VGR-R01 injection, one of the proprietary core products of Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as “Vitalgen”), was granted implied CTA approval by the Center for Drug Evaluation of the NMPA for the treatment of Bietti crystalline dystrophy (BCD) on November 1, 2022, which is the world's first therapeutic product approved for clinical trial. On September 21, 2023, the surgical administration of all subjects was successfully completed in the Phase I/II clinical trial of VGR-R01, marking another critical step toward the marketing of this world-first therapeutic product.
Under the leadership of the principal investigators Professor Wenbin Wei from Beijing Tongren Hospital, Capital Medical University, and Professor Xiaodong Sun from the Shanghai General Hospital, the safety and efficacy clinical study of VGR-R01 was officially initiated in March 2023; and the enrollment and administration of VGR-R01 to all subjects were completed in half a year. So far, the Phase I/II clinical trial of VGR-R01 has yielded encouraging data: all subjects showed good safety and tolerability, and no adverse events (AE) possibly related to VGR-R01 was reported. The preliminary efficacy data showed that VGR-R01 could rapidly reverse or control disease progression in all dose groups in the dose escalation phase, enabling BCD patients with an average course of more than 10 years to improve the best corrected visual acuity (BCVA) or functional vision in dim conditions (Modified Rivermead Mobility Index, MRMI), thereby improving the quality of life (VFQ-25 score). The Vitalgen project team plans to discuss the further development and approval plan of the product with regulatory authority in the near future, and strive to promote the faster approval of the product to help BCD patients out of the dilemma of no cure for medical.
Figure 1 Investigators from Beijing Tongren Hospital, Capital Medical University
Figure 2 A BCD patient is receiving a surgery in Beijing Tongren Hospital, Capital Medical University
Dr. Xiaoping Zhao, CEO of Vitalgen, said: “I would like to thank all the BCD patients who participated in this study, the Suzhou Aitong Public Service Center (an organization of patients with retinal pigment degeneration), and all the investigators from Beijing and Shanghai study sites for your cooperation with Vitalgen to achieve another major milestone in BCD treatment.
So long as all God's children be
Relieved from hunger, free from cold
It cares not if, from wooded mountains
It comes to vast sufferings untold.
As a rare disease with a high incidence in East Asian populations, BCD is not concerned by European and American pharmaceutical companies, and our local enterprises need to shoulder the heavy responsibility. The original intention of the establishment of Vitalgen is to commit to making all diseases have the chance to cure, and develop a true global FIC gene therapy product. VGR-R01 best explains Vitalgen's positioning: starting from the clinical needs of Chinese patients, design the gene therapy product, establish the disease animal model, and finally return to the clinical treatment, reflecting the original intention of the Vitalgen team.
Vitalgen will continue to efficiently promote the follow-up clinical research of VGR-R01, looking forward to contributing important breakthroughs in China's independent innovation in the biotechnology industrial revolution, and bringing light to many BCD patients. “
