Research

Latest Research

  • Nightstar Therapeutics raises $75 Million in IPO to fund Pivotal Phase 3 Gene Therapy Study Nightstar Therapeutics held it’s Initial Public Offering on Thursday September 28th, raising $75 Million which will be used to advance the company’s gene therapy treatment for Choroideremia along with other Retinal Degenerative Diseases the company is working on.  The company, which was spun out of Oxford University has completed Phase 1/2 Clinical Trials and is ...
  • 4DMT Now Recruiting for Natural History Studies! 4D Molecular Therapeutics Enrolling Patients in Natural History Study in Lead Clinical Program to Develop Gene Therapy Treatment for Choroideremia  Intravitreal delivery to the retina is critical to Choroideremia treatment 4D Molecular Therapeutics (4DMT), a leader in gene therapy product discovery and development, is accepting patients for enrollment in its Choroideremia Natural History Study (NHS). This study ...
  • 4DMT Announces Plans for CHM Natural History Study 4DMT is accepting inquires from interested choroideremia (CHM) patients for their upcoming Natural History Study.  In partnership with the Choroideremia Research Foundation, 4DMT seeks to develop a gene therapy product optimized for intravitreal administration to treat Choroideremia. Natural History Studies track disease progression and are an essential first step in developing efficient clinical stages of testing ...
  • Gene Therapy Trial Results Released! In a letter published by the New England Journal of Medicine today, Dr. Robert Maclaren released positive long-term results from the initial 6 Choroideremia patients treated in the clinical trial at the University of Oxford. These results, which show sustained effects of gene therapy, represent another critical step toward the approval of gene ...

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Research Types

Gene Therapy

Genetic diseases like Choroideremia are caused by a mutation, or defect, in the body’s DNA.  These genetic mutations prevent the body from producing a beneficial protein necessary for certain cells to survive.  Gene therapy is a type of treatment for genetic diseases in which the normal gene is delivered into the affected cells, enabling this protein to be produced and restore normal cellular function.

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 Stem Cell Therapy

The vision loss in Choroideremia is caused by the progressive death of specific cells in the retina which are responsible for creating vision.  Restoring lost vision requires replacing those cells, and stem cells are a potential candidate for creating transplants to place into these affected areas.

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Pharmacological Therapy

Doctors commonly prescribe medications to treat a wide variety of diseases affecting the human body.  For Choroideremia and other retinal diseases, scientists are working to develop and test medications which can slow down or stop the progression of vision loss.

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Retinal Prosthetics

Vision loss in Choroideremia progressively continues until people lose all their sight.  For these individuals, retinal prosthetics can be surgically implanted to restore a small amount of vision.

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