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Ranging from rare retinal dystrophies to common refractive errors, congenital ophthalmic diseases pose a significant challenge but also an opportunity for innovative therapeutic interventions. At Protheragen, our team of experienced biologists is dedicated to unraveling the complex molecular mechanisms of these debilitating diseases and providing expert therapy development services.
Congenital ophthalmic diseases, or congenital eye diseases, are a group of vision impairments or blindness that manifest at birth or during early infancy, often resulting from genetic mutations or developmental anomalies in the eye. These conditions can affect various structures of the eye, including the cornea, retina, and optic nerve, and can lead to severe visual impairment or complete blindness. The impact of these diseases on individuals and their families is profound, emphasizing the urgent need for effective therapeutics and therapies.
The landscape of drug and therapy development for congenital ophthalmic diseases has seen significant advancements in recent years. The integration of genetic research, molecular biology, and novel gene-editing technologies has paved the way for targeted and personalized therapies. Notably, gene therapy has emerged as a promising approach, with several clinical trials demonstrating safety and efficacy in treating specific genetic conditions. For instance, the success of RPE65 gene replacement therapy has set a precedent for the potential of gene therapy in congenital ophthalmic diseases.
Table 1. Clinical trials registered on clinicaltrials.gov for different congenital ophthalmic diseases. (Niu Y., et al., 2024)
NCT Number | Conditions | Study Title | Study Status | Phases |
---|---|---|---|---|
NCT03957954 | Limbal Stem-cell Deficiency | Stem Cell Therapy for Limbal Stem Cell Deficiency | Recruiting | Phase1 |
NCT04995926 | Limbal Stem-cell Deficiency | Labial Mucosal Epithelium Grafting for Corneal Limbus Substitution | Recruiting | Unknown |
NCT05909735 | Limbal Stem-cell Deficiency|Congenital Aniridia | Treatment of LSCD With DM | Recruiting | Phase1 |
NCT04773431 | Limbus Corneae|Limbus Corneae Insufficiency Syndrome | Safety Evaluation of LSCD101 Transplantation for Limbal Stem Cell Deficiency | Completed | Phase1 |
NCT04642729 | Macular Corneal Dystrophy | Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery | Enrolling_by_invitation | Unknown |
Disclaimer: Protheragen focuses on providing preclinical research service. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Protheragen leads the way in pioneering groundbreaking drug and therapy solutions for congenital ophthalmic disorders. Our extensive range of services includes gene therapy, gene editing, and the creation of innovative pharmaceutical compounds. By harnessing state-of-the-art technologies, we target and rectify genetic mutations at their core, providing a beacon of hope for conditions that were once deemed untreatable.
A-B | ||
Acorea, Microphthalmia and Cataract Syndrome | Aniridia | Ankyloblepharon |
Anophthalmia | Aphakia | Axenfeld-Rieger Syndrome |
Blepharophimosis | Buphthalmos | |
C-E | ||
Cochleosaccular Degeneration with Progressive Cataracts | Cystic Eyeball | Cornea Plana 1 |
Coloboma | Congenital Fibrosis of the Extraocular Muscles | Cornea Plana 2 |
Cryptophthalmos | Distichia | Ectropion |
Cyclopia | Ectopia Lentis | Entropion |
I-N | ||
Iridogoniodysgenesis, Dominant Type | Keratoglobus | Megalocornea |
Iris Hypoplasia with Glaucoma | Marcus Gunn Phenomenon | Megalopapilla |
Microphthalmia | Nance-Horan Syndrome | Norrie Disease |
Microspherophakia | Nasolacrimal Duct Obstruction | |
P-Z | ||
Polycoria | Primary Juvenile Glaucoma | Ptosis (Eyelid) |
Persistent Fetal Vasculature | Syndromic Microphthalmia | Zonular Cataract and Nystagmus |
Sensory Ataxic Neuropathy, Dysarthria, and Ophthalmoparesis |
As we continue to advance our research and development, our commitment to excellence and innovation remains unwavering, ensuring that Protheragen remains at the forefront of congenital ophthalmic disease therapeutics. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
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