Novel haemophilia B gene therapy approved
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
List view / Grid view
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
The new members of the Cell and Gene Therapy Catapult’s board hold expertise across therapy development, digitalisation and leadership.
Q&A with Dr Jin Li and Dr Chad Galderisi of ICON Specialty Laboratory Solutions on how Next Generation Sequencing can support more accurate monitoring of chronic myeloid leukemia patients.
This in-depth focus features articles on bioreactor size, as well as bioprocess development and manufacturing of bioconjugated products.
European Pharmaceutical Review Issue 2 includes articles on cell and gene therapy, from supply chain and manufacturing to microbial testing.
A study has demonstrated how 3D printing can be used to manufacture tailored, safe, and effective treatments for patients with rare diseases via pharmaceutical compounding.
This approval follows a development and commercialisation agreement made in September 2023 between Sandoz and Samsung Bioepis for the biosimilar ustekinumab.
Patients with moderate-to-severe atopic dermatitis can strive for both little to no itch and clearer skin with AbbVie’s upadacitinib, topline Phase IIIb/IV study results suggest.
26 April 2024 | By Veolia Water Technologies & Solutions - Sievers Analytical Instruments
Watch this on demand webinar to hear about the latest RMM technology and how it aligns with USP testing criteria. This webinar will cover testing strategy, and data will be presented to effectively demonstrate suitability of the Soleil for its intended use and correlation to plate counts.
The driving factor for protein particle formation is surface mediated in protein only formulations in manufacturing, research asserts.
Despite some challenges, for the Alzheimer’s drug development pipeline, 2024 is a “learning year”, an expert on the disease says.
An “innovative, life-extending treatment” for BRAF V600E mutation-positive glioma has been recommended for certain young people on the NHS.
Initial trial results show the gene therapy enabled up to 85 percent of muscle fibres expressing microdystrophin in boys with Duchenne muscular dystrophy eight weeks post-injection.
Authorisation of the antibiotic combination, which targets multidrug-resistant bacteria, could help to address the global challenge of antimicrobial resistance (AMR).
The novel approval provides a dedicated therapy option for patients with ALK-positive early-stage lung cancer.