Search Results for: zOLGENSMA

Swiss site opens to boost Novartis CAR-T production

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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…

December 4, 2019

CAR-T at a crossroad as industry looks to allogeneic

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As cell therapies move through the clinic towards commercialization, respondents to a KNect365 industry survey are beginning to look to allogeneic – or off-the-shelf – products as the next big thing. Almost 200 people contributed to the Cell Therapy Analytics Report from KNect365, revealing their current position within the burgeoning cell and gene therapy space and offering up their thoughts and predictions for the future. The majority worked within companies developing oncology products, and the largest group – 41% –…

October 18, 2019

Vector market moving in right direction: Shortage driving CDMO M&A

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Demand for viral vectors has emerged as a major M&A driver in the gene therapy CDMO space says an industry expert. Viral vectors are hollow viruses developers can fill with genetic material. When one of these vectors ‘infects’ a cell the genes inside are inserted and expressed as proteins. Vectors are vital for the production of gene therapies, including new products like Spark Therapeutics’ Luxturna (voretigene neparvovec-rzy) and AveXis’ Zolgensma (onasemnogene abeparvovec-xioi). The problem is that, for the past few…

October 1, 2019

In the pipeline: Surge of cell and gene therapies likely in 2020

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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 number of investigational new drug (IND) applications for cell and gene therapies it is receiving. “The FDA commissioner has anticipated that he thinks there will be between 10-20…

September 30, 2019

Pfizer pumps $500m into NC gene therapy site

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The expansion in North Carolina will create 300 jobs and is the latest investment by Pfizer in its burgeoning gene therapy network. The $500 million (€450 million) investment in Sanford, North Carolina will see the Big Pharma firm construct a facility to support production of its recombinant adeno-associated virus (rAAV) vectors for use in its gene therapies and vaccines. Mike McDermott, president of Pfizer Global Supply, said the Sanford expansion will “strengthen Pfizer’s leadership in gene therapy manufacturing technology†and…

August 22, 2019

Pfizer licenses AAV platform for ataxia gene therapy

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Pfizer will use REGENXBIO’s adeno-associated virus (AAV) delivery platform for a gene therapy aimed at treating neurodegenerative disease Friedreich’s ataxia. In REGENXBIO’s second quarter 2019, a major milestone was reached with the approval of Novartis/Avexis’ Zolgensma (onasemnogene abeparvovec). The gene therapy uses the NAV Technology Platform, created and validated by REGENXBIO’s founders and collaborators at the University of Pennsylvania, which uses over 100 adeno-associated viral (AAV) vectors to deliver engineered genes to cells. The firm, which is also developing its…

August 9, 2019

COGS crisis: Cell therapy sector must rethink CMC says expert

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The cell therapy sector faces a cost of goods sold (COGS) crisis and needs to get beyond the “product is the process†mind-set says Anthony Davies, CEO of Dark Horse Consulting. Cell and gene therapies have attracted a lot of attention in recent years. In part this is because drugs like Yescarta, Kymriah and Zolgensma have been approved as one-off cures. However, their cost – $373,000, $475,000 and $2.1m per vial in the US, respectively – has also seen them…

August 6, 2019

BIA Separations providing chromatography columns for AveXis

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BIA Separations’ prepacked chromatography columns “contribute to a robust manufacturing process†for the recently approved gene therapy Zolgensma, says AveXis. In May, the US Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovec), a gene therapy designed to halt progressive spinal muscular atrophy (SMA). Novartis subsidiary AveXis developed and commercialized the $2.1 million one-off treatment, but – as the saying goes – it takes a village to raise a child. Zolgensma relies on Regenxbio’s adeno-associated virus (AAV) vector technology, and…

July 24, 2019

‘Bioprocessing ecosystem is on fire,’ says analyst

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Sales grew 21% year-on-year at Sartorius’ H1 2019, while Repligen reported preliminary Q2 growth of nearly 50%. The bioprocessing sector ‘is on fire,’ says Jefferies’ analyst Brandon Couillard. Repligen Corporation published its preliminary Q2 results in an SEC filing last week, saying it expects sales of $70-71 million, compared to $47.7 million for the three months ended June 30, 2018. This would represent a 48% increase year-on-year. “The 8-K did not offer much color on specific drivers, but momentum across…

July 23, 2019

AveXis: ‘Speedy scale-up driven by Novartis acquisition’

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With its own facilities, support from Novartis and now dedicated space at Catalent’s Paragon, AveXis says it has the most gene therapy manufacturing capacity in the industry. AveXis has inked a deal with contract development and manufacturing organization (CDMO) Paragon Gene Therapy to secure dedicated manufacturing space for its one-off spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec), approved in the US in May. Paragon’s facility in Maryland will also provide process development for clinical supply of other gene therapy…

July 18, 2019