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.
List view / Grid view
Gene therapy
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.
news
Almost 400 cell and gene therapies in US pipeline, finds report
New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.
news
Nanoparticle gene therapy drug delivery for paediatric brain cancer tested in mice
A study has shown that PBAE nanoparticles could replace viral vectors in gene therapies and provide a safe treatment option for paediatric brain cancer patients.
news
Gene therapy receives Champalimaud Vision Award
The Champalimaud Vision Award has been awarded to research teams for the development of a gene therapy...
