19 Jul
NIB is honoured to be a part of a groundbreaking gene therapy development

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NIB is honoured to be a part of a groundbreaking gene therapy development


National Institute of Biology (NIB) is a proud technical supplier to AveXis, which recently received U.S. Food and Drug Administration (FDA) approval for Zolgensma®, the first and only gene therapy for pediatric patients less than 2 years of age with spinal muscular atrophy (SMA), a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis.

“NIB is honored to be a part of this groundbreaking moment,” said dr. Matjaž Kuntner, the director of NIB. “We are proud to contribute our technical expertise to help accurately characterize the drug, which will ultimately have a profound effect on the SMA community.”

NIB serves as a contract lab for AveXis, providing time-effective support of downstream process development and consultation on specific analytical techniques, such as droplet digital polymerase chain reaction or real-time PCR, required for accurate characterization of the drug product.

About NIB
Department of Biotechnology and Systems Biology at the National Institute of Biology is holder of worldwide recognized expertise in the field of virus diagnostics, characterization and quantification. We are involved in development of purification processes of gene therapy drugs with absolute quantification of viral genomes by droplet digital PCR (ddPCR), quantification of host cell DNA, characterization of nucleic acid impurities by high-throughput sequencing (HTS), and complementary analysis of virus particles with electron microscopy (EM). For mentioned technologies we develop different assays and perform their validation and other custom contract research for pharmaceutical industry.

Zolgensma® is a registered trademark of AveXis.