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FDA gives first RMAT designation to a cardiac gene therapy
A regenerative medicine advanced therapy (RMAT) designation has been given to a gene therapy for Danon disease by the US Food and Drug Administration (FDA).
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Video: Understanding current regulatory thinking on contamination control strategy
Virtual seminar presented by Ziva Abraham, President, and CEO of Microrite designed to improve your understanding on current regulatory thinking on designing an effective contamination control strategy.
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New UK point of care manufacturing regulatory framework
A first-of-its-kind regulatory framework for point of care manufacturing of innovative medicines is set to be introduced in the UK, ensuring these products are as safe as conventional medicines.
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EMA validates first regulatory submission for CRISPR-based medicine
The first regulatory submission for a CRISPR-based therapy has been validated by the European Medicines Agency, to treat sickle cell disease and transfusion-dependent beta thalassemia.
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UK ATMP clinical trials report 2022 published
T cells were revealed as the most commonly investigated cell type continuing to increase, found CGT Catapult's 2022 UK ATMP clinical trials report.
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Potential blockbuster drugs to watch in 2023
Lecanemab for Alzheimer's and the first potential gene therapy primed for US launch for haemophilia A are two potential blockbuster Drugs to Watch™, says a 2023 report.
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Moderna to obtain first acquisition for $85 million
Moderna has agreed on its first acquisition for $85 million, enabling it access to cell-free DNA synthesis and amplification technologies.
whitepaper
Whitepaper: Imaweld® heat sealable & weldable tube bioprocess media
Imaweld® is distinct due to its composition, which seals and welds tubes and highly endorsed in biopharma for cell membrane and cell culture applications
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Breakthrough gene therapy gives hope for Artemis-SCID
A new gene therapy developed by UC San Francisco has enabled ten young Artemis-SCID patients to achieve full T-cell immunity.
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Whitepaper: Optimising the efficiency of QC laboratory: The power of automation
Charles River launches Endosafe Nexus 200, emphasises the importance of automated endotoxin testing systems to improve the efficiency of QC labs.
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Whitepaper: Single-use technologies in biosimilar development
ImaLINK™ manifold, which is designed to minimise the need for fittings, connections or assembly of all leak points, entrapment areas are eliminated.
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FDA approves first gene therapy for high-risk early bladder cancer
The first gene therapy for high-risk non-muscle-invasive bladder cancer has been approved by The US Food and Drug Administration (FDA).
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CHMP meeting highlights – December 2022
A total of five drugs were recommended for approval at the CHMP’s December meeting, including a gene therapy and two cancer drugs.
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CHMP adopts positive opinion for haemophilia B gene therapy
Gene therapy etranacogene dezaparvovec has been granted a positive opinion by the CHMP as a haemophilia B treatment for adults in Europe.
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First patient given base-edited CAR T cells
The first ever use of base-edited T-cells have helped to treat a patient with relapsed T-cell acute lymphoblastic leukaemia in a clinical trial.
