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Two Very Different Flavors Of Ocular Gene Therapy Get A Boost

 
• By Mark Ratner

The 2021 collaboration between Abbvie and Regenxbio to develop an anti-VEGF gene therapy for chronic retinal diseases is a noteworthy deal in the gene therapy space. But for both clinical and commercial reasons, it is a one-off opportunity and not an indicator of the overall potential of gene therapies to address eye diseases. Interest in optogenetics, however, which is the introduction of light-sensing genes into cells in the eye to restore vison, may be on the rise.

Science Matters
• Source: Alamy

The first thing that comes to mind when thinking about gene therapy is its ability to replace a missing gene or correct a defective one. A gene therapy vector can also be used to insert instructions for cells to begin making a therapeutic protein. Regenxbio Inc.’s RGX-314, which uses an adeno-associated virus (AAV) vector to deliver an inhibitor of vascular endothelial growth factor (VEGF), is one such example.

In a move to boost its eye care portfolio following the acquisition of Allergan plc, AbbVie Inc. licensed rights to Regenxbio’s RGX-314, which is in a pivotal trial...

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