Abbvie puts first toe in gene therapy, courtesy of Regenxbio

With safety concerns continuing to dog AAV-based gene therapies Regenxbio’s ability to bag Abbvie as partner for RGX-314, its gene therapy for eye diseases, is encouraging. Shares in the small biotech opened 23% higher, with a hefty $370m up front contributing to the rise. On a call executives declined to say whether Abbvie had seen any of the data due on RGX-314, though the assumption is presumably yes. Most importantly, investors are waiting for a first look at suprachoroidal delivery of RGX-314, requiring a simple in-office procedure rather than the surgery involved in subretinal administration, and success could substantially broaden commercial potential. Still, many groups are going after longer-acting VEGF agents, an area that is already very competitive; Regenxbio is arguably one of the most advanced with a gene therapy, however, so perhaps Abbvie thought the opportunity was worth a bet. The big pharma group has a large eye care division, though this will be its first step into gene therapy. The terms of the deal see the partners sharing future development costs, with Abbvie leading commercialisation; Regenxbio will co-commercialise and share US profits, and receive royalties in “late teens/early 20s” percent elsewhere.