Foundations and Advocacy Groups

Choroideremia Research Foundation (CRF)

In partnership with the Choroideremia Research Foundation, 4DMT seeks to develop a gene therapy product optimized for intravitreal administration to treat Choroideremia.

4DMT is conducting a Natural History Study for Choroideremia (CHM).

Natural History Studies track disease progression and are an essential first step in developing efficient clinical stages of testing in rare diseases. The purpose of this study is to evaluate disease progression as well as determine the feasibility of measuring endpoints for subsequent clinical trials in the hopes of providing medical benefit through gene therapy to all choroideremia patients and their families.

Two clinical sites are actively recruiting for the study:

  • Retina Foundation of the Southwest, Dallas, Texas (Principal Investigator Dr. David Birch). For eligibility information, contact  (214) 363-3911 ext. 114 or 116.
  • Retina Vitreous Associates of Los Angeles (“LA Retina” – Principal Investigator Dr. David Liao). For eligibility information, contact the Clinical Trial Department at (310) 289-2478.

This website will be updated as additional study sites open throughout the United States.

Eligibility requirements include but are not limited to: participants must be male, age 14 years or older, and have a CLIA Certified Genetic Diagnosis with visual acuity ≥ 20/200 or better.

Study participation requirements:

Attend Study Site Visits

  • An initial screening visit followed by a visit every 6 months, for 24 months for a total of 5 visits

Assessments Include

  • Medical history review
  • Visual and eye exam
  • Photography and imaging

Email Questions to CHM@4Dmoleculartherapeutics.com

For more information about Choroideremia, visit the Choroideremia Research Foundation (CRF) online. Additional clinical trial information including contact numbers may be found at clinicaltrials.gov, NCT 02994368.