ViGeneron Announces EMA Approval of Clinical Trial Application for VG901, a Novel Gene Therapy for CNGA1-Associated Retinitis Pigmentosa

/Part of the content is from the ViGeneron official website

August 22, 2023, Munich, Germany

WuXi ATU Strategic Partner ViGeneron GmbH (ViGeneron) recently announced that its gene therapy product VG901 has passed the Clinical Trial Application (CTA) by the European Medicines Agency (EMA). WuXi ATU extends its warm congratulations to ViGeneron for this significant milestone!

VG901 is a potentially revolutionary gene therapy for CNGA1-related Retinitis Pigmentosa (RP), a condition for which there are currently no approved treatments available in the industry. In 2020, ViGeneron and WuXi Advanced Therapies (WuXi ATU) entered into a strategic collaboration, with WuXi ATU’s CTDMO platform enabling the development of VG901 from its initial stages through to clinical approval.

As a global CTDMO in cell and gene therapies, WuXi Advanced Therapies has supported multiple clients in obtaining clinical trial approvals in the United States, Europe, South Korea, China, and Singapore. WuXi Advanced Therapies integrates robust testing capabilities with process development and manufacturing capacities, committed to accelerating and transforming the development, testing, production, and commercialization of cell therapies, gene therapies, and other advanced therapeutic treatments. It provides end-to-end services from DNA to BLA, helping global clients bring more innovative therapies to market faster for the benefit of patients.

Related Reading:WuXi AppTec Announces Production Collaboration with ViGeneron for Next-Generation Ophthalmic Gene Therapy Products | Bilingual News

According to the ViGeneron press release,VG901 is a gene replacement therapy that utilizes ViGeneron's next-generation vgAAV gene therapy platform based on AAV2, delivering the CNGA1 gene via intravitreal injection (IVT) to reduce the risk of retinal damage.Compared with subretinal injection, this improved vector provides a more convenient administration method and a wider distribution of the vector. Side-by-side comparison through in vivo expression with competitive capsid proteins demonstrated higher transduction efficiency. Moreover, intravitreal injection avoids the risk of retinal damage associated with subretinal administration and is less invasive than suprachoroidal injection. Due to vgAAV's ability to effectively cross biological barriers, this vector is suitable for various administration routes and target organs.

In previous preclinical study results, VG901 was shown to supplement the CNGA1 gene in a mouse model of retinitis pigmentosa. A GLP safety study involving a single intravitreal injection and six months of observation post-treatment confirmed that VG901 has good safety. Currently, WuXi AppTec has successfully assisted clients in completing GMP production for the drug at the clinical stage.

According to a press release from ViGeneron, Retinitis Pigmentosa (RP) is a group of hereditary retinal degenerative diseases that cause progressive vision loss, with no approved treatments currently available. RP initially manifests as night blindness during childhood or adolescence, gradually progressing to tunnel vision, central vision impairment, progressive vision deterioration, and ultimately leading to complete blindness. Retinitis Pigmentosa is the most common type of Inherited Retinal Diseases (IRDs). Statistics show that 1 in 3,500 to 1 in 4,000 people are affected by RP in the United States and Europe. It has been reported that mutations in the CNGA1 gene, which encodes a subunit of the CNG channel in rod photoreceptors, account for approximately 2%-8% of autosomal recessive Retinitis Pigmentosa (arRP).

WuXi ATU is committed to accelerating and transforming the development, testing, manufacturing, and commercialization of cell therapies, gene therapies, and other advanced therapeutic drugs. With a unique business model, it closely integrates robust testing capabilities with process development and manufacturing platform capabilities, providing partners with CGT therapy services from discovery to preparation for marketing application submission (DNA to BLA).

As of the second quarter of 2023, WuXi AppTec's Advanced Therapies Business Unit provided process development, testing, and manufacturing services for a total of 69 projects, including 7 Phase 3 clinical projects (with 2 projects in the marketing authorization application review stage and 2 projects in the preparation stage for marketing authorization application), 10 Phase 2 clinical projects, and 52 preclinical and Phase 1 clinical projects. In the first half of 2023, the company assisted a client in completing an FDA Biologics License Application (BLA) for what will become the world’s first innovative Tumor-Infiltrating Lymphocyte (TIL) therapy; it also helped a client complete a BLA for a lentiviral vector (LVV) project used in CAR-T cell therapy, becoming the first CGT CDMO in China to pass the LVV registration on-site inspection by the Center for Food and Drug Inspection (CFDI) of the National Medical Products Administration.

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ViGeneron is committed to bringing innovative gene therapies to those in need. The company is actively advancing its gene therapy pipeline for ophthalmic diseases while collaborating with leading biopharmaceutical companies in the fields of retinal diseases, central nervous system disorders, cardiovascular conditions, and other therapeutic areas. ViGeneron’s three next-generation gene therapy platforms aim to overcome the limitations of current adeno-associated virus (AAV) therapies: The first is the vgAAV gene therapy vector platform, which enables efficient transduction of target cells, overcomes biological barriers, and allows novel, minimally invasive administration routes (such as intravitreal injection and systemic delivery); the second is the REVeRT (Recombination via mRNA trans-splicing) technology platform, which facilitates highly efficient recombination of large gene fragments (>5Kb) in any tissue through capsid targeting; the third is an AAV gene therapy platform based on CRISPR-Cas, capable of modulating one or more genes by upregulating or downregulating target gene expression in vivo. ViGeneron is a privately held company founded in 2017 in Munich, Germany, by a seasoned team with extensive project experience in AAV vector technology and clinical ophthalmic gene therapy. For more information, please visit www.vigeneron.com.

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