gene therapy
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Eli Lilly discontinues Gaucher type 1 gene therapy trial from Prevail buyout
Lilly disclosed the decision as part of its second-quarter results, removing a phase 1/2 study of LY3884961 in Gaucher disease type 1 (GD1) from its pipeline.
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NueCell Bio launches in San Diego as specialized cell therapy translational partner
The company’s scientific capabilities include chimeric antigen receptor T-cell (CAR-T) therapies and treatments derived from induced pluripotent stem cells (iPSCs).
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Johnson & Johnson expands in vivo CAR-T push with US$2.58B deal with Sail Biomedicines
Under the agreement, the companies will advance Sail’s lead programme, SAIL-0839, which is being developed to engineer CD4+ and CD8+ T-cells inside the body to treat immune-mediated diseases.
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Cyllene Therapeutics secures US$37.8 M to advance gene therapy for severe neurogenic bladder disorders
The financing round was co-led by M Ventures, the venture capital arm of Merck KGaA, and GordonMD Global Investments.
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Vertex to acquire Crinetics in US$10B deal to expand rare endocrinology portfolio
Under the definitive agreement, which both companies’ boards unanimously approved, Vertex will pay US$85.00 in cash for each outstanding Crinetics share.
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Vertex Casgevy wins landmark FDA approval for toddlers with SCD and TDT
The expanded label is expected to make about 5,500 additional children in the United States eligible for treatment.
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Moderna launches in Vivo CAR-T mRNA therapy mRNA-6007 for autoimmune diseases
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.
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CellSave Arabia collaborates with HMS Group for UAE stem cell care
Under this agreement, CellSave Arabia will act as the exclusive provider of cord blood, cord tissue, placental tissue, cord vessel, and amnion preservation services.
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Schneider Children’s Medical Center administers world’s first WWOX gene therapy for rare epilepsy
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.
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LEO Pharma acquires Replay to advance their gene therapy pipeline for rare genetic skin diseases
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.