news
EC grants Orphan Drug Designation to FLT190, a Fabry Disease treatment
FLT190 gene therapy has been granted Orphan Drug Designation based on preliminary trial data and the positive opinion of an EMA committee.
List view / Grid view
Gene therapy
news
Almost 400 cell and gene therapies in US pipeline, finds report
New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.
news
European patent upheld for foundational CRISPR intellectual property
Arguments filed in opposition to a patent for foundation CRISPR-Cas9 intellectual property have been rejected by the European Patents Office.
news
CRISPR-edited T cells active in patients months after initial infusion
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.
news
Demand for ‘specialist skills’ in UK cell and gene therapy, says report
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.
news
FDA releases guidance on gene therapy manufacturing
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.
news
UK accounts for 12 percent of global cell and gene therapy clinical trials
New research has found that the cell and gene therapy clinical environment in the UK has encouraged commercial sponsorship from around the world.
news
Nanoparticle gene therapy drug delivery for paediatric brain cancer tested in mice
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.
article
Viral vectors for gene therapy
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…
article
Bioprocessing & Bioproduction In-Depth Focus 2019
Within this in-depth focus are articles exploring the potential of gene therapies and manufacturing viral vectors as well as a discussion on recent M&As within the biopharmaceutical industry.
news
Investment in UK cell and gene therapy manufacturing set to continue
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.
news
UK authorisation granted for first in-human CAR-Treg cell therapy study
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.
news
CAR-T cell therapy granted RMAT for relapsed/refractory multiple myeloma
An investigational CT053 CAR-T cell therapy for RRMM has been given Regenerative Medicine Advanced Therapy designation.
news
New report predicts growth of gene therapies for neurology indications
A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge.
news
Clinical trial shows cell therapy could be safe for liver disease patients
A new cell therapy has been tested in patients with liver cirrhosis and found no significant adverse effects.
