Search Results for: zOLGENSMA

Ionis pledges to up its RNA game in SMA and elsewhere

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Self-proclaimed pioneer of RNA-targeted medicine Ionis Pharmaceuticals says it is looking to develop an “even better medicine†to Spinraza to treat spinal muscular atrophy (SMA). At its annual meeting of stockholders earlier this month, Ionis Pharmaceuticals spoke about the success of its flagship molecule, Spinraza (nusinersen), an oligonucleotide approved by the US Food and Drug Administration (FDA) in December 2016 for the treatment of SMA. “Spinraza is the standard-of-care for all forms of the disease SMA. It is a medicine…

June 12, 2019

PE to boost Vibalogics’ viral vector capabilities

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Private equity firm Ampersand Capital Partners has bought CDMO Vibalogics and intends to expand its capabilities to service the demand for gene therapy services. The price of the German contract development and manufacturing organization (CDMO) has not been disclosed, but Ampersand’s acquisition will help widen Vibalogics’ global reach on the back of increased demand for viral vector and gene therapy services. “Vibalogics can now strengthen and expand the position in both of its home markets, the USA and Europe, as…

June 2, 2019

Partnerships abet vendors in tackling cell & gene logistics and automation

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With Thermo Fisher teaming with automated tech company Scinogy and GE Healthcare collaborating with logistics firm World Courier, both vendors have increased their advanced therapy services. The recent approval of Novartis’ one-off gene therapy Zolgensma (onasemnogene abeparvovec) has cemented the advent of advanced therapies. With hundreds more cell and gene therapies in development, the US FDA has predicted that up to 20 such products will be approved each year by 2025. Vendors, therefore, have been increasing their commercial services technologies…

June 2, 2019

Personalized medicines need personalized pricing plans, Orchard

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There is no one-size-fits-all model for cell and gene therapy pricing plans says Orchard Therapeutics, but industry must adapt a system set up for chronic care to incorporate curative one-off treatments. There have been questions over how payors and insurance firms would cope with such next-generation therapeutics have been asked ever since Novartis launched its first cell and gene therapy product Kymriah (tisagenlecleucel) in 2017 at a list price of $475,000. But the recent US Food and Drug Administration (FDA)…

May 29, 2019

Cost of Goods Is Crucial for the Future of Regenerative Medicine: CAR-T Cell Therapy Provides a Case Study in Perspective

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In the history of regenerative medicine, 2017 was a critical year. With approvals for Kymriah (tisagenlecleucel) from Novartis AG, Yescarta (axicabtagene ciloleucel) from Kite Pharma (a Gilead company), and Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics, cell and gene therapies finally made their mark on the regulatory landscape. Then in 2018, those products began both treating patients and bringing in revenues for their sponsor companies. “Patients are being treated, and biotechnology and pharmaceutical companies are being paid for treating them,†said…

April 18, 2019

Novartis grabs AstraZeneca’s CO plant and staff for AveXis gene therapy

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Novartis has acquired a biomanufacturing facility – earmarked for closure by AstraZeneca – ahead of the potential launch of AveXis’ gene therapy for spinal muscular atrophy, Zolgensma. Since Novartis bought AveXis for $8.7 billion (€7 billion) in May 2018, the Swiss pharma giant has rapidly grown its gene therapy manufacturing network. The latest action sees the firm add the 700,000 square-foot Longmont facility to its AveXis operations in what it says is preparation for the launch of Zolgensma (onasemnogene abeparvovec-xioi1),…

April 2, 2019

AveXis on gene therapies: ‘We’ll get better at this and it will get cheaper’

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AveXis has spent about $1 billion in preparing to launch its spinal muscular atrophy (SMA) gene therapy later this year. The Novartis subsidiary is pumping a further $60 million into its North Carolina facility. In May 2018, Gene therapy firm AveXis announced a $55 million (€49 million) investment to establish a manufacturing center in Durham County, North Carolina. Nine months on, and AveXis is doubling down on the site, revealing plans to invest a further $60 million and create 200…

February 19, 2019

Big Pharma and CAR-T: Testing the water or in at the deep end?

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Following Novartis and Gilead’s success, others are “scrambling to build up their T-Cell capabilities,†say venture capitalists. We look at where Big Pharma stands in this blossoming sector. Big Pharma firms will be looking at strategies to enter the chimeric antigen receptor (CAR) T-cell space, if they have not already, spurred on by the approvals in 2017 of Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) . This was the view of commentators and analysts at Phacilitate in Miami, Florida last month. Anna…

February 13, 2019

Beyond approvals: Top 10 cell and gene therapy milestones

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M&A, global partnerships and product approvals have all propelled regenerative medicines into the public discourse. We present the breakthroughs of 2018 and look at where the sector will head in 2019. The “new frontier of medicine†arrived in the US in 2017 through the approval of chimeric antigen receptor (CAR) T-Cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), and gene therapy Luxturna (voretigene neparvovec). But it was last year, 2018, which delivered the true potential of this burgeoning sector into…

February 6, 2019

Finding closure: Lowering the costs of cell and gene therapies

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Fully closed and automated platforms are key to reducing the current high costs of cell and gene therapy manufacturing, say industry experts. Cell and gene therapies (CGTs) have come a long way over the past two years, resulting in two chimeric antigen receptor (CAR) T-cell therapies – Kymriah and Yescarta – and the novel gene therapy Luxturna reaching commercialization. But now these therapies have proved themselves, the major talking point among delegates at the Phacilitate conference in Miami, Florida last…

January 30, 2019