According to researchers, Zolgensma, made by Novartis, will lead the global spinal muscular atrophy market and generate billions in sales.
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FLT190 gene therapy has been granted Orphan Drug Designation based on preliminary trial data and the positive opinion of an EMA committee.
New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.
Arguments filed in opposition to a patent for foundation CRISPR-Cas9 intellectual property have been rejected by the European Patents Office.
The first US in-human trial of CRISPR-Cas9-edited T cells has concluded with no ill effects and showed that nine months later the infused cells were still active against cancer.
A skills demand report has stated that employment in the UK cell and gene therapy industry is expected to double by 2024, but must be supported by targeted academic courses.
In an effort to support innovation in the development of gene therapy products, the FDA has released guidance to provide regulatory clarity for product developers.
New research has found that the cell and gene therapy clinical environment in the UK has encouraged commercial sponsorship from around the world.
A study has shown that PBAE nanoparticles could replace viral vectors in gene therapies and provide a safe treatment option for paediatric brain cancer patients.
The first gene therapy products are appearing commercially and the field is growing rapidly with over 350 clinical trials taking place in H1 of 2019 alone. So why is this area attracting so much attention? Dr Philip Probert and Dr Stuart Jamieson discuss the potential of gene therapy and the…
An additional 6,000m2 of cell and gene therapy manufacturing space is expected to become available within the next 12 months, according to new data.
Authorisation has been granted in the UK for the STEADFAST clinical study of TX200, which is the first-in-human trial to evaluate a CAR-Treg cell therapy.
An investigational CT053 CAR-T cell therapy for RRMM has been given Regenerative Medicine Advanced Therapy designation.
A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge.