As new gene therapies make their way through the approval process, companies are scrambling to develop manufacturing practices capable of producing high volumes of clinical grade product while keeping costs down. Pall Biotech’s single-use chromatography capsules deliver efficient purification for large-scale production.
A task force of the Alliance for Regenerative Medicine (ARM) consisting of gene-editing therapy developers today sided with the emerging scientific consensus against germline gene editing in human clinical research without coming to terms with legal, ethical, and other implications—part of a statement intended to articulate a bioethical framework for use of the technology in therapeutic applications.
Using a modular CRISPR base editing system called DOMINO, scientists at MIT have engineered living cells to be computing and recording devices. DOMINO can execute cascades of DNA writing events in response to biological signals, enabling the deep interrogation of biology and allowing engineered cells to process, monitor, and store information occurring within cells and/or their environments.
Pfizer will expand its manufacturing of gene therapies in North Carolina, where the pharma giant joined Gov. Roy Cooper in announcing plans for a $500 million facility expansion in Sanford that is expected to add 300 jobs to its local workforce of 650.
A synthetic genetic circuit for segregating plasmid DNA and controlling asymmetric cell division has been engineered into bacteria. This circuit has also been used to couple cell division and motility, enabling the physical separation of genetically distinct cells. This stem-cell-like functionality could contribute to the engineering of multicellular systems from prokaryotic hosts.
SillaJen and collaboration partner Transgene today separately acknowledged the failure of their lead product Pexa-Vec (pexastimogene devacirepvec) in a Phase III trial assessing a combination of the oncolytic virus and the Bayer/Amgen cancer drug Nexavar® (sorafenib) in liver cancer
Dana Carroll, a pioneer of zinc finger nucleases, has become a sage of the genome editing field. He is keen to facilitate realistic discussions about genome editing’s therapeutic potential.
Custom-engineered biological systems are slipping the surly bonds of natural biological pathways, exploring new regions of genomic space, and expanding molecular diversity.
According to Inscripta, CRISPR technology and machine learning can supercharge the iterative development of biological systems.
AAV-mediated transgene expression has been underestimated by historical methods, assert scientists based at Children’s Hospital of Philadelphia. They introduce an approach that they say better reveals the true extent of AAV-mediated gene transfer, which will be crucial for genome editing applications of AAV and future studies of AAV vector biology.
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