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.
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Gene therapy
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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.
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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.
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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.
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Alternative viral delivery of gene therapy developed
New nanocapsules are a promising platform for many types of gene therapies due to their small size and superior stability.
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BPSA releases guide to production of cell and gene therapies
The Bio-Process Systems Alliance, the primary international industry association for single-use bio-processing, is pleased to announce the release of its latest whitepaper...
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Gene therapy receives Champalimaud Vision Award
The Champalimaud Vision Award has been awarded to research teams for the development of a gene therapy...
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Regulatory, clinical and logistics challenges of Advanced Therapy Medicinal Products (ATMPs) in clinical research
Advanced Therapy Medicinal Products (ATMPs) are new therapeutics that require different procedures for clinical trials compared to traditional medicinal products. It is important to understand the nature and the manufacturing process of an ATMP for set-up and execution of the clinical development and later marketing of these therapies.
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FDA approves CAR T cell therapy for children with leukaemia
The FDA has approved CAR T cell therapy for treatment of children with acute lymphoblastic leukemia...
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Personalised medicine, batch size of ONE, the new challenge to fill-finish?
Wenzel Novak PhD gives some considerations on container, environment, process and automation for small batch sizes in the downstream process of fill-finish…
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Collaboration is ‘game changer’ in delivering cell and gene therapies
Collaboration between the pharmaceutical industry, patients and health regulators will be a ‘game changer’ in delivering exciting new cell and gene therapies to treat conditions like cancer and vision loss.
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Abeona Therapeutics receives FDA orphan designation for gene therapy
26 May 2017 | By Niamh Marriott, Junior Editor
The FDA has granted Orphan Drug Designation for Abeona Therapeutics’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa...
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G is for Gene
22 November 2016 | By Niamh Louise Marriott, Digital Content Producer
G is for Gene - the next instalment of our daily pharmaceutical alphabet bringing you up to date with all the latest gene research...
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Sanfilippo type A Gene therapy trial shows biopotency signals, provides Abeona Therapeutics
2 August 2016 | By Abeona Therapeutics Inc
Abeona Therapeutics provides an update on its first Sanfilippo patient and reports that early biopotency signals suggest a reduction of heparan sulfate in the urine of patients and if confirmed will suggest that intravenous administration of ABO-102 may ultimately provide both central nervous system and systemic benefits to patients...
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Stem cell gene therapy for Sanfilippo disease ready for human trial
11 May 2016 | By Victoria White, Digital Content Producer
University of Manchester and CMFT researchers are to work with Orchard Therapeutics to test a stem cell gene therapy for Sanfilippo disease in a human trial...
