Roche Holding AG received CE Mark approval for its Elecsys Apolipoprotein E4 (ApoE4) biomarker test, an in vitro diagnostic immunoassay to identify the ApoE4 gene variant from a blood sample. With APOE4 considered as the strongest genetic risk factor for developing Alzheimer’s disease, the test provides a fast, reliable way to determine whether an individual carries the genetic variant without the need for DNA‑based genotyping.

Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.

Facioscapulohumeral muscular dystrophy (FSHD) is a muscle wasting disease caused by aberrant expression of double homeobox protein 4 (DUX4). When DUX4 is activated in skeletal muscle, it triggers myocyte cell death after several transcriptional changes, thus genetic DUX4 silencing arises as a promising approach for treating FHSD.

Acumen Pharmaceuticals Inc. has announced a $35.75 million private placement to advance molecules from its amyloid-β oligomer-selective Enhanced Brain Delivery (EBD) portfolio for the treatment of Alzheimer’s disease.

G2Gbio Inc. secured a ₩20 billion (US$13.35 million) investment from Samsung Epis Holdings Co. Ltd. and a joint research and license agreement for two assets, including a long-acting obesity treatment, with subsidiary Samsung Bioepis Co. Ltd. March 16.

G2Gbio Inc. secured a ₩20 billion (US$13.35 million) investment from Samsung Epis Holdings Co. Ltd. and a joint research and license agreement for two assets, including a long-acting obesity treatment, with subsidiary Samsung Bioepis Co. Ltd. March 16.

Shanghai Unixell Biotechnology Co. Ltd. has obtained IND clearance from the FDA for UX-GIP001, its iPSC-derived allogeneic cell therapy for focal epilepsy. A phase I study will evaluate UX-GIP001 in patients with drug-resistant epilepsy.

Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.

Researchers from Chemicare Srl and the University of Piemonte Orientale have presented preclinical results regarding their (SOCE) negative regulator CIC-39. Researchers evaluated the dysregulation of SOCE in both ex vivo and in vivo models of Duchenne muscular dystrophy (DMD), as well as evaluated the therapeutic potential of CIC-39 in DMD.