Fit for the Eye: Aptamers in Ocular Disorders

Area of Science:

• Ophthalmology
• Biotechnology
• Nucleic Acid Therapeutics

Background:

• The eye is a historically attractive site for therapeutic intervention.
• Aptamers, a class of nucleic acid ligands, have emerged as promising therapeutic agents.
• Previous therapeutic applications for aptamers were not initially focused on ophthalmology.

Purpose of the Study:

• To review the discovery and development of aptamers for ophthalmic indications.
• To highlight key technological advancements enabling aptamer development.
• To discuss the potential of aptamers for future ophthalmic therapies.

Main Methods:

• Review of the literature on aptamer discovery and development in ophthalmology.
• Focus on three specific aptamers: Macugen (approved), Fovista, and Zimura (late-stage development).
• Analysis of technological improvements in aptamer selection and chemical optimization.

Main Results:

• One aptamer, Macugen, has been approved for ophthalmic use.
• Two other aptamers, Fovista and Zimura, are in late-stage clinical development for eye conditions.
• Technological advancements in aptamer selection and optimization were crucial for identifying suitable candidates.

Conclusions:

• Aptamers demonstrate significant potential as therapeutics in ophthalmology.
• Lessons learned from these aptamers can guide future development and identify new indications.
• Aptamers offer distinct advantages for various therapeutic applications beyond ophthalmology.