Ocular Efficacy, PK and Toxicology

The Pharmaron team in the US has over a decade of ophthalmic cell and gene therapy experience, which enables the team to support the advancement and IND approval of your ocular product through preclinical efficacy, PK and biodistribution, safety and GLP-toxicology studies. For anterior or posterior disease indications that cause functional impairment, our team of ocular scientists and board-certified veterinary ophthalmologists help develop your study design, data acquisition/interpretation and clinical translation using state-of-the-art instrumentation for comprehensive, descriptive and functional evaluation of critical endpoints.

View as PDF: Ocular Efficiency, PK and Toxicology

Download E-book: Ocular Cell and Gene Therapy Preclinical Reference Guide

Download E-book: EYEdentify Ocular Consideration


Disease Models

  • Inherited retinal disorder models
  • Royal College of Surgeons (RCS–/–) rats; Rhodopsin (RHO) P23H transgenic rats; Pde6β–/– mice (rd1, rd10); Rd12 mice
  • Wet age-related macular degeneration (AMD)
  • Glaucoma
  • Diabetic retinopathy

Ocular Biodistribution

  • Aqueuos humor (taps) and vitreous humor (vitrectomy)
  • Tissues (cornea, conjunctiva, sclera, choroid, ICB, TM, lens, retina, RPE, optic nerve)

Routes of Delivery

  • Specialized ocular routes of administration:(intravitreal, sub-retinal, suprachoroidal, intracameral, subconjunctival)


  • rodent, non-rodent

Facility Accreditations

  • AAALAC, USDA, OLAW; ISO(10933) certification; successful FDA inspection in 2021 (NAI)

Learn more about Pharmaron’s expertise by registering for our monthly CGT webinar series, which is focused on identifying key cell and gene therapy questions that arise during drug discovery and development. Register here.

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