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Pharma Horizons: the future of cell and gene therapy
EPR's latest Pharma Horizons report explores the latest advances in cell and gene therapy for quality control, manufacturing, analytical development and more.
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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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USP boosts support for AAV-based gene therapy manufacturers
Alongside publication of the proposed General Chapter < 1067 >, the new resources from USP aim to help advance AAV gene therapies.
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Advancing endotoxin testing: implementing sustainable recombinant LAL solutions
7 March 2025 | By Fujifilm Wako
Discover the benefits of sustainable recombinant LAL for endotoxin testing. Learn about its relevance, selection considerations, and seamless integration into your lab processes with expert insights.
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CHMP meeting highlights – February 2025
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of four new medicines plus the extension of therapeutic indications for 16 medicines.
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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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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.
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Cell and Gene Therapy Containment
Overcoming Challenges in Cell and Gene Therapy Containment.
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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.
