Biotech
A new cancer trial aimed at training the immune systems of children and young people to fight the disease offers “a real sense of hope”, according to the chief executive of one of its funders. Up to 60 patients with difficult-to-treat solid tumours will be recruited in the UK and the US for the study, [...]
A research team at Johns Hopkins Medicine and the Johns Hopkins Bloomberg School of Public Health reports developing a therapeutic intranasal (nose-delivered) DNA vaccine against tuberculosis (TB) that fuses two genes with the goal of directing the immune system to fight drug-tolerant bacterial “persisters” that can survive prolonged antibiotic therapy and contribute to disease relapse. [...]
Genentech antibody emugrobart has failed in two rare diseases, raising questions over whether it can preserve muscle in ongoing obesity trials. Roche’s Bay Area subsidiary disclosed the discontinued programmes in separate 19 March letters to the patient communities for spinal muscular atrophy (SMA) and facioscapulohumeral muscular dystrophy (FSHD). For both conditions, Genentech said it had [...]
A gene editing tool safely inserted large DNA segments in mice, pointing to a potential new route for genetic medicines. The technique uses circular, single-stranded DNA rather than the usual double-stranded form of DNA, the molecule that carries genetic instructions. The approach is known as INSTALL, short for "integration through nucleus-synthesised template addition of large [...]
Biodegradable nanoparticles have been engineered to help the immune system find and destroy diseased immune cells in an early mouse study. Researchers said the work advances efforts to engineer immune cells inside a patient's own body to tackle cancers and autoimmune diseases such as lupus. Engineered immune cells have already been used to treat a [...]
A new Dravet syndrome drug has shown promising results in early clinical trials in children with the rare and hard-to-treat form of epilepsy. Dravet syndrome is a rare genetic disorder that causes severe epilepsy that is often resistant to treatment and can also lead to speech and developmental delays. Around 3,000 people in the UK [...]
Atavistik Bio has raised US$160m to develop new treatments for rare blood disorders and cancers using a drug design approach aimed at previously hard-to-target proteins. Atavistik Bio develops oral medicines designed to bind to what are known as allosteric sites on proteins. Proteins are molecules in the body that control many biological processes. Most small-molecule [...]
Azenta has acquired UK Biocentre, a UK provider of sample management, storage and high-throughput sample processing services, in a deal worth up to £20.5m. The acquisition was completed through Azenta’s wholly owned subsidiary Azenta UK Ltd. Azenta said the deal strengthens its ability to deliver end-to-end lifecycle solutions in the UK while expanding its presence [...]
Generate:Biomedicines has raised US$400m in the year’s biggest biotech IPO so far, with proceeds earmarked for clinical trials and R&D on its artificial intelligence platform. The drug developer, which focuses on immunology and inflammation as well as oncology, priced shares at US$16 and began trading on the Nasdaq Global Market under the symbol GENB. [...]
By Sanius Health Gene therapy has now moved decisively from scientific ambition to clinical reality, and in doing so, it has redefined what is possible for patients living with severe genetic conditions such as sickle cell disease, where curative intent is no longer a distant aspiration but a regulated, deployable therapeutic pathway. What we are [...]
