The National Institute of Neurological Disorders and Stroke (NINDS) has awarded a 3-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital to fund completion of preclinical studies, manufacturing and preparation of an IND application for a first-in-human trial to advance adeno-associated virus (AAV)-ATP7A gene therapy for the treatment of Menkes disease.

Eilean Therapeutics LLC has announced the acquisition of Ness Therapeutics Inc. in an all-equity transaction, including Ness’ best-in-class tyrosine-protein phosphatase non-receptor type 2 (PTPN2) inhibitors with application in immuno-oncology.

A male patient who harbored a deletion in the intron 7-exon 8 region of the dystrophin gene, DMD, was showing symptoms that matched with Becker muscular dystrophy.

Scientists from Sun Yat-sen University Cancer Center (SYSUCC) and affiliated organizations have reported data from a study that assessed the role of discs large-associated protein 5 (DLGAP5) in lung adenocarcinoma (LUAD).

Fibrocor Therapeutics Inc. has entered into a research and development collaboration with the Mcquade Center for Strategic Research and Development LLC (MSRD).

Researchers from Alumis Inc. have presented preclinical data for a novel, potential first-in-class tyrosine kinase 2 (TYK2) inhibitor – A-005.