MENLO PARK, Calif. (TheStreet) -- An important clinical trial catalyst is approaching for Avalanche Biotechnologies (AAVL) and its gene therapy platform. Results from a phase IIa study of Avalanche's AVA-101 in wet age-related macular degeneration are expected later this month or in July. The looming Avalanche data will also be another opportunity to test investors' love affair with gene therapy.
Wet age-related macular degeneration (wet AMD) is a chronic disease of the eye that is associated with the proliferation of blood vessels in the retina. Blood and fluids can leak from these new vessels, leading to thickening and bulging of the macular portion of the retina. Wet AMD causes a progressive loss of vision.
The current standard of care for wet AMD involves drugs which block VEGF, a protein that promotes the growth of blood vessels and is expressed at abnormally high levels in patients with the disease. These anti-VEGF compounds reduce retinal thickness, significantly improve vision and have a good safety profile. The two leading anti-VEGF drugs for wet AMD are Regeneron Pharmaceuticals' (REGN) Eylea and Roche's (RHHBY) Lucentis.
Eylea and Lucentis are major medical breakthroughs for wet AMD but the drugs are not without their downside, which is the opportunity Avalanche is seeking to exploit.
The drugs need to be injected directly into the eye and must be administered chronically in order for patients to maintain any benefit. Lucentis, for example, provides best results when injected once per month.