenge, some companies are designing new approaches to make a single therapy suitable for everyone.
    In the case of GenSight, the company is developing a treatment that combines gene therapy with a wearable device to potentially treat all types of RP. The gene therapy is used to introduce a light-sensitive protein within neurons in the optic nerve. A pair of goggles helps redirect and concentrate the light toward the modified cells, which will then convert it into a signal for the brain.A phase one and two trial is currently testing this technology.
    3. Nanoscope is focused on proprietary ambient light-activated optogenetic therapy to restore vision in people suffering from RP, Stargardt,and dry AMD.
    Genetic retinal degenerative diseases cause vision loss as cells of the retina gradually become dysfunctional. Nanoscope’s proprietary gene therapies correct the condition by delivering light-sensitive molecules, called Multi-Characteristic Opsins (MCO), into retinal cells.
    4. ProQR Therapeutics has developed several treatments for gene mutation of CEP290, USH2A and the exon 13.
    5. MEIRAGTx sponsors AAV-RPGR gene therapy for RPGR-associated x-linked retinitis pigmentosa (XLRP), and has obtained positive results in the previous phase I and II clinical trials. This therapy lies in the intravitreal administration of vectors carrying thenormalcopy of the gen RPGR.
    How to make the best preparation of the future treatments and increase the success rate of the future treatments? To maintain a healthy retinal environment and protect the optic nerve function are two traits towards the path to success. Currently, there are two ways available of neuroprotection and keeping a balanced retinal environment.
    1. StemActiv - Pretreatment for gene therapy and stem cell treatment. StemActiv not only increases blood flow to the retina, improves oxygen and nutrients supply to activate dormant rods and cones, but also contains anti-inflammatory, anti-oxidative and anti-apoptosis components. It restores the dormant optic cells function and optimizes the microenvironment of the retina. Genetic material and stem cell can be injected into a certain zone in the eyes and eventually cure RP, but without a healthy retinal environment, an inflamed surrounding could end in a complete failure of any therapy.
    2. AC STIM enhances the activity and function of the retinal cells, thereby reinforcing visual signaling along the optic nerve and restoring impaired vision. The lower frequency increases energy production ATP in the retina, reverts the damaging retina, preserves visual acuity and the visual field. The higher frequency helps relax the retinal tissue, increases blood supply to the retina, stimulates and energizes dormant rods and cones. It also attracts adult stem cells to help activate possible cell regeneration.
    For more details of StemActiv and AC STIM pretreatment for gene and stem cell therapy, please review: www.restorevis...

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