gene therapy

Under the definitive agreement, which both companies’ boards unanimously approved, Vertex will pay US$85.00 in cash for each outstanding Crinetics share.

The expanded label is expected to make about 5,500 additional children in the United States eligible for treatment.

The announcement came during Moderna’s Science Day, where the biotechnology company outlined plans to broaden its pipeline across oncology, rare diseases, and immune-mediated disorders.

Under this agreement, CellSave Arabia will act as the exclusive provider of cord blood, cord tissue, placental tissue, cord vessel, and amnion preservation services.

The therapy builds on long-standing research led by Prof. Rami Aqeilan of the Hebrew University of Jerusalem, who helped uncover the critical role of the WWOX gene in brain development.

LEO Pharma will gain access to Replay’s proprietary high-payload herpes simplex virus (HSV) delivery platform, an innovative technology designed to deliver large therapeutic genes directly into skin cells.

The hospital’s team modified the patient’s hematopoietic stem cells to correct the underlying genetic defect, then reinfused the corrected cells back into the body.

The acquisition centers on anitocabtagene autoleucel, known as anito-cel, an investigational CAR-T cell therapy targeting multiple myeloma.

The acquisition gives Lilly access to Orna’s innovative platform, which combines circular RNA (circRNA) with lipid nanoparticle-based therapies.