VGR-R01, a gene therapy developed by Shanghai Vitalgen BioPharma Co., Ltd (hereinafter referred to as “Vitalgen”) for the treatment of Bietti crystalline dystrophy (BCD), was granted the Orphan Drug Designation (ODD) by the United States Food and Drug Administration (hereinafter referred to as “FDA”) on October 29, 2024 (EST). VGR-R01 was previously granted the Breakthrough Therapy Designation by the Chinese Center for Drug Evaluation of the National Medical Products Administration (NMPA) based on its promising Phase 1/2 data obtained. The granting of ODD by FDA represents a significant milestone in the international rollout of VGR-R01 and is expected to expedite the progresses of clinical trials and drug registration. In addition, VGR-R01 would be qualified to receive a variety of ODD incentives, including tax credits for qualified clinical trials, reduced or waived fees for regulatory activities and potential seven years of market exclusivity after approval.