Gene therapy

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB).  People with dystrophic epidermolysis bullosa often suffer from large open wounds that last for years or decades. It’s an intensely painful condition, and medical treatment for it has been limited largely to palliative […]

Irish biotech company OmniSpirant and French biotech EVerZom have joined other partners to launch INSPIRE, a €12.8 million lung cancer project funded by Horizon Europe.   The project gathers European partners spanning across biotechnology, medical devices, cancer research, academia and patient advocacy. Over the next three years, the consortium will progress the development of a regenerative gene […]

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry news.  The industry has seen breakthroughs announced in malaria treatment, epilepsy and major depressive disorders. The NHS in England has fast tracked another […]

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases.  The research focuses on a group of small, ubiquitous viruses called ‘parvoviruses’ (from the Latin word ‘parvus’ meaning ‘small’, ‘puny’ or ‘unimportant’). It was led by scientists at the MRC-University of Glasgow […]

News round-up for 28 November – 2 December by DDW Digital Content Editor Diana Spencer  The last seven days have seen breakthroughs announced in malaria treatment, epilepsy and major depressive disorders. The NHS in England has fast tracked another cancer drug, and, in Covid-19 news, researchers have emphatically proven that prednisolone should not be prescribed […]

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy.   The therapy, which is being developed in partnership with Roche, has been granted Priority Review by the FDA, with a regulatory action date […]

PerkinElmer has launched ready-to-use adeno-associated virus vectors (AAV) detection kits to support researchers working on gene therapies for a variety of serious diseases.   The validated and fully automatable assays are built on PerkinElmer’s AlphaLISA technology which requires no separation and are optimised, no-wash AAV detection assays. The new offering provides researchers expanded options to measure […]

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry news.  Gene therapies have made the headlines over the last week, with treatments for rare AADC deficiency, lung cancer and haemophilia B given […]

News round-up for 21-25 November by DDW Digital Content Editor Diana Spencer  Gene therapies have made the headlines over the last few days, with treatments for rare AADC deficiency, lung cancer and haemophilia B given the go ahead from regulators. This week has also seen the first immunomodulatory treatment for type 1 diabetes approved in the […]

The US Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with haemophilia B.   The drug is approved for the treatment of adults with haemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening haemorrhage or have repeated, serious […]