Therapeutic Class

Sarepta plans gene editing center; files DMD therapy for review

Genetic disease-focused firms need infrastructure says Sarepta, which shared plans for a gene editing center of excellence at the JP Morgan healthcare conference. The gene editing center will be in Durham, North Carolina according to CEO Douglas Ingram, who said investing in capacity and knowhow is key to Sarepta’s longevity. “If one is going to be an enduring genetic medicine company, we are going to need infrastructure,” he said, citing partnerships as an important part of the expansion. “We’re deepening…

Horizon says base editing tech could make cell therapies safer

Horizon Discovery Group says a new DNA editing tech could move multi-gene knockout cell therapies through trials with improved safety profiles. The technology in question – called the Pin Point system – is an editing platform that allows developers to precisely modify DNA at the base-level. The approach differs from CRISPR/Cas9 and other conventional editing techniques that create cuts in genes, which could result in unintended changes and lead to deleterious effects in patients Horizon says. The firm will offer…

Tessa and CellVec both set up Singapore plants to support cell & gene therapies

Tessa Therapeutics will open a plant in Singapore for its CAR-T programs, while CellVec has opened a viral vector facility on the island state to support customers’ gene therapy projects. Singapore-based biotech Tessa is developing its own autologous cellular therapies and has told us it plans to open a 90,000 square-foot plant to support clinical and commercial efforts. “Tessa will use the new facility for the clinical and future commercial manufacturing of our late-stage clinical programs,” the firm said, adding…

Novartis inks 5 years vector supply extension with Oxford Biomedica

Oxford Biomedica will keep supplying Novartis with the vectors needed to make Kymriah and other CAR-T products under a five-year deal extension. The new agreement will see Oxford Biomedica receive $75 million for manufacturing, undisclosed process development payments, a facility reservation fee as well as previously agreed royalties on chimeric antigen receptor (CAR-T) product sales. The extension deal also requires the firm ensure at least two manufacturing facilities are capable of commercial supply. Oxford Biomedica will make the vectors at…

Back to school: GE and UMass partner on academic viral vector plant

GE Healthcare has partnered with UMass to establish a viral vector manufacturing facility – its latest tie-in with academia in the cell and gene therapy space. The planned facility at the University of Massachusetts (UMass) Medical School’s Worcester campus will provide recombinant adeno-associated virus (AAV) vectors for preclinical research. High demand and a shortage of capabilities for viral vectors means the 3,220 square-foot facility, decked out with GE Healthcare’s viral vector FlexFactory platform, will help alleviate the current wait researchers…

Charles River bids $380m to buy cell therapy services firm HemaCare

HemaCare will boost Charles River Laboratories’ offering in the cell therapy materials and leukapheresis space – a market predicted to grow to $2 billion in the next decade. The deal will add expand Charles River’s cell therapy offering. HemaCare provides human primary cells as well as services used in the discovery, development and commercial production of cell therapies. Leukapheresis – the separation of white cells from blood samples – is a core HemaCoare offering. The firm provides leukapheresis services for…

AI will help Novartis make personalized meds, says Amazon

Amazon says Novartis’ personalized medicines operations will benefit from AI and machine learning collaboration. Novartis confirmed it had asked Amazon Web Services (AWS) to rejig is supply lines last week. The drug firm said it “will leverage AWS’s broadest portfolio of cloud services to build an enterprise-wide data and analytics platform that will transform business operations. According to AWS, the plan is to use artificial intelligence and machine learning to “reimagine” the Swiss firm’s manufacturing, supply chain, and delivery operations.…

Polyplus ups reagent range to support growing CGT space

Polyplus-transfection SA has expanded its reagent portfolio, citing the growing number of candidate cell and gene therapies nearing commercialization as the driver. The French biotech launched a range of sizes of its PEIpro-GMP reagent in single-use bags and bottles this week. Transfection reagents facilitate cellular uptake of nucleic acids. Polyplus premiered PEIpro-GMP last year, marketing it as “the first transfection solution for the gene and cell therapy industry that is compliant with global cGMP viral vector manufacturing requirements.” The expanded…

Swiss site opens to boost Novartis CAR-T production

Novartis has opened a CHF 90 million ($91 million) manufacturing site in Stein, Switzerland to support its cell and gene portfolio. In August 2018, Novartis announced a Swiss facility would support production for its burgeoning chimeric antigen receptor T cell (CAR-T) products and pledged CHF 90 million in phased investments to support the project. The facility in Stein, about 20km east of Basel, opened its door last week ahead of the predicted 2020 date, bolstering the firm’s global network. “The…

Boston next-gen manufacturing hub will ease bottlenecks, consortium

Cell and viral vector backlogs reduce patient access to next-generation medicines and slow R&D says the consortium behind a $50 million innovation hub. The group, led by Harvard University, made the comments this week, explaining that while some contract development and manufacturing organizations (CDMOs) offer cell line and vector services, growing demand means developers can face delays. “Manufacturing backlogs are slowing the production of cells that are essential to research, holding up the availability of new treatments headed for the…