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Duchenne gene therapy interim trial outcomes “striking”
The gene therapy demonstrated consistent evidence of positively changing disease trajectory in Duchenne muscular dystrophy, new interim data shows.
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Gene therapy
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FDA approves first-of-a-kind gene therapy for dystrophic epidermolysis bullosa
With clinical data showing the gene therapy can address large and challenging chronic wounds, the FDA’s decision will help improve quality of life for dystrophic epidermolysis bullosa (DEB) patients.
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First-in-human data for gene therapy signals potential in childhood blindness
Developed and manufactured under a Manufacturer’s ‘Specials’ Licence (MSL), the gene therapy could provide a novel treatment option for paediatric patients at the earliest stage of the rare eye disease.
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NICE recommends gene therapy for severe sickle cell disease
The committee’s approval of the one-time gene therapy means the healthcare inequalities for patients with this genetic blood disorder can begin to be re-addressed.
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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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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.
