Biochromatography development and manufacturing company, BIA Separations will work with AveXis for the commercial purification process of its newly approved gene therapy drug Zolgensma (onasemnogene abeparvovec-xioi) and the rest of its pipeline.
A BIA Separations spokesperson told us that it started working with AveXis, a Novartis company, in other capacities several years ago, when it gaged the available technology for the purification of gene therapy products.
The treatment has been shown to halt disease progression in SMA patients and has become the first gene therapy for this treatment indication approved by the US Food and Drug Administration.
Zolgensma is a one-time infusion that provides a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression. If untreated SMA, a rare genetic disease, leads to progressive muscle weakness, paralysis, and can lead to permanent ventilation or death by age two.
BIA Separations will use its experience in viral and DNA purification for adeno-associated virus (AAV), and chromatographic technology for the purification and analysis of biomolecules as it supports AveXis’ gene therapy pipeline.
Andy Stober, SVP of technical operations for AveXis, stated that the work BIA has done in AAV purification and chromatographic technology were ‘important contributions’ in bringing gene therapies forward.
He added that BIA has shared and operated with “the same sense of urgency” as AveXis to “bring gene therapy to the SMA community.”
After the approval of Zolgensma, Catalent Biologics’ newly purchased subsidiary Paragon entered an agreement with AveXis as well. Per the agreement, Paragon will develop and manufacture Zolgensma in a dedicated production space at its commercial manufacturing center in Maryland, US.
Paragon will provide AveXis with access to Catalent’s AAV gene therapy development, manufacturing, and process characterization capabilities, and process development for clinical supply of any additional viral therapies in AveXis’ pipeline.
Additionally, Novartis entered an agreement with SAVSU Technologies to use the company’s cold chain shipment technology as AveXis’ pipeline develops.