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The development of ophthalmic therapeutics includes drug discovery, formulation development, and preclinical testing to ensure safety and efficacy. As a research service provider, Protheragen is committed to providing global pharmaceutical companies with one-stop solutions for the development of therapies for ophthalmic diseases.
The field of ophthalmic disease therapeutics development encompasses the research, design, and clinical translation of innovative interventions aimed at addressing a wide range of vision-threatening conditions. From the debilitating impact of age-related macular degeneration (AMD) to the progressive vision loss associated with glaucoma, the landscape of ocular diseases presents a formidable challenge that requires a multifaceted approach to provide effective therapeutics and restore visual function.
At the core of ophthalmic therapeutics development lies a deep understanding of the intricate pathophysiology underlying these complex eye disorders. Researchers and drug developers must delve into the nuanced mechanisms driving the onset and progression of diseases like AMD, diabetic retinopathy, cataracts, dry eye, and others, in order to identify robust targets and design tailored therapeutic interventions.
The field of ophthalmic drug discovery has witnessed a remarkable surge of advancements in the past decade, as evidenced by the steady stream of FDA approvals for groundbreaking therapeutics. Notably, the emergence of innovative therapeutic strategies, including gene therapy, stem cell therapy, and targeted modulation of pathways like the complement system, visual cycle, and mTOR signaling, has opened up new avenues for tissue repair, regeneration, and achieving lasting therapeutic benefits.
Table 1. Summary of active ocular gene therapy programs. (Rodrigues G. A., et al., 2019)
Company | Program/ Product | Vector Technology | Disease/ Mechanism | Administration Route | Status |
Spark Therapeutics, Inc. | LUXTURNA™ (voretigene neparvovec-rzyl) | AAV2 | Confirmed biallelic RPE65 mutation–associated retinal dystrophy; RPE65 gene delivery | Subretinal injection | US approval (2017) |
Nightstar Therapeutics | NSR-REP1 | AAV2 | Choroideremia; REP1 gene delivery | Subretinal injection | Phase III |
RegenXBio Inc. | RGX-314 | NAV AAV8 | Wet AMD; anti-VEGF monoclonal antibody fragment | Subretinal injection | Phase I |
National Eye Institute, US National Institutes of Health | scAAV2-P1ND4v2 | AAV2 | LHON | Intravitreal injection | Phase I |
Applied Genetic Technologies Corporation | XLRS | rAAV2tYF | X-linked retinoschisis; hRS1 gene delivery | Intravitreal injection | Phase I/II |
GenSight Biologics | GS010 (rAAV2/2-ND4) | AAV2 | LHON | Intravitreal injection | Phase I/II |
Sanofi Genzyme | SAR422459 (with Oxford BioMedica) | Lentivirus (LentiVector) | Stargardt disease; ABCR gene delivery | Subretinal injection | Phase II |
Allergan plc | RST-001 (acquired RetroSense Therapeutics LLC) | Undisclosed | Retinitis pigmentosa; channelrhodopsin-2 optogenetic gene therapy | Intravitreal injection | Phase I/II |
Oxford BioMedica | OXB-201 | Lentivirus (LentiVector) | Wet AMD; endostatin and angiostatin gene delivery | Subretinal injection | Phase I |
Eyevensys | EYS606 | EyeCET (electrotrans-fection) | Noninfectious uveitis; anti–tumor necrosis factor-α plasmid delivery | Ciliary muscle transfection | Phase I/II |
National Eye Institute, US National Institutes of Health | RS1 AAV vector | AAV8 | XLRS; RS1 gene delivery | Intravitreal injection | Phase I/IIa |
Adverum Biotechnologies (formerly Avalanche Biotherapeutics) | ADVM-032 | AAV.7 m8 (4DMT) | Wet AMD; anti-VEGF (ranibizumab) | Intravitreal injection | Undisclosed |
Benitec Biopharma | BB-201 | Novel AAV | Wet AMD | Intravitreal injection | Preclinical |
4D Molecular Therapeutics | 4D-110 (with Roche) | Therapeutic vector evolution | Choroideremia; REP-1 | Intravitreal injection | Preclinical |
iVeena | IVMED-50 | AAV | Wet AMD; Flt23k gene delivery | Intraocular injection | Preclinical |
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 is at the forefront of ophthalmic disease therapeutics development, offering a comprehensive suite of services designed to accelerate the discovery and delivery of novel therapeutics for eye diseases.
The services we can provide include but are not limited to:
By Therapeutic Strategies
By Molecule Types
We utilize state-of-the-art equipment and techniques, including high-throughput screening platforms, advanced analytical instrumentation, and specialized in vitro and in vivo models, to drive the discovery and development of innovative ocular therapies. 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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