To ensure the UK’s prominence in the cell and gene therapy arena, the CGT Catapult has published a roadmap for the research and adoption of these cutting-edge therapies.
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The US FDA has published two draft guidance documents, one on gene therapy products and the other CAR T-cell therapies, for developers and manufacturers.
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
The one-time gene therapy treatment for rare, neurodegenerative disease metachromatic leukodystrophy (MLD) is the most expensive drug ever evaluated by NICE.
Here we run down four new research collaborations in the cell and gene therapy sector, announced by Bayer, BMS, Pfizer and Moderna.
On 30 November and 1 December 2021, European Pharmaceutical Review hosted its first ever event – The Future of QA/QC for Complex Biologics Online Summit. Bringing together more than 600 attendees, 15 expert speakers and Thermo Fisher Scientific and Merck as sponsors, the summit gave industry experts the chance to…
BMS’s Orencia (abatacept) has been approved by the US FDA for prophylaxis of acute graft-versus-host disease (aGvHD) in patients aged two years plus.
Many hands make light work: why the new Bespoke Gene Therapy Consortium is a godsend for ambitious biopharma teams
In late October, we learned that an exciting and ambitious new entity – the Bespoke Gene Therapy Consortium (BGTC) – had come into being in the US, designed to encourage the delivery of more gene therapies for rare diseases. The consortium is an interesting construct, headed and funded by the…
Giroctocogene fitelparvovec had a mean annualised bleeding rate (ABR) of just 1.4 percent over two years, with no bleeding events in the first year post infusion.
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
Features in this in-depth focus highlight some of the important developments in the cell and gene therapy market, as well as the key manufacturing strategies to consider when taking them from concept to commercialisation.
A newly developed investigational microneedle patch caused mice to regrow hair faster than minoxidil, a leading alopecia treatment.
The Parkinson’s disease market is set to triple in sales by 2029 driven by novel biologics and delivery systems, reports GlobalData.
Ritlecitinib was shown to reduce scalp hair loss to less than or equal to 20 percent after six months in Pfizer’s Phase IIb/III trial.
AstraZeneca’s Saphnelo (anifrolumab-fnia) has been approved in the US for patients with moderate to severe systemic lupus erythematosus.