Gene Therapy Milestones

2006 - Researchers use gene therapy to successfully treat Usher 1B (animal model). Researchers use gene therapy to successfully treat Stargardt disease (animal model). Foundation partners with Oxford Biomedica to develop gene therapy for Stargardt disease. 2007 - Phase I clinical trials of gene therapy for LCA begin. Nanoparticles show promise for delivering healthy genes to the retina. 2008 - More than 140 genes linked to retinal degenerative diseases. Young adults who were nearly blind from LCA gain some vision in Phase I gene therapy clinical trials. Gene therapy successful in treating achromatopsia (animal model). Foundation establishes partnership with Genable to develop gene therapy for dominant retinitis pigmentosa. 2009 - Foundation partners with AGTC to develop gene therapies for retinoschisis and achromatopsia. More than 20 people who were nearly blind experience some vision restoration in three Phase I clinical trials of gene therapy for LCA.Researchers use gene therapy to successfully treat Stargardt disease (animal model). Foundation partners with Oxford Biomedica to develop gene therapy for Stargardt disease. 2007 Phase I clinical trials of gene therapy for LCA begin. Nanoparticles show promise for delivering healthy genes to the retina. 2008 More than 140 genes linked to retinal degenerative diseases. Young adults who were nearly blind from LCA gain some vision in Phase I gene therapy clinical trials. Gene therapy successful in treating achromatopsia (animal model). Foundation establishes partnership with Genable to develop gene therapy for dominant retinitis pigmentosa. 2009 Foundation partners with AGTC to develop gene therapies for retinoschisis and achromatopsia. More than 20 people who were nearly blind experience some vision restoration in three Phase I clinical trials of gene therapy for LCA.



1995

  • First successful gene therapy (animal model).

1997

  • Discovery of Stargardt disease gene (ABCR).

1998

  • Discovery of Usher 2A gene.

2000

  • Gene therapy restores vision in dogs with Leber congenital amaurosis.

2001

  • More than 100 genes known to cause retinal degenerative diseases.

2005

  • Discovery of CFH gene — implicated in 50 percent of age-related macular degeneration cases.

2006

  • Researchers use gene therapy to successfully treat Usher 1B (animal model).
  • Researchers use gene therapy to successfully treat Stargardt disease (animal model).
  • Foundation partners with Oxford Biomedica to develop gene therapy for Stargardt disease.

2007

  • Phase I clinical trials of gene therapy for LCA begin.
  • Nanoparticles show promise for delivering healthy genes to the retina.

2008

  • More than 140 genes linked to retinal degenerative diseases.
  • Young adults who were nearly blind from LCA gain some vision in Phase I gene therapy clinical trials.
  • Gene therapy successful in treating achromatopsia (animal model).
  • Foundation establishes partnership with Genable to develop gene therapy for dominant retinitis pigmentosa.

2009

  • Foundation partners with AGTC to develop gene therapies for retinoschisis and achromatopsia.
  • More than 20 people who were nearly blind experience some vision restoration in three Phase I clinical trials of gene therapy for LCA.