This article highlights a new partnership between GenKOre and Revvity. Both of these companies have different areas of expertise:
GenKOre has created a new, small version of CRISPR-Cas9. Just like the original CRISPR-Cas9, GenKOre’s small version can edit DNA (our genetic code). The benefit of the small size is that GenKOre’s small CRISPR-Cas9 can fit into an adeno-associated virus (AAV), unlike the original, much larger, CRISPR-Cas9 version. AAVs are how many gene therapies are delivered, so a smaller CRISPR-Cas9 able to fit into an AAV is an important development. In addition to this small CRISPR-Cas9, GenKOre has created other gene therapy tools - all deliverable by AAV.
Revvity’s expertise is in developing novel AAVs, which complements GenKOre need to deliver their new compact CRISPR-Cas9 tool. Some types of AAVs are better at delivering therapies to specific cells in the body than others. Revvity is working on creating advanced AAVs that are both efficient and safe.
Overall, GenKOre has the knowledge needed to create new gene therapies, while Revvity has the know-how to deliver these gene therapies to tissues like the retina.
What this means for Usher syndrome: Both companies hope to combine their unique areas of expertise to develop therapies for rare diseases. One key goal of their collaboration is to develop effective gene therapy options for two retinal degenerative diseases, one of which is USH2A. If the strategies these companies use to develop a USH2A therapy are successful, they could also apply them to develop treatments for other USH subtypes.
