news
CRISPR-based microbial gene therapy delivers promise
A microbial gene therapy can “eliminate antibiotic-resistant E. coli strains in the gut”, according to first-in-human study interim results.
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Gene therapy
news
First redosable gene therapy approved
The “landmark approval” of a redosable gene therapy for dystrophic epidermolysis bullosa “ushers in a whole new paradigm to treat genetic diseases".
news
New UK point of care manufacturing regulatory framework
A first-of-its-kind regulatory framework for point of care manufacturing of innovative medicines is set to be introduced in the UK, ensuring these products are as safe as conventional medicines.
news
Breakthrough gene therapy gives hope for Artemis-SCID
A new gene therapy developed by UC San Francisco has enabled ten young Artemis-SCID patients to achieve full T-cell immunity.
news
FDA approves first gene therapy for high-risk early bladder cancer
The first gene therapy for high-risk non-muscle-invasive bladder cancer has been approved by The US Food and Drug Administration (FDA).
podcasts
EPR Podcast Episode 16 – Evolving QC for Cell & Gene Therapy – Félix Montero Julian and Thomas Jones, bioMérieux
What’s new in the cell and gene therapy landscape? What is the role of quality control in efforts to scale up manufacturing of CGT products and alleviate talent constraints? Find out more in this podcast with bioMérieux…
news
Lilly will acquire Akouos to accelerate hearing loss therapies
Eli Lilly and Company’s $610 million acquisition of Akouos will fast-track gene therapies that restore, improve and preserve hearing for patients worldwide.
news
First patient dosed with HIV gene therapy
According to researchers, the CRISPR-based gene therapy, EBT-101, has the potential to change the future of HIV therapeutics.
article
Early phase CGT trials: key design elements correlating to distinctive biological characteristics
With increasing numbers of cell and gene therapies (CGTs) entering clinical trials, Dr Dave Li and Dr Anna Baran of KCR Consulting discuss how the biological characteristics of CGTs should be taken into account with early phase trial designs.
news
Gene therapy shows promise in Wiskott–Aldrich syndrome
Lentiviral gene therapy reduces infections and bleeding episodes in children with a severe immune disorder Wiskott–Aldrich syndrome (WAS), finds new study.
article
Challenges in downstream purification of advanced therapies
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
news
ABPI publishes new roadmap to aid in ATMP development
The roadmap clarifies the processes to develop advanced therapy medicinal products (ATMPs), providing guidance on key considerations.
podcasts
EPR Podcast Episode 5 – ATMP manufacturing and QC – Rey Mali, Accellix and Félix Montero-Julian, bioMérieux
Join Rey Mali and Félix Montero-Julian as they discuss the challenges facing the manufacture and QC of advanced therapy medicinal products (ATMPs) and potential solutions.
news
Pepsin key for fast and efficient AAV peptide analysis, finds study
Researchers have created a new workflow using pepsin to expedite the LC-MS analysis of adeno associated virus (AAV) capsid proteins for gene therapies.
news
Gene therapy successfully treats rare immunodeficiency in paediatric patients
In three Phase I/II trials an experimental gene therapy caused all but two patients with severe combined immunodeficiency to acquire and retain robust immune function for more than two years.
