Alleged manipulated and potentially inaccurate clinical data in the BLA for the $2.1m Zolgensma spark possibility for criminal penalties against AveXis.
The summer months are traditionally slow on movements across the industry, but July saw a number of manufacturing partnership deals take place as companies look ahead to product commercialization.
AveXis and BIA Separations announce an agreement for the commercial purification process of SMA gene therapy drug Zolgensma and further gene therapy candidates.
Catalent Biologics works with Novartis to develop and manufacture its SMA gene therapy treatment and provide manufacturing capacity, as the treatment enters the market.
BMS’ capture of Celgene caught much attention, due to the size and scale of the acqusition, but rival companies have taken the chance to snap up employees that have left after the deal.
Bluebird has received EU conditional marketing approval for patients with transfusion-dependent β-thalassemia, becoming the first treatment from its pipeline to receive approval.
Novartis receives US FDA approval for its gene therapy, Zolgensma, which is a treatment for spinal muscular atrophy, and prices the drug at $2.1m per patient.
This month’s round-up of the most popular stories of the month, featuring a bold prediction of future growth for Chinese biotech and bad news for a potential treatment for Alzheimer’s disease.
In a step towards the commercialisation of its ‘off-the-shelf’ CAR-T therapies, Cellectis begins construction of a facility in France and enters lease agreement for US facility.
Regenerative medicine raised $13.3bn in funds during 2018 but investors question whether treatments can be manufactured consistently, according to AMR annual report.
Biogen agrees Nightstar acquisition for $800m to take on its pipeline of AAV treatments for retinal disorders, diversifyingy its pipeline in the rare disease market.
The market for cell therapy products is set to expand based on increasing investment from the industry and the implementation of advanced manufacturing technologies.
Celltrion Healthcare’s head of medical and marketing outlines how the US biosimilar market is evolving and how manufacturing efficiency could become a key distinguisher from competitors.
In its full-year financials, Novartis announced plans to transform its cell therapy manufacturing process through greater investment and utilisation of digital technologies.
In addition to the $55m previously invested, Novartis will spend a further $60m in expanding AveXis’ site in North Carolina enabling the manufacture of multiple gene therapies simultaneously.
NICE recommends CAR T-cell treatment Kymirah to be covered by the Cancer Drugs Fund to treat adult patients with lymphoma, despite previous negative appraisals.
The contract market for bringing biologics through the pipeline to commercialisation is growing at a rapid clip, with a recent report suggesting a high CAGR in the sector.
2018 saw an increase in the number of biosimilar approvals, and the expiry of patents on major products that allowed the entry of biosimilars across Europe.
With nearly 300 cell and gene therapies in development, targeting more than 100 diseases, 2019 could be set to be a big year for cell and gene therapies.
Securing the clinical supply chain comes down to the notion of ‘compliance’, which is transitioning from a ‘nice to have’ to a ‘need to have,’ says Cryoport.
After the business was asked by the US FDA to provide more data to secure an approval for its biosimilar, Sandoz decided not to pursue its regulatory filing any further.
CBMG announced a manufacturing collaboration agreement with Novartis at the end of last month to produce CAR-T treatment Kymriah for the Chinese market and the company has further ambitions in the pipeline.
Novartis’ experimental marketing strategy, whereby the pharma giant used its scale to insure against failed CAR-T therapies, helped defend Kymriah from competition, says consultant.
Major pharmaceutical companies and CMOs in the US have announced emergency contingency plans, including facility closures and the relocation of drug products, as Hurricane Florence approaches southeastern shores.
The US Food and Drug Administration has rejected Regeneron’s eye candidate, Eylea, but the biotech says it expects a final regulatory decision within two months.
The US FDA has released draft guidance documents on gene therapy programmes, which “should help sponsors avoid expending time and resources on unproductive drug development efforts,” says expert.
The French allogeneic T-cell developer says CRISPR/Cas9 technology is an “easy-to-use and cheap tool” for gene editing and plans to license the patents to others.
