Companies Dominating the Choroideremia Treatment Landscape
- Novartis AG
- Spark Therapeutics
- Bluebird Bio Inc.
- Biogen Inc.
- Copernicus Therapeutics, Inc.
- 4D Molecular Therapeutics
- Wize Pharma Inc.
- Retina Implant AG
- Hoffmann-La Roche Ltd
- Kiora Pharmaceuticals
- Astellas Pharma Inc.
- Pixium Vision
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In the News
- Biogen Inc., announced that the final patient has been enrolled in the international Phase 3 STAR clinical trial evaluating timrepigen-emparvovec, an experimental gene therapy for the treatment of choroideremia.
- Astellas Pharma and 4D Molecular Therapeutics, Inc. acquire rights for Astellas to use the R100* intravitreal retinotropic vector invented by 4DMT for gene targets involved in rare monogenic eye diseases Announced license agreement (with option to add up to two additional targets) for an additional option exercise fee, potentially implicating rare monogenic eye diseases. R100 is a vector invented by 4DMT for intravitreally delivery of the adeno-associated virus, AAV. It's capable of breaking into the internal membrane wall and effectively transducing the whole retina, resulting in a strong gene expression for retinal cells. The R100 vector is used, including 4D-150 in wet agerelated macular degeneration and diabetic macular edema, by all the three clinical stage4DMT ophthalmic product candidates.
