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Preparing for a new era in the rare disease sector
To mark Rare Disease Day 2025, EPR interviewed Christina Gkousgkouni, Head of Rare Diseases for Central South Europe, Sanofi.
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Gene therapy
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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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Innovating bioanalysis to advance oligonucleotide therapeutics
Here, Dr Jinpeng Li at WuXi AppTec reveals the analytical challenges that are hindering advancement of oligonucleotide therapeutics, medicines which have demonstrated clinical promise in the gene therapy space, and discusses promising analytical innovations.
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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.
whitepaper
Cell and Gene Therapy Containment
Overcoming Challenges in Cell and Gene Therapy Containment.
webinar
When innovative formulations cloud sterility testing
30 October 2024 | By Charles River
Webinar presented by Miriam Guest, Senior Principal Scientific Advisor at Charles River, to explore how ATP-bioluminescence outperforms traditional sterility testing in complex matrices.
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Quality from the start: designing a scalable AAV production platform
This Feature Partnership discusses AAVs and how manufacturers can overcome the key development challenges and create a scalable AAV production platform.
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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.
webinar
Monocyte activation test (MAT): Insight into examining regulations and markets prospects
9 September 2024 | By FUJIFILM Wako
Watch this webinar to learn about the current regulations on the monocyte activation test (MAT) and the new type of MAT entering the markets.
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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.
webinar
An alternative medium to support sterility testing using the Growth Direct® Rapid Sterility System
4 September 2024 | By Rapid Micro Biosystems
Watch on demand to learn how a new single medium, Rapid Sterility Medium (RSM), performs as well as or better for the growth promotion of test microorganisms compared to compendial sterility test media.
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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.
