StarryGene’ XMVA09, the World’s First AAV-Delivered Bispecific Antibody Gene Therapy Drug, Enters Phase II Clinical Trial

Recently, StarryGene successfully convened the Phase II clinical trial kick-off meeting for its independently developed XMVA09 Injection for the treatment of wet age-related macular degeneration (wAMD). This milestone marks XMVA09 Injection as the world’s first AAV-delivered bispecific antibody to enter Phase II clinical development, as well as the first ophthalmic AAV drug based on a non-natural capsid in China to enter Phase II clinical development, opening a new chapter in ophthalmic gene therapy both in China and globally. As a strategic partner of StarryGene, PackGene Biotech extends its warm congratulations on this significant progress. We look forward to continuing to work closely with StarryGene to accelerate the commercialization of XMVA09 Injection and benefit more patients.

wAMD is a leading cause of irreversible vision impairment among people over the age of 50, affecting the quality of life of approximately 200 million people worldwide. Under the current standard of care, patients need to receive intraocular injections of anti-VEGF drugs every one to two months. Treatment interruption or insufficient treatment may lead to reduced long-term efficacy and irreversible vision loss, while gene therapy holds the potential to achieve “one administration for lifelong efficacy.” As the world’s only gene therapy drug that combines dual antibody targets with intravitreal injection, XMVA09 Injection uses an AAV gene vector carrying a bispecific antibody gene targeting VEGF-A and Ang-2. Through intravitreal injection, it directly acts on RPE cells to express therapeutic proteins, with the goal of achieving long-term treatment for wAMD.

XMVA09 Injection was approved by the NMPA to enter clinical development in March 2024. In early April this year, the Phase I clinical trial successfully completed enrollment and treatment of all subjects. Preliminary results demonstrated favorable safety and tolerability, while efficacy data showed significant market advantages. The Phase II kick-off meeting for XMVA09 Injection in the treatment of wAMD, held in Tianjin, brought together leading experts in the field of wAMD treatment in China, StarryGene’ core management team, and representatives of clinical research partners to discuss the Phase II clinical study protocol and execution plan for XMVA09 Injection.

Dr. Miao Bolong, CMO of StarryGene, stated: “As the first innovative bispecific antibody therapy administered via intravitreal injection, XMVA09 Injection is expected to significantly reduce the injection frequency for patients with wAMD and become a first-line maintenance therapy for wAMD in the future. We firmly believe that, driven by the rich clinical experience of the expert team and close collaboration among all parties, the clinical trial of XMVA09 Injection will progress steadily and bring benefits to many patients as soon as possible.”

Dr. Cai Yuan, CEO of StarryGene, stated: “The initiation of this pivotal Phase II clinical trial represents a recent key milestone for StarryGene and a crucial step in validating our technological advantages. XMVA09 Injection has advanced smoothly to the next stage with excellent clinical benefits, thanks to the support of all our partners, the trust of all shareholders, and the dedication and close collaboration of our team. StarryGene will continue to focus on R&D, leverage cutting-edge gene therapy technologies to develop leading innovative products, and benefit the broadest possible patient population.”

About StarryGene

StarryGene is a high-tech enterprise focused on the development of ophthalmic gene therapy drugs, based on cutting-edge gene editing technologies and gene therapy product development for ophthalmic diseases. Its core team is a PhD entrepreneurial team led by a National Science Fund for Distinguished Young Scholars recipient. On the R&D side, the company has established two core technology platforms: an AAV-antibody gene drug platform comprising a bispecific antibody optimization platform and a capsid screening platform, and a gene editing platform.

The company is committed to developing ophthalmic gene therapy drugs that are independently accessible to Chinese patients, actively advancing gene therapy into major ophthalmic indications. Taking ophthalmology as its breakthrough field, and common ophthalmic diseases represented by age-related macular degeneration as its key entry point, StarryGene aims to help Chinese patients see longer and see better.

PackGene Biotech is honored to participate in StarryGene’ XMVA09 Injection project by providing a full suite of CMC services from plasmid to AAV, supporting the advancement of XMVA09 Injection into Phase II clinical development for wet age-related macular degeneration (wAMD). We pay tribute to and congratulate StarryGene on its innovation and achievements in the gene therapy field, and look forward to continued in-depth collaboration between both parties to jointly promote innovation and development in the biopharmaceutical industry and contribute to human health.