ViGeneron GmbH
ViGeneron is based on our passion to deliver gene therapy innovations and advances to people in need. Our proprietary science solves major limitations in current gene therapy approaches. At ViGeneron we are dedicated to developing innovative gene therapies to treat ophthalmic diseases with high unmet medical need, as well as partnering with leading biopharmaceutical players in other disease areas. The company’s pipeline is built on two unique and proprietary adeno-associated virus (AAV) technology platforms. The first, vgAAV gene therapy vector platform, allows superior transduction efficiency and intravitreal, less invasive treatment administration. The second, REVeRT vector platform, targets diseases caused by mutations in large genes.
Company details
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- Business Type:
- Manufacturer
- Industry Type:
- Clinical Services
- Market Focus:
- Internationally (various countries)
About Us
ViGeneron was founded in 2017 as a spin-off of the Ludwig-Maximilians-University (LMU) Munich. The company’s founding team includes highly experienced executives and internationally renowned experts with track records in developing retinal gene therapy programs from discovery to clinical stage.
A next generation gene therapy company
At ViGeneron, we are dedicated to developing innovative gene therapies to treat ophthalmic diseases with high unmet medical need.
Our two novel gene therapy platforms are designed to overcome the limitations of existing adeno-associated virus (AAV)-based gene therapies and enable the delivery of larger genes as well as superior transduction and less invasive administration routes.
With our next generation vector platforms, we are creating an in-house pipeline in ophthalmic gene therapy. Also, we are interested in partnerships with biopharmaceutical players in other disease areas.
The novel techniques we develop are based on well-founded scientific results. Together with leading biopharmaceutical partners, we are also addressing other exciting opportunities beyond ophthalmology, potentially in the fields of neuroscience, cardiovascular and liver.
Innovative Sciences
Gene therapy has become a clinical reality.
Adeno-associated virus (AAV) vectors are safe and efficient gene transfer tools based on naturally occurring non-pathogenic AAVs. AAV-based vectors have been extensively evaluated in preclinical and clinical studies and are increasingly used in the treatment of previously incurable diseases. These vectors have proven to be safe and efficient for the long-term expression of supplemented transgenes. Nevertheless, a broader use of AAVs in gene therapy is currently hampered by two important factors:
- The limited DNA uptake capacity, which precludes the treatment of large genes
- The limited ability to cross biological barriers, which demands local delivery and restricts the route of administration.
At ViGeneron we have developed two next-generation gene therapy platforms designed to overcome the above-mentioned limitations of existing AAV-based gene therapies.
ViGeneron’s pipeline is built on our novel and proprietary AAV technology platforms and addresses ophthalmic diseases with high unmet medical need.
Pipeline
ViGeneron’s pipeline in gene therapy addresses ophthalmic diseases with high unmet medical need, including two lead programs in development for inherited retinal diseases where no approved treatment options are currently available.
For more information about the indications we are working on and the therapies our products offer, please click on the individual indication.
Partnering Opportunities
At ViGeneron, we’re experts in developing new gene therapy solutions.
While we’re focused on gene therapy to cure ophthalmic diseases, our next-generation gene therapy technology platforms also offer opportunities for use in other diseases such as those affecting the central nervous system, liver, or cardiovascular system.
The vgAAV vector platform, based on novel engineered AAV capsids, enables a superior transduction of target cells and is designed to efficiently cross biological barriers. These attributes enable intravitreal, less invasive gene delivery but also allows vgAAV vectors to target a broad spectrum of cell types in the retina and potentially in other tissues, such as the central nervous system.
In addition, our REVeRT vector platform utilizes a unique mRNA trans-splicing technology to develop safe and effective AAV gene therapy targeting large genes (>5Kb). REVeRT achieves high reconstitution efficiencies at the mRNA and protein level and is broadly applicable for various disease areas.
We are actively looking to work with leading biopharmaceutical companies or academics that wish to exploit these opportunities.