ViGeneron presents preclinical data on intravitreal gene therapy of Stargardt disease at ESGCT

Munich, Germany, October 11, 2022 –– ViGeneron GmbH, a next-generation gene therapy company, today announced the presentation of preclinical data on its program VG801 in ABCA4‐associated Stargardt disease (STGD1). VG801 is based on the Company’s novel adeno-associated virus (AAV) technology platforms, enabling the efficient expression of large genes in retinal photoreceptors after intravitreal injection. The data from a Stargardt disease model will be presented in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Congress, Edinburgh, October 11-14, 2022.

Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron said:” Recombinant AAV vectors are an established platform for effective gene therapies today. However, their limited packaging capacity and transduction efficacy are a major challenge for the development of novel gene therapies. The data presented at ESGCT demonstrate how the combination of our REVeRT (REconstitution Via mRNA Trans-splicing) and vgAAV platforms may overcome these limitations and enable the efficient delivery of large genes with high transduction efficacy.”

The poster titled “A novel dual AAV approach for intravitreal gene therapy of Stargardt diseasewill be presented on October 13, 2022. It describes the potential of VG801 for the functional transfer of the ABCA4 gene in a Stargardt disease mouse model. Because the ABCA4 gene exceeds the packaging capacity of AAV vectors, the gene was transferred using the REVeRT technology. Split genes encoding the 5’ and 3’ parts of human ABCA4 were packaged into dual vgAAV vectors and full-length protein was generated via mRNA trans-splicing. REVeRT has demonstrated a high reconstitution efficiency at transcript and protein levels of ABCA4.

Stargardt disease is one of the most prevalent inherited retinal diseases, affecting approximately 1 in 8,000 to 10,000 people worldwide.[i] The most common form of the disease is caused by a genetic mutation of the ABCA4 gene, ultimately leading to vision loss. Currently, there are no treatments for Stargardt disease available.

“We are excited that our product candidate VG801 for Stargardt disease is ready for IND-enabling studies. With lead candidate VG901 for Retinitis Pigmentosa currently undergoing IND enabling studies we are on track to achieve our goal of providing novel treatment options for patients with eye disorders “, added Dr. Caroline Man Xu.

ViGeneron’s lead product candidate VG901 targets a disease gene for Retinitis Pigmentosa (RP), an inherited retinal disease that causes progressive vision loss and for which there are currently no approved treatments available. VG901 is developed with ViGeneron’s next generation vgAAV vector platform, which is designed to enable a superior transduction of retinal cells upon intravitreal delivery.

About ViGeneron

ViGeneron is dedicated to bringing gene therapy innovations to people in need. The company is advancing its proprietary gene therapy pipeline to treat ophthalmic diseases, while partnering with leading biopharmaceutical players in retinal diseases, CNS, and other disease areas. ViGeneron’s three novel next-generation gene therapy platforms are geared towards addressing the limitations of existing adeno-associated virus (AAV)-based gene therapies. The first, vgAAV vector platform, enables a superior transduction efficiency of target cells and is designed to overcome biological barriers, thus enabling novel, less invasive routes of administration such as intravitreal injections. The second, REVeRT (REconstitution Via mRNA Trans-splicing) technology platform, allows efficient reconstitution of large genes (>5Kb) in various tissues such as retina, brain, heart, liver, and skeletal muscle. The third, AAV Transactivation is a CRISPR-Cas–based AAV gene therapy platform that enables to regulate one or multiple genes in vivo. Privately-owned ViGeneron was founded in 2017 by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit

ViGeneron Contact
ViGeneron GmbH
Dr. Caroline Man Xu
Co-Founder and CEO
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Shaun Brown / Julia von Hummel
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