German Biotech to Develop Next-Generation Gene Therapy for Blindness

The German biotech ViGeneron has raised an undisclosed sum in a Series A round to fund the development of an ophthalmic gene therapy that could deliver bigger DNA sequences into a wider range of cells than current technology.

The round was led by two Chinese investors, the VC firm Sequoia Capital China and the pharma and medtech giant WuXi AppTec. ViGeneron will use the money to advance its preclinical gene therapy technology for the treatment of two undisclosed blindness conditions that currently have no approved treatments.

ViGeneron, based in Munich, develops gene therapies based on a viral vector called adeno-associated virus that is commonly used in gene therapies. However, this type of vector can have drawbacks such as being unable to cross physical barriers within the body like the blood-brain barrier. ViGeneron has genetically engineered adenoviral particles to cross these barriers more easily and could its gene therapies reach many types of cells in the retina.

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Another limitation of adenoviral vectors that ViGeneron aims to address is that they can’t carry long DNA sequences, which is problematic if you need to deliver large genes. To circumvent the problem, ViGeneron is developing vectors that carry different sections of the gene into a cell. These DNA sequences are separately transcribed into mRNA molecules that are then assembled together into a single mRNA molecule carrying the instructions to create a therapeutic protein.

The field of gene therapy has undergone a renaissance in recent years, and the FDA and EMA are seeing a surge of clinical trial applications using this technology. ViGeneron is part of a current trend of companies developing next-generation gene therapies that more efficient at delivering therapeutic genes. Other companies in the field are developing methods to turn the therapy on and off as needed, and to treat complex diseases that involve multiple genetic mutations.

The evolution of medicines from small molecules to proteins has driven increased therapeutic benefits in the past; the next generation of gene therapies holds tremendous promise for patients,” stated Caroline Man Xu, the CEO of ViGeneron.


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