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Researchers have developed a novel gene therapy that could help treat children with incurable genetic and neurodegenerative diseases.
The Pharmacovigilance Risk Assessment Committee (PRAC) provided safety warnings on COVID-19 vaccines, and found no evidence linking Zynteglo to blood cancer.
To promote the safe and ethical use of genome editing for the treatment of disease and genetic disorders WHO has published the first global recommendations.
The journey from large molecule to approved biotherapeutic is long, costly, complex and fraught with risk – yet rewards for success are substantial.
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
The betibeglogene autotemcel (beti-cel) gene therapy caused paediatric and adult beta thalassaemia patients to achieve transfusion independence lasting over two years.
The Cell and Gene Therapy Catapult’s new consortium aims to accelerate therapeutic development by developing process analytical technologies (PAT) for cell and gene therapy manufacturing.
A rapid sterility test method that can be validated as quickly and confidently as it provides test results.
This webinar presents the fundamentals of a contamination control strategy including forms of contamination, as well as cleaning and disinfection.
Pictorial that explains how Aramus TM single-layer fluoropolymer bag can withstand gamma irradiation and cold-chain temperatures down to -196°C.
The first-in-human study will evaluate the safety and efficacy AGT103-T, a cell and gene therapy product for HIV, in up to six participants.
27 May 2021 | By Repligen
Watch this on-demand webinar, where we reviewed VPT and how it can be used in various applications, from protein therapeutics to gene therapy.
A new report suggests pharma is working hard to combat worldwide viral vectors shortage that could threaten the COVID-19 vaccine rollout.
In its latest meeting the CHMP recommended eight medications for approval, 17 medicines for indication extension and concluded reviews of two COVID-19 treatments.