Cell and gene therapy

A gene therapy targeting a rare brain disorder has received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). PTC Therapeutics’ one-time gene replacement therapy Upstaza was given a positive opinion for the treatment for aromatic L-amino acid decarboxylase (AADC) in patients 18 months or […]

By Dr. Deborah Phippard, Chief Scientific Officer of Precision for Medicine and Anshul Mangal, President & General Counsel, Project Farma. In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are transforming the way diseases are treated but are also aiming to cure entirely new patient populations. New applications and […]

Featuring Specialty Spotlight on Immunology, the magazine is available to download and read for free on the DDW App.    Key features of the spring issue include:  Can age-related disease be a thing of the past? Cell & Gene Therapies: Opportunities for Innovation; Animal Research: Reinventing the Norm, plus exclusive interviews and articles on genomics, biomarkers and the microbiome.    Exclusive DDW & SLAS Europe supplement    […]

The Cell and Gene Therapy Catapult (CGT Catapult) has published recommendations on how the UK can continue to maintain its global advantage in the adoption of Advanced Therapy Medicinal Products (ATMPs).    The organisation’s ‘National Cell and Gene Therapy Vision for the UK’ outlines the provision of cell and gene therapies in the UK to date […]

The International Society for Cell & Gene Therapy (ISCT) has established a task force to address practical, ethical, and regulatory issues that arise from the use, and potential misuse, of expanded access within the cell and gene therapy (CGT) sector.   Context  Expanded access is a pathway available under FDA regulations that in limited circumstances […]

Orgenesis, a global biotech company working with cell and gene therapies, and The Johns Hopkins University in the US, have collaborated to construct a cell and gene therapy processing facility for point of care treatment of patients at Johns Hopkins which is planned to start in the second quarter of 2022 and is expected to be […]

Ori Biotech, a producer of cell and gene therapy (CGT) manufacturing technology, has secured over $100 million in an oversubscribed Series B funding round and will use the funds to further expand key personnel across all functions, in order to rapidly transition through pre-commercialisation to the launch of its CGT manufacturing platform.   The Ori Platform  The Ori platform […]

Chimeric Antigen Receptor or CAR-T cells, a new form of immunotherapy with genetically engineered immune system cells, has been approved for the treatment of multiple myeloma, a type of blood cancer.  Context  In a case study published in Nature Medicine, Sinai scientists reported a previously unknown neurological side effect resembling symptoms Parkinson’s Disease in a patient […]

The Cell and Gene Therapy Catapult (CGT Catapult) has published new data in their 2021 Cell and Gene Therapy GMP manufacturing report and 2021 UK cell and gene therapy skills demand report. These reports highlight the continued growth across the UK’s Advanced Therapy Medicinal Product (ATMP) manufacturing capacity and strong demand for highly skilled personnel […]

The Cell and Gene Therapy (CGT) Catapult has today published its Annual Review 2021, highlighting its key initiatives and impact in creating opportunities and investment in the UK cell and gene therapy industry.  2021 has reportedly emphasised the strength of the UK cell and gene therapy industry which experienced significant expansion despite the Covid-19 pandemic. The UK […]