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
Data presented at the American Academy of Neurology (AAN) meeting this week on experimental therapeutics for Huntington’s disease gave some cause for optimism. And, as good research does, they identified new questions as they answered current ones. At the meeting, researchers presented new data on antisense oligonucleotide HTTRx (RG-6042, Ionis Pharmaceuticals Inc./Roche Holding AG) that
Dear Duchenne Community, We want to provide you with an update on the progress of SolidBiosciences IGNITE DMD Phase I/II clinical trial for our investigational microdystrophin gene transfer, SGT-001. Since we communicated preliminary clinical data in February, we advanced the study to evaluate SGT-001 at a higher dose in the second cohort of patients. As
NIH scientists and funding contributed to development of experimental treatment A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined
The past 10 years witnessed a significant increase in the approval of cellular and gene therapy products worldwide. The US Food and Drug Administration (FDA) approved 3 gene therapy products within the last 4 months of 2017. The objective of this study was to examine the approval characteristics, discontinuations and cost of all cellular and
Researchers have infused cells edited using CRISPR-Cas9 into two patients in a trial conducted at the University of Pennsylvania, NPR reports today (April 16). A university spokesperson confirmed in an emailed statement to The Scientist that the trial is underway and that two patients, one with multiple myeloma and one with sarcoma, have been treated so far. The study is
Researchers discovered thousands of genes essential for cancer’s survival and ranked which ones show the most promise as drug targets for developing new treatments In one of the largest studies of its kind, researchers used CRISPR technology to disrupt every gene in over 300 cancer models from 30 cancer types and discover thousands of key
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency.
