Le Parisien, one of France’s leading daily newspapers, published yesterday a full-page report highlighting results obtained by our team in collaboration with the University of California – UC Irvine – in Professor Krzysztof Palczewski’s lab. These results demonstrate the restoration of vision in mice blind from birth, with the objective of developing precision gene therapies for humans.
A single—or sometimes only a few—erroneous letters in the genetic code can be sufficient to cause severe genetic diseases. The central challenge is therefore to correct these mutations in a precise and safe manner, in order to restore natural biological function.
Recent advances in genome editing, such as base and prime editing systems, now make such precise corrections possible. However, fully realizing their therapeutic potential critically depends on the development of delivery vectors capable of safely and efficiently transporting these complex molecular systems into cells.
These delivery vehicles—the mRNA-based biological vectors developed by GEG-Tech—have enabled research partnerships with leading academic institutions, including UC Irvine on the US West Coast and the Broad Institute of MIT and Harvard on the East Coast.
Results obtained at UC Irvine using our proprietary mRNA vector system demonstrate that a single disease-causing mutation can be corrected efficiently and safely.
In a mouse model of inherited human blindness, delivery of a base-editing system via an mRNA-based vector resulted in precise gene correction and robust restoration of retinal activity, representing a first-of-its-kind demonstration and opening the door to a new era of precision gene therapy.
The next step will be the creation of a US-based venture in partnership with UC Irvine and Professor Krzysztof Palczewski, combining our respective technologies to develop these next-generation gene therapies.
