The most common form of hereditary deafness in humans is caused by mutations in the GJB2 gene, which encodes the gap junction protein connexin 26. That regulates the transport of potassium and metabolites between inner ear cells. The coding sequence of this gene fits in an adenovirus-associated vector (AAV), making it an attractive approach for gene therapy.
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.
In keeping with federal standards for classifying race and ethnicity data, the U.S. FDA issued a draft revision to broaden its 2016 guidance on the collection of such data in clinical trials.
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.
Hebecell Corp. and Logomix Inc. have established a strategic partnership to research and develop gene-edited natural killer (NK) cells and discover genetic modifications that can create next-generation designer NK cells. Under the agreement, Logomix provides genome editing capabilities to Hebecell for development of next-generation designer Protonk cells.
Shares in Uniqure NV fell sharply on inconclusive interim data from a U.S. phase I/II trial of its gene therapy for Huntington’s disease. The stock (NASDAQ:QURE) hit a six-year low during trading on June 21, ending the day at $11.62, down 40%.
With its Duchenne muscular dystrophy (DMD) drug, vamorolone, under U.S. FDA review, Santhera Pharmaceutical AG has signed a $231 million-plus-royalties North America commercialization deal with Catalyst Pharmaceuticals Inc.
After almost 30 years in business, Sangamo Therapeutics Inc. is finally nearing a BLA filing for one of its programs. But the company, wounded by the recent loss of alliances with Biogen Inc. and Novartis AG, is also running out of cash and investor interest – and it badly needs a new deal to stay afloat.
