news
CHMP meeting highlights – December 2022
A total of five drugs were recommended for approval at the CHMP’s December meeting, including a gene therapy and two cancer drugs.
List view / Grid view
Gene therapy
news
CHMP adopts positive opinion for haemophilia B gene therapy
Gene therapy etranacogene dezaparvovec has been granted a positive opinion by the CHMP as a haemophilia B treatment for adults in Europe.
news
First patient given base-edited CAR T cells
The first ever use of base-edited T-cells have helped to treat a patient with relapsed T-cell acute lymphoblastic leukaemia in a clinical trial.
news
Oligonucleotide synthesis market to reach $16.7 billion by 2027
Ongoing clinical trials for oligonucleotide-based therapies will propel the oligonucleotide synthesis market to reach $16.7 billion by 2027, states a new report.
news
FDA approves first gene therapy for Haemophilia B
Hemgenix, the first gene therapy for adults with Haemophilia B has been approved by the US Food and Drug Administration (FDA).
webinar
Optimise fermentation processes by implementing respiratory gas analysis PAT
22 November 2022 | By Thermo Fisher Scientific
Watch on-demand to learn how Sanofi utilises online process mass spectrometry for continuous respiratory gas analysis to improve process insight and drive better outcomes.
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.
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.
