Therapeutics Development Services
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Therapeutics Development Services

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.

Therapeutics Development in 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.

Nanotechnology-based strategies for therapeutics of eye disease. Fig.1 Nanotechnology-based strategies for ophthalmic disease therapeutics development. (Weng Y., et al., 2017)

Ophthalmic Diseases Therapeutics Development

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.

Our Services

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:

  • Target Identification and Validation
    Our in-depth expertise in ocular disease pathophysiology allows us to expertly identify and validate promising molecular targets that hold the potential to address the underlying mechanisms driving various ophthalmic conditions. By leveraging the latest advancements in genomics, proteomics, and advanced screening technologies, we can uncover novel therapeutic targets with high specificity and translational relevance.
  • Preclinical Drug Discovery and Development
    Protheragen's seasoned team of medicinal chemists, pharmacologists, and biologists work in tandem to design, synthesize, and thoroughly evaluate novel chemical entities and biologics for their ocular therapeutic potential. Our robust drug discovery platform, encompassing structure-activity relationship studies, in vitro and in vivo pharmacology assessments, and comprehensive safety evaluations, enables the efficient generation of lead candidates poised for clinical advancement.
  • Innovative Ocular Drug Delivery Solutions
    Recognizing the unique challenges posed by the eye's intricate anatomy and physiological barriers, Protheragen has established specialized capabilities in the development of innovative ocular drug delivery systems. Our expertise spans the design and optimization of advanced formulations, including ocular inserts, punctum plugs, micro-implants, hydrogels, contact lenses, liposomes, and nanoparticles, to enhance the targeted delivery and sustained release of ophthalmic therapeutics.

Types of Ophthalmic Diseases Therapeutics

By Therapeutic Strategies

  • Antibiotic Development
  • Antifungal Drug Development
  • Antivatival Drug Development
  • Anti-inflammatory Drug Development
  • Targeted Therapy Development

By Molecule Types

  • Small Molecule Drug Development
  • Cell Therapy Development
  • Gene Therapy Development
  • Therapeutic Antibody Development
  • Therapeutic Peptide Development
  • Therapeutic Protein Development

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.

References

  • Weng Yuhua, et al. "Nanotechnology-based strategies for treatment of ocular disease." Acta pharmaceutica sinica B 7.3 (2017): 281-291.
  • Rodrigues, Gerard A., et al. "Pharmaceutical development of AAV-based gene therapy products for the eye." Pharmaceutical research 36.2 (2019): 29.