Source BIOPROCESS ONLINE Innovation in gene therapy brings the potential for transforming patient care and obviating the need for chronic therapy through single-dose cures. Despite the potential long-term benefits of this new therapeutic modality, gene therapy companies face a number of underappreciated challenges. While there have been recent curative achievements in hepatitis C virus, curative
ZYNTEGLO is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT) European marketing authorization for ZYNTEGLO follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval bluebird bio, Inc. announced today that the European Commission (EC)
Source: Nature Study of almost half a million people links mutation that protects against HIV infection to an earlier death. The scientist who edited the genomes of twin girls in an attempt to make them resistant to HIV might have inadvertently shortened their life expectancy. People with two disabled copies of the CCR5 gene — the version
Encouraging Clinical Data Generated from Ongoing Proof-of-Concept Study Conducted by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget); Enrollment Expected to Complete in the First Half of 2020 Leverages Orchard’s Expertise in Treating Neurometabolic Diseases Using its Investigational Autologous Hematopoietic Stem Cell Gene Therapy Builds Upon Orchard / SR-Tiget Partnership to Develop Novel Gene Therapy Candidates Orchard Therapeutics, Fondazione
In light of additional data from ongoing trials of onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis), as well as the treatment’s final FDA label and just-announced launch price, the Institute for Clinical and Economic Review (ICER) today published an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA). Since the publication of ICER’s Final Evidence Report on
Source STAT he Food and Drug Administration on Friday approved the first gene therapy for a type of spinal muscular atrophy, a lifesaving treatment for infants that will also be the most expensive drug in the world. Known as Zolgensma, the gene therapy treats children under 2 years of age with spinal muscular atrophy, an
Source RAPS Addressing manufacturing controls for the cell and gene therapy industry, this article discusses criticality of establishing Chemistry Manufacturing Controls (CMC) Readiness, Critical Quality Attributes (CQAs) and Critical Process Parameters (CPPs) for cell and gene therapy products. The author suggests manufacturers need sound drug development and manufacturing facility plans covering a product’s lifecycle and
In Nov. 2018, The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) published technology roadmaps addressing needs and gaps in three key product areas: gene therapy, antibody-dug conjugates, and vaccines. The roadmaps were developed with the collaborative input of industry, academic, and government experts. This piece highlights technology opportunities for gene therapy manufacturing. The NIIMBL
Machine learning algorithms predict the repairs made to DNA after Cas9 cuts. The papersM.W. Shen et al., “Predictable and precise template-free CRISPR editing of pathogenic variants,” Nature, 563:646–51, 2018. F. Allen et al., “Predicting the mutations generated by repair of Cas9-induced double-strand breaks,” Nat Biotechnol, 37:64–72, 2019. During gene editing with CRISPR technology, the Cas9 scissors that
Source: Reuters Novartis AG is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal muscular atrophy (SMA), a treatment that could cost more than a million dollars, but the gesture comes with strings attached. The Swiss drugmaker wants insurers to commit to coverage for patients identified with the rare
