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Sustained Therapeutic delivery for the eye

How it Works

Avalanche delivers its therapeutic treatments using an adeno-associated virus (AAV) as a vector to deliver and express, or transduce, a functional gene to the cells of the eye to promote continuous protein production.

To create a safe therapeutic vector, viral genes are removed from AAV and replaced with specific genes encoding a therapeutic protein that potentially treats a variety of ophthalmic diseases. AAV can infect a variety of retinal cell types and remain stable, resulting in long-term therapeutic protein expression. In clinical studies, AAV appears to be safe and well-tolerated when injected into the retina.

Additional Information on How it Works:

AVA-101 is injected into the eye in a short surgical procedure. Photo 1 AVA-101 is injected into the eye in a short surgical procedure. Photo 2
AVA-101 is injected into the eye in a short surgical procedure.
AVA-101 creates an Ocular BioFactory TM that secretes therapeutic protein. This in turn blocks VEGF signaling, treating the disease.
AVA-101 creates an Ocular BioFactoryTM that is designed to secrete therapeutic protein. The protein in turn blocks VEGF signaling, treating the disease.

More great reads

The results of a Phase 1, 52-week trial of Avalanche Biotechnologies’ rAAV.sFLT-1 (AVA-101) gene therapy for the treatment of wet age-related macular degeneration (wAMD) have been published online by The Lancet. The study sought to evaluate the safety of rAAV.sFLT-1 via a single subretinal injection. Results of the study demonstrate that rAAV.sFLT-1 was safe and well tolerated, and support ocular gene therapy as a potential long-term treatment option for wAMD.

“The use of gene therapy to reprogramme genetically normal cells of the retina to take on additional functions opens a new chapter in potential AAV applications.”
– Dr. Robert MacLaren, Moorsfield Eye Hospital

The complete article and editorial commentary by Dr. MacLaren, are available from The Lancet.

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