Therapeutic Class

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

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…

Yescarta 2.0? How Kite plans to scale out CAR-T therapies

CAR-T pioneer Kite Pharma says scaling out capabilities, embracing digital technologies, and industry collaboration are among the efforts needed to bring cell therapies to maturity. In 2017, Gilead company Kite Pharma became the second company to achieve US regulatory success for a chimeric antigen receptor (CAR) T-cell therapy. Two years on, and while sales of the product Yescarta (axicabtagene ciloleucel) are rising – Q2 2019 saw a 25% quarterly growth to $120 million –the sector itself has somewhat stalled with…

Treefrog wins EU funding for mass-produced cell therapy tech

Treefrog Therapeutics has received €10 million ($11 million) over the past five months to support its mass-produced pluripotent stem cell platform C-Stem. In September, Bordeaux, France-based Treefrog Therapeutics received €450,000 from the French government and €2.4 million from the EU’s Horizon 2020 program to help acelerate the cGMP certification and scale-up of its C-Stem technology platform. It comes five months after the firm raised €7 million in a Series A funding. “C-Stem is a technology that enables the mass-production of…

Zolgensma data: ‘Heavy-handiness’ of FDA a warning to industry, not just Novartis

The US FDA may have made an example of Novartis in the way it handled the revelation of Zolgensma data manipulation, says the Alliance for Regenerative Medicine. A month after the US Food and Drug Administration (FDA) approved the one-time gene therapy Zolgensma (onasemnogene abeparvovec), marketing authorization holder Novartis alerted the agency to a data manipulation issue that impacted the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA). In its response,…

GSK pays Immutep on advancement of Ab for Ulcerative Colitis

The first patients have been dosed in a Phase II trial for GSK2831781, GSK’s ulcerative colitis candidate based on Immutep’s depleting anti-LAG antibody technology. In 2010, GlaxoSmithKline (GSK) licensed ImmuTune-IMP731, a therapeutic antibody for the treatment of autoimmune diseases, from Immutep in a deal that included milestone payments of £64 million ($80 million). This week, GSK is paying out £4 million of this due to the first patient being dosed in GSK’s Phase II clinical trial evaluating the candidate –…

Biocon buys Pfizer R&D plant to bolster 28-strong biosimilar pipeline

Biocon has acquired an R&D plant in Chennai from Pfizer healthcare India and plans to set up the 60,000 square-foot space to support its 28 molecule-strong biosimilar pipeline. Financial details of the acquisition have not been divulged, but a spokesperson from Indian drugmaker Biocon told this publication it has “acquired R&D capital assets from Pfizer healthcare India Ltd.” Specifically, this is a 60,000 square-foot facility at the TICEL Bio Park in Chennai, India, which will be established as a “bench…

Sarepta looks to PD optimization to progress DMD gene therapy

With Brammer and Paragon contracts, Sarepta Therapeutics has ample gene therapy manufacturing capacity but says it is now working on analytical and process development yield optimization. In 2018, Sarepta announced it was adopting a “hybrid manufacturing” approach to its gene therapy pipeline meaning it would build internal expertise in adeno-associated virus (AAV)-based manufacturing while partnering with contract manufacturers. As such, the firm entered into a long-term partnership with Brammer Bio – now part of Thermo Fisher – and Paragon Bioservices…

Pfizer adds $19m plant in NC clinical gene therapy shake-up

Pfizer will relocate clinical manufacturing activities from its Chapel Hill site to a newly acquired facility nearby in the latest ramp up of its North Carolina gene therapy capabilities. This week, big biopharma firm Pfizer acquired a piece of land and newly constructed building at 1219 Shiloh Glenn Drive, North Carolina – about 2 miles west of Raleigh-Durham International airport – set to become a center of clinical manufacturing for its gene therapy programs. Pfizer confirmed the news first broken…

GE and Germfree in modular gene therapy manufacturing collaboration

GE Healthcare has teamed with cleanroom production firm Germfree to develop a modular biomanufacturing facility for emerging biotherapeutics and gene therapies. Bioprocess vendor GE Healthcare is no stranger to modular manufacturing platforms, with the likes of Bayer, Pfizer and Lonza all opting for its off-the-shelf KUBio platform modular biologics factory. Furthermore, to date 63 manufacturers have selected the firm’s FlexFactory bioprocess platform, which uses predominantly single-use technology. And now the company has partnered with Florida-based Germfree to co-develop a new…

CGT CMC: Plan ahead, avoid comparability studies says Novartis

Making changes to the manufacturing process for cell and gene therapies is possible but cumbersome due to the need to perform comparability studies, says a Novartis veteran. Speaking at the Cell & Gene Therapy Bioprocessing & Commercialization conference – part of Biotech Week Boston – this week, Novartis’ Sergio Fracchia advised developers of advanced therapies to think about their manufacturing needs several years in advance to avoid future pitfalls. Specifically, Fracchia – who serves as Global regCMC Director, Cell and…