▎Edited by the WuXi AppTec content teamOn March 1, StarryGene announced that the IND application for its self-developed gene therapy XMVA09 injection, a Class 1 innovative drug, has been approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, and is proposed for development toWet Age-Related Macular Degeneration（wAMD). According toStarryGene Press Release,XMVA09 is a product that combinesAchieving Bispecific TargetingAndIntravitreal Injection of CapsidGene therapy drugs.Screenshot source: CDE official websiteStarryGene was founded in 2019 as a high-tech company focused on developing cutting-edge gene editing technologies and gene therapy products for ophthalmic diseases.Ophthalmic Gene Therapy Drug Development，with age-related macular degeneration and other common ophthalmic diseases as the main entry point.wAMD is a common fundus disease, characterized by abnormal proliferation and leakage of blood vessels in the macular area of the retina, leading to a sharp decline in vision.This disease is one of the leading causes of blindness in the elderly, significantly impacting the quality of life for patients.According toStarryGene Press Release Introduction,Compared to the traditional antibody therapeutic drugs with an injection cycle of 1 to 3 months,XMVA09 asGene therapy drugs can produce sustained therapeutic effects, with the hope of achievingOne injection for life-long effectiveness。XMVA09Using a completely new AAV capsid, which can infect the retinal pigment epithelium (RPE) cells closely attached to the choroidal lesion through intravitreal injection, providing a more convenient approach for subsequent clinical applications. In addition, the drugAdopting a bispecific antibody design, targeting both vascular endothelial growth factor (VEGF) and angiopoietin-2 (ANG-2) dual targets simultaneously., improved the therapeutic effect of the drug, and expanded the coverage for patients who are insensitive to VEGF.
XMVA09 previously demonstrated promising clinical outcomes in an investigator-initiated trial (IIT). Among the six enrolled wAMD subjects, all of whom had not received anti-VEGF prospective loading treatment prior to drug administration, favorable therapeutic effects were still observed. The first patient, who had the longest observation period, showed a significant improvement in best-corrected visual acuity (BCVA) after nearly a year of clinical follow-up. The subject with the most optimal clinical benefit (from the high-dose group) exhibited a BCVA increase of 17 letters from baseline at the most recent follow-up. The interim results of the IIT indicate,XMVA09 Injection Demonstrates Reliable Safety and Good Tolerability, and has preliminarily achieved the expected research outcomes in evaluating efficacy.Founder and Chief Executive Officer (CEO) of StarryGeneDr. Cai YuanStarryGene expressed great delight at the approval of XMVA09 Injection's IND. This is the result of years of relentless efforts by the StarryGene team and also a manifestation of StarryGene's commitment to treating ophthalmic diseases.
Chief Medical Officer of StarryGeneDr. Miaobo LongStarryGene stated that the IIT research on XMVA09 injection conducted at the First Affiliated Hospital of the University of Science and Technology of China has achieved encouraging interim results. Regarding the approval of this IND, the preparatory work for the Phase 1 clinical study is also basically ready, and it is believed that it will be fully launched soon.
[1] The First Gene Therapy Drug for wAMD with Both Bispecific Targets and Intravitreal Injection Capsid, "XMVA09 Injection," Successfully Approved for IND!. Retrieved Mar 1, 2024, from https://mp.weixin.qq.com/s/FY6M5M06rOLLxTs73EbuzgThis article was compiled and edited by the WuXi AppTec content team based on publicly available information. Individuals are welcome to share it on their social media circles. For authorization to repost, please leave a message on the "MedView" WeChat Official Account to contact us. For other collaboration inquiries, please contact wuxi_media@wuxiapptec.com.
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