Search Results for: zOLGENSMA

Novartis prepped for ‘unprecedented’ Zolgensma demand

by

With over one million square-feet of manufacturing space, Novartis says it is prepared for the imminent approval of AveXis’ SMA gene therapy Zolgensma. Speaking during its Q1 2019 results, Novartis said it is set for the imminent arrival of gene therapy Zolgensma (onasemnogene abeparvovec), added to the firm’s pipeline through the acquisition of AveXis. The one-time therapy targeting spinal muscular atrophy (SMA) Type 1, will be reviewed by the US Food and Drug Administration in May, despite the recent news…

April 25, 2019

Hurdles Ahead for Cell and Gene Therapy Makers

by and

Significant growth of the cell and gene therapy (CGT) pipeline in recent years demonstrates the enormous potential of these modalities to treat or even cure otherwise intractable diseases. Several CGT products have been approved for clinical use over the past five years. More than 75 such products have come to the market around the world so far. They include chimeric antigen receptor (CAR) T-cell therapies that involve genetic engineering of patient cells ex vivo as well as in vivo gene therapies…

April 19, 2022

Scaling AAV Production: Easing the Transition from Laboratory Scales to Commercial Manufacturing

by

Adenoassociated virus (AAV) has emerged as the leading vector for gene therapy delivery. Compared with options such as lentivirus and adenovirus, AAV exhibits a strong safety profile because it has low pathogenicity and requires a helper virus to replicate. AAV is also capable of long-term gene expression, and it can infect both dividing and nondividing cells (1–5). Developers of advanced therapies have found such advantages to be quite attractive. As of January 2021, two gene therapy products have gained US…

March 16, 2022

2021 CGT moments: First place for second line CAR-T

by

Advancements in CAR-T development and access, in vivo gene editing, and CDMO M&A all feature in Propel BioSciences’ Susan Nichols top 10 advanced therapy moments of 2021.  After two years away, BioProcess Insider attended the highly anticipated presentation at Phacilitate’s Advanced Therapies Week in Miami, Florida, where Susan Nichols (CEO of Propel BioSciences) highlighted 10 moments from 2021 that defined and drove the cell and gene therapy (CGT) space.  Some unexpected milestones made the list, such as the arrival of messenger…

January 31, 2022

Novartis looks to gene therapy 3.0 with an eye on lowering COGS

by

Novartis is confident the manufacturing costs of gene therapies will fall as it improves processes and brings on board next generation technologies such as CRISPR and gene editing. Swiss Pharma giant Novartis has “always been a company that is multi-modality in really trying to address an unmet need in a number of diseases,†Shephard Mpofu,  chief medical officer at Novartis Gene Therapies said yesterday. Highlighting the firm’s success in small and large molecules, he reminded delegates at Phacilitate’s Advanced Therapies…

January 26, 2022

CBM and UPenn enter gene therapy manufacturing pact

by

A five-year deal will see the Center for Breakthrough Medicines (CBM) gain commercial rights to certain gene therapy manufacturing and analytics platforms from the University of Pennsylvania. The King of Prussia, Pennsylvania-based contract development and manufacturing organization (CDMO) has entered a collaboration with the University of Pennsylvania Gene Therapy Program (GTP) to help small biotech firms, universities, and large pharma firms advance gene therapies through the clinic. The collaboration brings CBM access to UPenn’s gene therapy expertise, which has helped…

January 20, 2022

Optimizing the AAV Transfection Process in Suspension Cells

by and

Given the potentially curative nature of gene and gene-modified cell therapies and the successful launches of several such products over the past five years, markets and investments are growing significantly in the sector. In the United States alone, the US Food and Drug Administration has granted approval for several genetic therapies, including Strimvelis (autologous CD34+, developed by GlaxoSmithKline and later sold to Orchard Therapeutics) in 2016 Luxturna (voretigene neparvovec, Spark Therapeutics), Yescarta (axicabtagene ciloleucel, Kite Pharma/Gilead), and Kymriah (tisagenlecleucel, Novartis)…

October 29, 2021

AGC Bio takes the Longmont jump, buying CO plant from Novartis

by

CDMO AGC Bio has picked up a second site in Colorado, buying a cell and gene therapy facility in Longmont from Novartis. The 622,000 square-foot facility in Longmont – north of Denver, Colorado – will become part of AGC Biologics’ ever-growing manufacturing footprint, and will go hand-in-hand with its cell and gene therapy site in Milan, Italy, added through the €240 million ($284 million) acquisition of Molmed last year. “The Longmont facility fits perfectly into AGC’s long-term growth and expansion…

July 2, 2021

Overcoming Obstacles in AAV Viral Vector Manufacturing

by

Rapidly growing interest in gene therapy has led to the need for more cost-effective and scalable viral-vector manufacturing platforms. Adenoassociated virus (AAV) has become a vector of choice because of its safety profile (nonpathogenic infection). In addition, AAV cannot replicate on its own and is not integrated directly into the host genome. AAV vector manufacturing using human embryonic kidney (HEK) cells in either adherent or suspension mode includes several typical processing steps: cell expansion, plasmid transfection, viral-vector production, cell lysis,…

June 18, 2021

Novartis exiting Longmont due to changing gene therapy capacity need

by

Novartis will close its Longmont, Colorado biomanufacturing after over anticipating the amount of capacity needed to produce Zolgensma and other gene therapies. When Novartis acquired AveXis in May 2018, it quickly began expanding its manufacturing network to support the then lead candidate Zolgensma (onasemnogene abeparvovec-xioi1). By the time the gene therapy won US approval as a treatment for spinal muscular atrophy (SMA) in May the following year, Novartis was confident it had built up a sufficient production footprint for launch…

March 26, 2021