Fujifilm to make AAVs for Regenxbio gene therapies
Under terms of the deal, contract development and manufacturing organisation (CDMO) Fujifilm Diosynth Biotechnologies will manufacture Regenxbio’s lead candidates – including RGX-314 and RGX-501 – as it looks to take them through late-stage clinical trials and into commercialisation.
Speaking this week on a financial call, CEO Ken Mills said the deal with Fujifilm “expanded the breadth and depth capabilities in development” and secured “access to long term dedicated cGMP suite capacity” at up to a 2,000L scale.
“This partnership enhances our existing internal capabilities through which we were able to produce NAV [adeno-associated virus] AAV at up to 200 liter scale and it fortifies our position of leadership in AAV manufacturing.”
All is Regenxbio’s candidates use viral vectors from its proprietary gene delivery platform, called the NAV Technology Platform. The technology uses NAV AAVs as a delivery vehicle by inserting a healthy gene inside the NAV’s shell, before administering the vector into the patient. Once inside the body the healthy gene payload is delivered to an affected cell’s nucleus where it transcribes its DNA into RNA. This in turn encodes a specific protein that the body was missing and, when the body replicates the cell, it has the potential to correct the disease.
Regenxbio has its own facility in Rockville, Maryland and the deal does not restrict the firm from investing in its own cGMP manufacturing capabilities.
Fujifilm has invested in commercial gene therapy capabilities on the back of increased demand from biopharma.
Earlier this year, the CDMO opened an 80,000 square-foot plant at its site in Texas, acquired from Kalon Therapeutics in 2014.