Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders. Now it’s on the cusp of its first approval.

A promising study out of the United Kingdom has partially restored the vision of multiple small children born legally blind. The experimental treatment uses gene therapy to resolve a retinal disorder called LCA4,

A gene therapy from Regeneron Pharmaceuticals Inc. improved hearing in nearly all deaf children in a clinical trial, results that could allow the company to seek regulatory approval for the one-time treatment.

A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic deficiency. Scientists at UCL and Moorfields Eye Hospital successfully injected healthy copies of the defective gene into the retina,

Before the therapy, the affected children were legally blind, only able to distinguish between light and dark. However, after receiving the treatment, all four toddlers demonstrated remarkable progress—some even beginning to draw and write.