Source BioProcess International Over 20 cell and gene-based therapies are expected to be filed or to receive approval decisions over the next 18 months, according to the Alliance for Regenerative Medicine (ARM). Earlier this year, the US Food and Drug Administration (FDA) laid out its action plan to deal with the large upswing in the
Source: Life Science Leader Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient. Exhibit A: Novartis’ Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment, priced at $2.125 million, is the world’s most expensive drug to date. But cell and gene therapies are their own animals; the differences between
Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase precision during gene therapy in humans. The new variant reduced unintended changes in DNA compared to its wildtype, suggesting it could play a role in gene therapies that require high precision. The study by researchers from Karolinska
