Company News

A UK biotech launched in 2023 to use protein degradation technology to develop new drugs for neurodegenerative diseases has raised $31 million in seed funding from a selection of UK and US investors. The company, TRIMTECH Therapeutics Ltd, has a pipeline of small molecule candidate therapies for disorders including Alzheimer’s and Huntington’s diseases. The drugs are intended to target the body’s ubiquitin-proteasome system to mark proteins for degradation. The proteins are toxic aggregates associated with a range of neurodegenerative and inflammatory disorders.

UK-based Centauri Therapeutics Ltd has selected a lead compound for clinical development capping a decade of research into potential agents for treating serious bacterial infections. The compound, as yet unnamed, has been designed to target Gram-negative infections in the lung. Infections from Gram-negative bacteria are typically difficult to treat because the bacteria are surrounded by a cell wall in addition to an outer membrane containing a complex molecule known as a lipopolysaccharide. By comparison, Gram-positive bacteria do not have this outer membrane.

An Israeli medical device company has been sold to Boston Scientific Corp of the US concurrent with the start of a pivotal trial intended to confirm the effectiveness of its lead product for hypertension. SoniVie Ltd has developed an intravascular device for renal denervation procedures. These procedures reduce activity in the renal nerves in the kidneys in order to help lower blood pressure. France-based Andera Partners has been a venture investor in the company since 2023. Separately, Boston Scientific has been a minority shareholder.

A new gene therapy has been given a positive opinion by the European Medicines Agency for the treatment of dystrophic epidermolysis bullosa (DEB), a rare skin disease. The therapy, Vyjuvek (beremagene geperpavec), has been cleared for the treatment of the disease in patients of all ages.

A new small molecule combination treatment for cystic fibrosis has been given a positive opinion by the European Medicines Agency – expanding the repertoire of protein modulators for treating this disease. The combination, Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor), was developed by Vertex Pharmaceuticals Inc. It modulates the cystic fibrosis transmembrane conductance regulator (CFTR) protein which is produced by a gene of the same name.

AMYRA Biotech AG, a Basel, Switzerland-based company working in digestive health innovation, has published a peer-reviewed literature review in Alimentary Pharmacology & Therapeutics supporting a novel approach to enzyme therapy for coeliac disease. The study underscores the critical role of the intestinal brush border membrane (BBM) in protein digestion and explores the potential benefits of supplementing exopeptidase activity in affected patients.

A new gene therapy for cystic fibrosis has entered clinical development in the UK with the aim of improving outcomes for people with cystic fibrosis regardless of their gene mutation. The trial specifically focuses on adults with cystic fibrosis who cannot benefit from the current modulators of the membrane protein – cystic fibrosis transmembrane conductance regulator (CFTR).

The US Food and Drug Administration has approved a small molecule drug to treat a rare genetic disorder that can cause progressive damage to the brain and other areas of the body. The drug, Ctexli (chenodiol), is a bile acid replacement therapy developed to treat cerebrotendinous xanthomatosis (CTX), a lipid storage disorder. Patients with CTX are deficient in an enzyme that helps the body break down fats.The disorder is caused by a reduced production of bile acid in the liver, resulting in the deposit of atypical cholesterol metabolites in the brain, liver and other parts of the body.

bluebird bio Inc is to be acquired by two private equity groups in a move to provide more capital to the gene therapy developer which was founded by two academics in 1992 and now has three approved products on the market. The transaction, which was announced on 21 February, will transform bluebird into a private enterprise from its current status as a publicly listed concern on the Nasdaq market. The buyers are Carlyle Group Inc and SK Capital Partners LP.

Novartis is to pay up to $3.1 billion to take full control of Anthos Therapeutics Inc, a Boston, US based company that it helped launch with Blackstone Life Sciences in 2019 to develop an asset for a cardiovascular disease. The company has a product in Phase 3 that was in-licensed from Novartis and is now being developed to prevent stroke and systemic embolism in patients with atrial fibrillation. Novartis had a minority stake in Anthos but will now take full control.