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Nature Communications publication reveals highly efficient AAV delivery of large genes using ViGeneron´s REVeRT technology
REVeRT, a dual vector approach based on mRNA trans-splicing, enables delivery of genes larger than the usual AAV packaging size in various tissues Potential to address prevalent inherited diseases caused by larger genes such as Stargardt disease Delivery of CRISPR modules allows bi-directional modulation of multiple genes ViGeneron GmbH (ViGeneron), a next-generation gene therapy company, announces a peer-reviewed publication in Nature Communications, showcasing the potential of its proprietary dual adeno-associated virus (AAV) vector technology based on mRNA trans-splicing, REVeRT (REconstitution Via mRNA Trans-splicing). The data demonstrate the delivery of genes larger than the usual AAV packaging size with high reconstitution efficiency, enabling gene supplementation or simultaneous knockout and transcriptional activation of different genes. While…
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ViGeneron announces EMA Approval of Clinical Trial Application for VG901, a gene therapy to treat Retinitis Pigmentosa
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…
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ViGeneron presents preclinical data on intravitreal gene therapy of Stargardt disease at ESGCT
Efficient transfer of larger gene via ViGeneron’s proprietary technology platforms REVeRT and vgAAV ViGeneron advances its preclinical programs for Stargardt disease and Retinitis Pigmentosa into IND-enabling activities and clinical stage development 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…
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ViGeneron announces follow-on collaboration with Daiichi Sankyo to develop a novel gene therapy for prevalent eye diseases
ViGeneron GmbH, a next-generation gene therapy company, today announced a follow-on collaboration with Daiichi Sankyo Company, Limited (“Daiichi Sankyo”) to utilize ViGeneron’s novel engineered recombinant adeno-associated virus vectors (vgAAVs) to address an undisclosed target for the treatment of prevalent eye diseases. Financial terms are not disclosed. ViGeneron has worked on this program together with Daiichi Sankyo since early 2021.This follow-on collaboration allows the companies to create and validate vgAAV-based therapeutic candidates for the undisclosed target through in-vivo animal studies. Through a unique and stringent in vivo selection procedure, where an AAV2-based peptide-display library was intravenously administered in mouse models followed by isolation of vector DNA from target cells after only…
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ViGeneron signs gene therapy strategic collaboration and option agreement with Regeneron for one inherited retinal disease target
Regeneron receives access to ViGeneron’s vgAAV capsids for one inherited retinal disease target and an option for an exclusive license to develop and commercialize the gene therapy product ViGeneron is eligible to receive upfront payment, research funding, option exercise fee, development and commercial milestone payments, plus royalties on net sales The collaboration further validates ViGeneron’s next generation vgAAV platform and ophthalmic gene therapy expertise ViGeneron GmbH, a next-generation gene therapy company, today announced a target-specific strategic collaboration and option agreement with Regeneron Pharmaceuticals Inc. (Regeneron) to develop and commercialize a gene therapy product based on ViGeneron’s novel engineered recombinant adeno-associated virus vectors (vgAAVs) to treat an inherited retinal disease (IRD).…
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ViGeneron announces research collaboration with Daiichi Sankyo to evaluate vgAAV for novel ophthalmic gene therapy
ViGeneron GmbH, a gene therapy company, today announced a research collaboration to utilize its novel engineered adeno-associated virus (vgAAV) vectors with Daiichi Sankyo Company, Limited (“Daiichi Sankyo”), for delivering a novel therapeutic protein to address an undisclosed target in a highly prevalent ophthalmic disease. ViGeneron’s vgAAVs enable the efficient transduction of target cells via intravitreal injection that allows efficient lateral spreading and minimizes the risk of collateral damage caused by conventional subretinal injection. “There is significant unmet medical need for a sustained therapy to treat eye diseases. ViGeneron’s innovative gene therapy expertise combined with Daiichi Sankyo’s ophthalmic knowledge creates the potential to develop a sustained novel gene therapy to overcome…
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ViGeneron signs global collaboration agreement for ophthalmic gene therapy development
. – Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target – The companies will use ViGeneron’s proprietary vgAAV technology to efficiently transduce target cells via intravitreal injections ViGeneron GmbH, a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. (Nasdaq: BIIB, Cambridge, Mass., USA) to develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors to treat inherited eye diseases. The companies will use ViGeneron’s proprietary vgAAV, novel engineered AAV capsids, to efficiently transduce retinal cells via intravitreal injections. “Gene therapy has become a clinical reality. At ViGeneron, we are…
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ViGeneron and WuXi Advanced Therapies enter Strategic Manufacturing Partnership for Next-Generation Ophthalmic Gene Therapy
ViGeneron, a gene therapy company, and WuXi Advanced Therapies Inc. (WuXi ATU), a leading Contract, Testing, Development and Manufacturing Organization (CTDMO), today announced a strategic partnership to accelerate production for the clinical development of VG901, a next-generation ophthalmic gene therapy program. ViGeneron’s innovative gene therapy pipeline addresses ophthalmic diseases with high unmet medical needs. Its lead product VG901 targets a disease gene for Retinitis pigmentosa (RP), for which there is currently no approved treatment option. VG901 is developed from ViGeneron’s proprietary next generation vgAAV vector platform that allows a superior transduction of retinal cells and intravitreal, a less invasive treatment administration. WuXi ATU will accelerate the development of VG901 by…
