IZB-alumnus ViGeneron GmbH, a next-generation gene therapy company, presented its VG901 program for CNGA1-associated retinitis pigmentosa (RP) in an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Los Angeles from May 16-23, 2023. VG901 is a gene supplementation therapy that uses vgAAV, ViGeneron’s proprietary adeno-associated virus (AAV) vector, to deliver the CNGA1 gene via intravitreal injection. The presentation featured in vitro and in vivo proof-of-concept efficacy data and details on the GLP (Good Laboratory Practice) safety study.

Dr. Caroline Man Xu, Co-founder, and CEO of ViGeneron said: “As a next-generation gene therapy company, ViGeneron is committed to developing innovative treatments for patients in need. Our VG901 program, which uses vgAAV vector to deliver the CNGA1 gene via intravitreal injection, has demonstrated promising preclinical data. This represents a significant step towards providing a novel, first-in-class therapy for patients born with CNGA1 mutations and potentially saving their eyesight as they age. We look forward to advancing this program into clinical development and providing a potential cure for patients who currently have no treatment options.”

Autosomal recessive retinitis pigmentosa (arRP) is a hereditary disease, with approximately 2% to 8% of all arRP cases being attributed to genetic defects in the CNGA1 gene. Loss of CNG channels in rod photoreceptors due to CNGA1 mutations leads to dysfunction and a slow decrease in cone function. The preclinical data shows that VG901 has the potential to supplement the CNGA1 gene in a mouse model of RP. Furthermore, a GLP-safety study applying a single intravitreal injection followed by a 6-month post treatment observation period confirmed the safety of VG901.

About Retinitis pigmentosa (RP)
Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. RP initially presents as night-time blindness during childhood and early adulthood, progressing to peripheral visual field loss and “tunnel vision”, central visual impairment, reduced visual acuity, in most cases the disease progresses to a complete loss of vision. It is estimated that 50-60% of RP are inherited autosomal recessively. Mutations in the CNGA1 gene, encoding a subunit of CNG channels in rod photoreceptors, are reported to cause approximately 2% – 8% of autosomal recessive retinitis pigmentosa (arRP).

ViGeneron develops two novel next-generation gene therapy platforms
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 www.vigeneron.com.