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Early-phase ATMP clinical trials see promising growth
Cell and Gene Therapy Catapult's (CGT Catapult) findings attest to the UK remaining “a highly attractive destination for clinical trials”.
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Gene therapy
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Long-term potential revealed for novel modifier gene therapy
The positive two-year findings from the Phase I/II gene therapy trial in retinitis pigmentosa reiterate the potential of mutation-agnostic therapies.
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Potential blockbuster drugs to watch in 2025
Eleven drugs are poised to make waves in the pharmaceutical industry in 2025, according to the annual report.
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New mass spectrometry Chapter could “revolutionise” biopharma quality assurance
Launch of the USP general method chapter signals a breakthrough in pharmaceutical drug impurity control, according to biopharma experts.
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AAV gene therapy manufacturing gets £1.4m boost
The funding aims to reduce the costs of AAV production for gene therapies, helping to accelerate treatment access for patients with rare diseases.
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European manufacturing facility to strengthen gene therapy supply
The new manufacturing facility in Finland will aid global supply of the first FDA-approved intravesical non-replicating gene therapy for adults with a common cancer.
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Gene therapy trial facilitates major vision improvement
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
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Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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Pfizer receives EU haemophilia B gene therapy approval
The single-dose gene therapy was approved for certain patients with haemophilia B in the US and Canada earlier this year, marketed as BEQVEZ.
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The route to enhanced gene therapies
In this article, Roger Palframan, Head of US Research at UCB, delves into the potential of gene therapy and which modalities have the most promise, what the industry should prioritise to advance the field, as well as what is needed to develop the workforce.
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Modifier gene therapy – clinical development and manufacturing considerations
Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the company’s promising modifier gene therapy candidates for ophthalmic disorders.
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Addressing drug delivery challenges of emerging drug modalities
The new company will work to develop solutions to overcome issues within new therapeutic modalities, in areas such as formulation, manufacturing and supply chain.
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Challenges and opportunities in the CGT supply chain
This article explores the unique logistics and supply chain challenges of cell and gene therapy development and the future potential of this innovative sector.
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“Landmark” genetic blindness CRISPR trial outcomes released
A Phase I/II trial has shown that a CRISPR-based gene editing therapy can be safely delivered to the retina and provide clinically meaningful outcomes.
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Enhancing medicine manufacturing through digitalisation
Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.
