One year after arriving on the scene with a $120 million series A, ophthalmic gene therapy specialist Beacon Therapeutics Ltd. has raised $170 million in a series B.
Curevac AG is casting off the deadweight of its pandemic push to translate its mRNA technology into a marketed COVID-19 vaccine, in a new €1.45 billion (US$1.6 billion) deal in which GSK plc will acquire full rights to infectious disease vaccines the two were co-developing.
Two drugs were pushed back by the EMA last week, with a recommendation that Ocaliva, currently the only second line standard of care for treating primary biliary cholangitis, be withdrawn from the market, and a refusal to grant conditional approval for masitinib in the treatment of amyotrophic lateral sclerosis.
CSL Behring’s expensive hemophilia B gene therapy is to be reimbursed by the U.K. National Health Service, after the company agreed to an outcomes-based payment scheme. The therapy, Hemgenix (etranacogene dezaparvovec), which has a U.K. list price of £2.6 million (US$3.3 million), was approved under a managed access scheme, in which data will be collected over five years to enable both the long-term effectiveness, and any adverse liver toxicity caused by the transgene, to be monitored.
The industry is again raising concerns that new EU health technology assessment rules coming into force on Jan. 12, 2025, will have the opposite of the desired effect and slow down access to innovative therapies.
New research has pinpointed gene signatures that determine what immune responses will be activated in the development of sepsis, pointing to novel targets and opening the way for the stratification of clinical trials and for patients to be treated on the basis of their immune response, rather than their symptoms.
Newco Yellowstone Biosciences Ltd. has been formed to develop soluble bispecific T-cell receptors against a novel class of tumor-specific antigens it has discovered in leukemia patients who were cured by a donor stem cell transplant. The company has proprietary access to a biobank of over 10,000 samples from more than 3,000 acute myeloid leukemia (AML) patients. A small number of these patients were cured by an allogeneic bone marrow transplant, whilst unusually, having no sign of graft-vs.-host disease.
New research has pinpointed gene signatures that determine what immune responses will be activated in the development of sepsis, pointing to novel targets and opening the way for the stratification of clinical trials and for patients to be treated on the basis of their immune response, rather than their symptoms.
Quoted biotech investor Syncona Ltd. is still struggling with the fallout from one of the worst bear markets on record, reporting its net asset value fell from £1.25 billion (US$1.58 billion) to £1.24 billion in its 2023-24 financial results on June 20. That is despite a restructuring toward more mature companies, with 70% of the portfolio now invested in clinical-stage assets.
Ionctura SA closed a €80 million (US$86 million) series B round as it prepares to accelerate development of lead asset roginolisib in the treatment of uveal melanoma. That follows publication of phase Ib data showing roginolisib, an orally available allosteric modulator of the delta isoform of PI3K (phosphatidylinositol-4,5-bisphosphate kinase), is distinguished from other drugs in this class, in being well-tolerated over long periods, with patients having been treated for up to 38 months.
