CNGA1

VG901 is the first-in-class and only clinical-stage therapy designed to deliver the functional CNGA1 gene intravitreally (IVT) to target retinal photoreceptor cells in patients with retinitis pigmentosa FDA Rare Pediatric Disease Designation qualifies ViGeneron for potential eligibility for a Priority Review Voucher (PRV) upon marketing approval The independent Data Safety Monitoring Board (DSMB) unanimously approved dose escalation in VG901’s ongoing Phase 1b clinical trial Jan. 08, 2025 -- ViGeneron GmbH, a next-generation clinical-stage gene therapy company, today announced two important milestones for its novel gene therapy candidate VG901, to treat patients with retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to VG901, and the independent Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing Phase 1b clinical trial. The FDA’s RPDD acknowledges the critical need for treatments addressing rare pediatric conditions like retinitis pigmentosa caused by CNGA1 mutations. This designation qualifies ViGeneron to potentially secure a Priority Review Voucher (PRV) upon VG901’s marketing approval. A PRV enables accelerated FDA review of any drug candidate in the company’s pipeline, reducing the review period to six months, and is transferable for use or sale. “This RPDD recognition from the FDA highlights the significant unmet medical need in retinitis pigmentosa and underscores VG901's therapeutic potential as the first-in-class and only clinical-stage therapy targeting retinitis pigmentosa associated with mutations in the CNGA1 gene,” said Dr. Caroline Man Xu, ViGeneron’s Co-founder and CEO. “In addition to the previously granted FDA Orphan Drug Designation for VG901, the RPDD designation further supports our efforts to accelerate the development of VG901.” The ongoing Phase 1b trial is designed to evaluate the safety and preliminary efficacy of VG901, which leverages ViGeneron’s novel next-generation vgAAV capsid to deliver the functional CNGA1 gene to retinal photoreceptor cells via intravitreal injection. The DSMB, composed of independent experts, conducts a rigorous evaluation of trial data after each cohort of patients. “The DSMB has unanimously recommended proceeding with dose escalation in the ongoing VG901 Phase 1b clinical trial,” commented Dr. Bart P Leroy, Ophthalmologist & Clinical Geneticist, Head of Department of Ophthalmology, Ghent University Hospital, Belgium, and the DSMB Chair for this trial. “No dose-limiting adverse events related to VG901 have been reported in the first-dose cohort to date. This marks a critical step toward advancing to the higher dose and represents an important milestone in its clinical development.” Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision”, central visual impairment, reduced visual acuity, and ultimately, complete blindness. Retinitis pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively. 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). VG901 is a first-in-class and only clinical-stage gene therapy to treat CNGA1-associated Retinitis Pigmentosa (RP). The therapy approach utilizes vgAAV, ViGeneron's novel proprietary adeno-associated virus (AAV) vector, to deliver the CNGA1 gene via intravitreal injection. VG901 has demonstrated in vivo functionality by delivering the CNGA1 gene in a mouse model, while a GLP safety study with a six-month observation period confirmed its safety, durable expression, and sustained tolerability. VG901 is currently in a Phase 1b clinical trial, an open-label, single-arm, dose-escalation study assessing the safety, tolerability, and preliminary efficacy of a one-time intravitreal administration of VG901. ViGeneron is dedicated to bringing gene therapy innovations to people in need. The company is advancing its proprietary, clinical-stage gene therapy pipeline to treat ophthalmic diseases, while partnering with leading biopharmaceutical players in retinal diseases, CNS, cardiovascular 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, the 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 and systemic administration. The second, the REVeRT (REconstitution Via mRNA Trans-splicing) technology platform, allows for efficient reconstitution of large genes (>5kb) in any tissue that can be targeted with a given capsid. The third, the AAV Transactivation is a CRISPR-Cas–based AAV gene therapy platform that enables the regulation of one or multiple genes in vivo by increasing or inhibiting their expression. 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. The content above comes from the network. if any infringement, please contact us to modify.

VG901 is the first and only clinical-stage therapy designed to deliver a functional CNGA1 gene to retinal photoreceptor target cells in retinitis pigmentosa patients VG901, using ViGeneron’s proprietary next-generation technology platform vgAAV, is administered intravitreally (IVT), offering enhanced ease of delivery, broader vector distribution, and mitigating the risk of retinal damage linked with subretinal administration VG901 granted Orphan Drug Designation by FDA MUNICH, Germany, April 10, 2024 (GLOBE NEWSWIRE) -- ViGeneron GmbH, a next-generation clinical-stage gene therapy company, today announced that the first patient has been dosed in its Phase Ib clinical trial evaluating intravitreal injection of VG901 to treat retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. This milestone marks an important advance as the company continues to leverage its next-generation technology platforms to develop groundbreaking gene therapies addressing critical unmet medical needs. “By delivering a functional CNGA1 gene to retinal photoreceptor target cells, VG901 offers a therapeutic potential in addressing the genetic root cause for patients with retinitis pigmentosa affected by CNGA1 mutations,” commented Prof. Dr. Katarina Stingl, Head of the Clinic for Hereditary Retinal Degenerations in the Center for Ophthalmology and the Center for Rare Eye Diseases at the University of Tübingen, Germany, and the Principal Investigator for this trial. "We are excited to learn about the potential of this novel therapy and hope to make a meaningful difference to patients' lives.” “Dosing our first patient in the VG901 Phase Ib clinical trial is a significant step forward for the company and for the patients we aim to benefit. VG901 has also been granted FDA Orphan Drug Designation status. We look forward to progressing the clinical development of this potentially transformative therapy,” said Dr. Caroline Man Xu, ViGeneron’s Co-founder and CEO. “Not only is the Phase Ib trial designed to provide key insights into the safety and preliminary efficacy of VG901, it is also a pivotal step in validating our next generation vector platform vgAAV, which has demonstrated superior transduction efficiency and enables intravitreal delivery.” The on-going Phase Ib clinical trial is an open-label, single-arm, dose-escalation study investigating the safety, tolerability, and preliminary efficacy of an intravitreal injection of VG901, a first-in-class CNGA1 gene therapy for autosomal recessive RP. For more information on the trial, please visit clinicaltrial.gov [NCT06291935]. About Retinitis Pigmentosa (RP) Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision”, central visual impairment, reduced visual acuity, and ultimately, complete blindness. Retinitis pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively. 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). About ViGeneron GmbH ViGeneron is dedicated to bringing gene therapy innovations to people in need. The company is advancing its proprietary, clinical-stage gene therapy pipeline to treat ophthalmic diseases, while partnering with leading biopharmaceutical players in retinal diseases, CNS, cardiovascular 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, the 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 and systemic administration. The second, the REVeRT (REconstitution Via mRNA Trans-splicing) technology platform, allows for efficient reconstitution of large genes (>5kb) in any tissue which can be targeted with a given capsid. The third, the AAV Transactivation is a CRISPR-Cas–based AAV gene therapy platform that enables the regulation of one or multiple genes in vivo by increasing or inhibiting their expression. 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 info@vigeneron.com ViGeneron Media Contact MC Services AG Shaun Brown / Julia von Hummel phone: +49 (0)89 2102280 vigeneron@mc-services.eu

MUNICH, Germany I August 22, 2023 I ViGeneron GmbH (ViGeneron), a next-generation gene therapy company, today announced that the European Medicines Agency (EMA) has approved the Clinical Trial Application (CTA) for VG901, a potentially transformative gene therapy to treat CNGA1-associated Retinitis Pigmentosa (RP), an ocular disorder currently lacking approved therapies. VG901 uses vgAAV, ViGeneron’s proprietary adeno-associated virus (AAV) vector to deliver the CNGA1 gene via intravitreal (IVT) injection, thereby reducing the risk of retinal damage, which is associated with subretinal administration. The Phase Ib dose-escalation clinical trial will evaluate safety, tolerability and efficacy of VG901 in patients with RP due to biallelic CNGA1 mutations. This trial is part of an international regulatory strategy for the clinical development of VG901. Further interactions with regulatory authorities are currently being prepared. Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron said: “This is an important step in our mission to bring a novel gene therapy to patients born with CNGA1 mutations, to save their eyesight, potentially. We are excited to develop this first-in-class gene therapy to provide a potential cure for patients who currently have no treatment options.” In preclinical studies, ViGeneron observed that VG901 has the potential to supplement the CNGA1 gene in a mouse model of RP. In addition, a GLP-safety study applying a single intravitreal injection followed by a 6-month post treatment observation period confirmed the safety of VG901. Furthermore, ViGeneron’s manufacturing partner, WuXi Advanced Therapies (WuXi ATU), has successfully completed GMP manufacturing of the clinical trial material. VG901 was developed using ViGeneron’s proprietary next generation gene therapy technology platform vgAAV – a platform based on AAV2 – to be delivered by IVT. This improved vector provides a greater ease of administration and wider vector distribution compared with subretinal injection, commonly used in retinal gene therapy studies. Side by side comparison of in vivo expression versus a competitor capsid demonstrated superior IVT transduction efficiency.  In addition, IVT delivery avoids the risk of retinal damage associated with subretinal administration and is also less invasive than suprachoroidal injection. Due to its ability to cross barriers, this vector is suitable for various routes of administration and target organs. It can be combined with our second platform, which allows delivery of genes larger than 5kb, as required for some more prevalent rare diseases. About Retinitis Pigmentosa (RP) Retinitis Pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. There are currently no approved treatments for RP. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision,” central visual impairment, reduced visual acuity and, ultimately, complete blindness. Retinitis Pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively. 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). About ViGeneron GmbH 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, cardiovascular 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 and systemic administration. The second, the REVeRT (REconstitution Via mRNA Trans-splicing) technology platform, allows efficient reconstitution of large genes (>5kb) in any tissue which can be targeted with a given capsid. The third, AAV Transactivation is a CRISPR-Cas–based AAV gene therapy platform that enables to regulate one or multiple genes in vivo by increasing or inhibiting their expression. 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 . SOURCE: ViGeneron