News

Netherlands’s- based VectorY Therapeutics BV has negotiated an option and licensing agreement with a US manufacturer of adeno-associated viral (AAV) capsids ahead of making its first regulatory filings for clinical trials of a new drug for amyotrophic lateral sclerosis. The supplier of the tool is Shape Therapeutics Inc of Seattle, Washington which is developing capsids for genetic medicines for difficult-to-reach tissues. Capsids are the protein shells of AAV viruses that can be used to carry a working copy of a gene into the nucleus of a target cell. 

Roche is to acquire 89bio Inc of San Francisco, US, to expand its cardiovascular, renal and metabolic disease portfolio with a drug that is being developed for metabolic dysfunction-associated steatohepatitis (MASH). MASH (previously called non-alcoholic steatohepatitis) is a liver disease that develops when fat builds up and causes inflammation. Without treatment, it can lead to cirrhosis and liver cancer.

Novo Nordisk A/S has announced plans to cut its workforce by 9,000 as part of a restructuring measure that will enable it to concentrate more resources on its diabetes and obesities medicines. The Danish company is a market leader in both sectors, alongside Eli Lilly and Co, but has experienced setbacks on the US market owing to product shortages and competition. This led to the resignation of its chief executive Lars Fruergaard Jørgensen in May. His successor, Mike Doustdar, is a long-time employee of the company and assumed the position of CEO in August.

Novartis is to spend $1.4 billion to expand its pipeline of drugs for cardiovascular diseases with the acquisition of US-based Tourmaline Bio Inc. Tourmaline’s lead asset, pacibekitug, a monoclonal antibody in development for atherosclrotic cardiovascular disease. The transaction is expected to close in the fourth quarter. Atherosclerotic cardiovascular disease is caused by a build-up of plaque in the arterial walls and includes coronary heart disease and cerebrovascular disease. 

Medicines Discovery Catapult (MDC) of the UK and Crown Bioscience have formed a strategic global alliance to accelerate innovation in radiopharmaceuticals through an integrated translational biology platform. The collaboration aims to support companies across the drug development pipeline by combining MDC’s expertise in radiochemistry, imaging and tissue analysis with Crown Bioscience’s preclinical oncology capabilities.

Quotient Sciences and CPI, a UK manufacturing entity, have agreed to form a joint venture aimed at accelerating the development of RNA-based therapies. The partnership, announced on 2 September, will combine Quotient’s Translational Pharmaceutics platform with CPI’s expertise in RNA manufacture and lipid nanoparticle (LNP) encapsulation, creating what the partners describe as the first fully integrated offering for mRNA drug product development.

Servier has entered into an agreement with Kaerus Bioscience, a UK-based biotechnology company co-founded by Medicxi, to acquire KER-0193, a potential treatment for Fragile X syndrome (FXS). Financial details include an upfront payment, with development and commercial earn-outs that could bring the total value of the deal to $450 million.

Enlaza Therapeutics Inc and Vertex Pharmaceuticals Inc agreed to collaborate on drug discovery aimed at creating small format drug conjugates and T cell engagers to treat certain autoimmune diseases and to improve conditioning in sickle cell disease and beta-thalassemia. Enlaza will receive $45 million in an upfront payment and equity investment. Vertex will work with Enlaza’s covalent biologics platform, called War-Lock, which uses proprietary non-natural amino acids to precision-engineer biologic medicines, resulting in an expanded therapeutic window. 

Replicate Bioscience has entered into a multi-year research collaboration with Novo Nordisk A/S to develop self-replicating RNA (srRNA) therapies for obesity, type 2 diabetes and other cardiometabolic diseases. The deal combines Novo Nordisk’s expertise in metabolic disorders with Replicate’s proprietary srRNA platform, designed to enable scalable and durable protein expression.

A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.