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Sustained Therapeutic delivery for the eye

AVA-311

AVA-311 is being developed for the treatment of Juvenile X-linked Retinoschisis (XLRS), an inherited retinal disease that occurs almost exclusively in males caused by mutations in the RS1gene located on the X chromosome. AVA-311 is comprised of an optimized AAV vector using Avalanche’s directed evolution technology that intravitreally delivers the RS1 gene in the 83 eye to potentially achieve a functional cure for patients.

In May 2014, Avalanche signed a collaboration agreement with Regeneron for the development of AVA-311.

 

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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