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Gene therapy

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.

article

Regulating therapies for rare diseases – recent approvals

8 August 2024 | By Catherine Eckford (European Pharmaceutical Review)

This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.

news

Pfizer receives EU haemophilia B gene therapy approval

26 July 2024 | By Catherine Eckford (European Pharmaceutical Review)

The single-dose gene therapy was approved for certain patients with haemophilia B in the US and Canada earlier this year, marketed as BEQVEZ.

article

The route to enhanced gene therapies

25 July 2024 | By Catherine Eckford (European Pharmaceutical Review)

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.

whitepaper

Application note: Automated nucleic acid amplification assay for mycoplasma detection in cell and gene therapy products

28 June 2024 | By bioMérieux

Ambili Menon, Pharma Quality Control, bioMérieux Inc. addresses the task of microbiological testing for advanced therapeutic medicinal products.

article

Drug approval roundup – May/June 2024

27 June 2024 | By Catherine Eckford (European Pharmaceutical Review)

This article highlights some of the key regulatory approvals granted in May and June in the EU and US so far this year, including for oligonucleotide and biologic therapies.

whitepaper

One HEK of a viral vector to develop

25 June 2024 | By Single Use Support

Viral vector manufacturing presents many challenges related to HEK cell line development, fluid transfer and product viability in the cold chain.

article

Modifier gene therapy – clinical development and manufacturing considerations

13 June 2024 | By Arun Upadhyay (Ocugen)

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.

article

Fresh or frozen? Navigating the cryopreservation dilemma for CGT

6 June 2024 | By Priya Baraniak (OrganaBio)

Cell and gene therapy development grapples with a paradox: fresh starting material is often equated with higher-quality drug products, but cryopreservation is crucial for scale-up. In fact, every approved autologous cell-based therapy relies on freezing cells. Reconciling these truths can be key for the success of any commercial therapy based on…

news

Oligonucleotide ALS therapy approved in EU

31 May 2024 | By Catherine Eckford (European Pharmaceutical Review)

The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.

article

Pioneering AAV gene therapy for Krabbe disease

30 May 2024 | By Catherine Eckford (European Pharmaceutical Review), Dr Maria Escolar (Forge Biologics)

Dr Maria Escolar, Chief Medical Officer, Forge Biologics, explores the advantages of the company’s novel AAV gene therapy for Krabbe disease, which could help to overcome some of the immune and safety challenges the gene therapy sector is facing.

article

Pharma Horizons: Cell and Gene Therapy

30 May 2024 | By European Pharmaceutical Review

This report is dedicated to exploring some of the latest innovations in the cell and gene therapy space, from development and quality control, through to manufacturing and clinical trials.

webinar

Pharmaceutical continuous manufacturing: what’s next for industry?

13 May 2024 | By European Pharmaceutical Review, sponsored by MasterControl

During this virtual panel, industry experts will discuss adoption of continuous manufacturing in the pharmaceutical industry.

news

“Landmark” genetic blindness CRISPR trial outcomes released

6 May 2024 | By Catherine Eckford (European Pharmaceutical Review)

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.

news

Enhancing medicine manufacturing through digitalisation

1 May 2024 | By Catherine Eckford (European Pharmaceutical Review)

Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.

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Gene therapy