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  • AboutUs_Normal-24 The EyeCRO Approach
    • About Us
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  • MiDrops MiDROPS™
  • InVivo Models
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    • Laser-induced Choroidal Neovascularization
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Preclinical Ophthalmic Contract Research

gene therapy

Gene-based agents for the treatment of congenital eye diseases

July 7, 2016 //  by stanselb

Pharmacologists at LMU have developed gene-based agents for the treatment of congenital eye diseases. The first of these is now undergoing a phase-I clinical trial in color-blind patients at the University Medical Center in Tübingen. Is this approach translatable in principle to other visual disorders? Michalakis: About 200 genes have been identified which, when mutated, lead to monogenetic …

Category: NewsTag: blindness, eye health, gene therapy, ophthalmic research, retinitis pigmentosa, visual acuity

Avalanche Biotechnologies Closes Transaction With Annapurna Therapeutics

May 12, 2016 //  by stanselb

Avalanche Biotechnologies, Inc. (AAVL) today announced the closing of the previously announced transaction with Annapurna Therapeutics.  “The new management team is focused on applying our extensive clinical development expertise to advance the company’s gene therapy programs into the clinic.  We intend on dosing our first patients for A1AT deficiency by the end of this year and for HAE in 2017 …

Category: NewsTag: Age-related macular degeneration, Avalanche Biotechnologies, clinical development, eye disease, gene therapy, wet amd

Advancing Therapeutic Strategies for Inherited Retinal Degeneration: Recommendations From the Monaciano Symposium

March 5, 2015 //  by stanselb

Over the past 3 decades, global research efforts have unveiled the genetic complexity of the group of rare disorders collectively referred to as retinal dystrophies.  Causative mutations for dozens of retinal dystrophies have now been identified, resulting in the discovery of multiple new genes, biological pathways necessary for photoreceptor cell function, and pathogenetic mechanisms involved in …

Category: NewsTag: Electroretinography, gene therapy, inherited retinal degeneration, intravitreal injection, photoreceptor cell, retinal dystrophies, retinal pigment epithelium

Spark Surges in IPO as Prices Fuel Gene Therapy

February 4, 2015 //  by stanselb

Spark Therapeutics Inc. hit a $1.2 billion valuation on the biotechnology firm’s first day on the market, reflecting growing investor enthusiasm for the once-beleaguered field of gene therapy, a field that has produced the first $1 million drug.  The treatments focus on diseases where a single mutation in the genetic code is known to be the cause. Fix or replace the genetic code, and, …

Category: NewsTag: Age-related macular degeneration, Avalanche, Biogen Idec Inc., biotechnology, blind, eye disorders, gene therapy, Pfizer Inc., Spark Therapeutics Inc.

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