A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Treated Previously with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) in an Antecedent Study (SOLSTICE)

  • Sponsor: NightstaRx, Ltd, a Biogen Company
  • Principal Investigator: Kimberly Stepien, MD
  • Study Coordinator: Nickie Stangel

Study objective:

Long-term follow up evaluating the safety and efficacy of AAV2-REP1 used in antecedent choroideremia treatment studies.

Study design:

The study will consist of up to 9 visits over a maximum 48-month study period. Subjects will rollover into the current study when they complete the antecedent study. No further gene therapy will be given in this protocol.

Inclusion criteria:

  • Have received a sub-retinal injection of AAV2-REP1 for CHM in an antecedent study

Exclusion criteria:

  • There are no exclusion criteria for this study

Contact Nickie Stangel with questions: 608-263-8783. For more information about this study: visit