news
MHRA approves first gene therapy for infusion into the brain
Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.
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Gene therapy
article
Heart failure: clinical trials update 2022
EPR rounds up results from several key clinical trials on therapies for hypertension and heart failure presented at the American Heart Association’s Scientific Sessions 2022.
article
Building an integrated vector supply to support autologous cell therapy for solid tumours
Facing limited viral vector manufacturing capacity, should companies consider further integrating their viral vector supply to address this issue? James Nanista and Josh Hunt of Adaptimmune reflect on the options.
news
$8 million grant awarded to new US stem cell clinic
New US stem cell clinics given an $8 million grant will boost industry collaboration and advance regenerative medicine research, clinical trials and pioneering training.
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
First patient receives new engineered mRNA epigenetic
OTX-2002, a programmable epigenetic medicine is the first mRNA therapy to target MYC dysregulation and could treat hepatocellular carcinoma.
news
Astellas invests $50 million in Taysha Gene Therapies
Taysha will license AAV gene therapy candidates TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy to Astellas.
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
$38 million will fund rare disease research over four-year period
Research for rare diseases including ALS will be supported over the next four years by $38 million in FDA funding.
news
Funding boost for CPI’s RNA vaccine innovation centre
The UK Government’s Vaccine Taskforce (VTF) has funded CPI’s innovative training academy and manufacturing facility to further the development of novel RNA therapies and vaccines.
news
Pilot to support ATMP research and development
Five academic and non-profit organisations developing advanced therapy medicinal products (ATMPs) will benefit from the European Medicines Agency’s (EMA) scheme.
video
On-demand webinar: Effective contamination control strategy
Discover the significance of accurate microbial identifications in building an effective contamination control strategy to keep up with the latest regulatory requirements.
webinar
Validation and implementation of a novel automated PCR technology for mycoplasma testing
22 September 2022 | By bioMérieux
This on-demand webinar presents the regulatory submission strategy for implementation and validation of a new PCR-based technology for mycoplasma testing of different products.
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.
whitepaper
Product hub: How can CGT manufacturers ensure product safety with Microbial Solutions QC test methods?
Innovation forms the basis of every emerging technology. That is certainly the case with cell and gene therapies (CGTs), and the search for efficient quality control (QC) test methods continues to grow in this pharmaceutical sector. One of the most important factors to consider while deciding on a QC method…
