The US Food and Drug Administration has approved a new indication for Casgevy, a cell-based gene therapy for two blood disorders, making it available to individuals as young as two years. The disorders are sickle cell disease, a rare inherited blood disorder, and transfusion-dependent beta thalassaemia. Casgevy (exagamglogene autotemcel) was first approved by the FDA in 2023 for children with sickle cell disease from the age of 12 years.
uniQure NV has raised $259 million from a public share offering on Nasdaq a little more than a week after receiving positive feedback from the US Food and Drug Administration for a marketing authorisation application for the gene therapy AMT-130. Proceeds from the offering were announced on 25 June. They will be used to advance the therapy, which has been developed to treat Huntington’s disease, towards commercialisation. The company is expected to file an application for an accelerated approval of the therapy during the third quarter.
TRIMTECH Therapeutics Ltd, which is developing small molecule drugs that target and degrade protein aggregates associated with neurodegenerative diseases, has raised an additional $14 million in seed funding, bringing the total for the round up to $47 million. The financing was led by Johnson & Johnson Innovation and the Business Growth Fund Ltd (BGF), which joined existing investors.
Eli Lilly and Company has broadened an existing partnership with Shanghai, China-based Abbisko Therapeutics Co Ltd to collaborate on the discovery and development of new, but still unidentified medicinal products. In a statement released on 23 June, the US company said the upgraded collaboration is valued at about $1.9 billion.
California, US-based Frazier Life Sciences has led a $115 million Series A financing for RQ Biotechnology Ltd, a UK company developing engineered antibody therapies for influenza in order to provide durable protection against serious viral infections. The investor syndicate also includes the venture capital groups Forbion and EQT Life Sciences, alongside founding investor LifeArc Ventures.
AbbVie Inc, which for years marketed Humira, a former blockbuster medicine, is to replenish its portfolio with the acquisition of Apogee Therapeutics, a clinical stage biotech company with candidate products for inflammatory and immunological diseases. Announced on 22 June, the all-cash transaction values Apogee at $135.11 per share or approximately $10.9 billion. This represents a premium of about 49.5% over Apogee’s closing price on Nasdaq the day before the announcement.
The US Food and Drug Administration has approved a new carbapenem antibiotic from GSK Plc to treat complicated urinary tract infections – the first oral treatment of its kind for the disease. The authorisation, announced on 17 June, is for Utebzi (tebipenem pivoxil), a broad-spectrum carbapenem antibiotic of the beta-lactam class. The treatment will be available as a tablet, in addition to its availability as an in-hospital intravenous therapy.
uniQure NV has reached an agreement with the US Food and Drug Administration to proceed with a marketing authorisation application for its gene therapy for Huntington’s disease, a rare, inherited neurodegenerative disorder. The application is expected to be filed in the third quarter of this year, the company announced on 17 June.
Ona Therapeutics SL, a 2019 spin-out from a Spanish research institute and foundation, has raised $86.6 million from venture capitalists to advance a portfolio of early-stage antibody-drug conjugates (ADCs) for refractory cancers. The Series B round was co-led by Columbus Venture Partners of Spain and Mérieux Equity Partners of France. They were joined by the existing Spain-based investors Alta Life Sciences and Asabys Partners and Bpifrance, the French public investment bank.
GSK Plc is to acquire the US oncology developer Nuvalent Inc for $10.6 billion, giving it two late-stage products for non-small cell lung cancer to complement its own Phase 3 antibody-drug conjugate (ADC) for a different lung cancer. The deal, which will be financed with new and existing debt facilities, also gives the UK company a third lung cancer drug in Phase 1 and a preclinical portfolio of undisclosed cancer assets.