While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

Cirrus Therapeutics Inc. has closed an $11 million seed financing to advance its pipeline of gene and cell therapies designed to preserve sight and extend ocular healthspan in patients with chronic blinding diseases.

When Robert Kennedy Jr. announced the cancellation of 22 projects related to mRNA vaccines and the end of new investments in that technology, the U.S. Secretary of Health only mentioned their use against respiratory viruses, without referring to other applications. The vaccines whose safety and effectiveness Kennedy is questioning are based on the same molecular principles as cancer vaccines under development. “Continued investment in mRNA technology is essential to fully realize its potential in oncology and ensure that promising strategies like neoantigen-based vaccines reach clinical application.” Kazuhiro Kakimi, professor at the Department of Immunology at Kindai University Faculty of Medicine, told BioWorld.

An ongoing concern for scientists is that there will be across-the-board funding cuts. This is already happening in mRNA research, where reductions affected coronavirus-related projects. During the pandemic, efforts focused on this pathogen, and once the health emergency was over, grants for antivirals were eliminated. However, these drugs could stem future outbreaks. Despite the cuts, recent research continues to demonstrate the potential of mRNA, not only for the development of antivirals, but also for obtaining more effective and longer-lasting vaccines.

Researchers from the University of California, Davis have designed a novel gene therapy vector that selectively targets and kills cells infected with Kaposi’s sarcoma-associated herpesvirus (KSHV).

Entos Pharmaceuticals Inc. has been awarded a $4 million grant by California Institute for Regenerative Medicine (CIRM) to support the completion of IND-enabling activities with ENTLEP-001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy.

Milleporesigma, Merck KGaA’s North American life sciences business, agreed to acquire Mirus Bio LLC from Gamma Biosciences LP for $600 million. Mirus develops tailored transfection solutions that deliver nucleic acid into cells used in production of viral vector-based gene therapies. It also provides contract development and manufacturing services. The transaction is expected to close in the third quarter of 2024, subject to U.S. regulatory approvals and customary closing conditions.

Neurochase Ltd. filed a patent for a septum-sealed transcutaneous, fluid-transferring device through which repeated access for the removal or delivery of fluid to the central nervous system (CNS) can be gained.