NICE has said “no” to regular NHS funding for a rare disease gene therapy from Orchard Therapeutics Ltd. in draft guidance – although experience from Novartis AG’s pricey Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy shows this could change. That’s because this week NICE published final guidance that recommends funding for Zolgensma, thought to be the world’s most expensive drug, after an initial rejection late last year.
Researchers at Oregon Health & Science University have turned acetaminophen's toxicity into an asset, using it to select genetically modified hepatocytes in vivo.
Researchers at Oregon Health & Science University have turned acetaminophen's toxicity into an asset, using it to select genetically modified hepatocytes in vivo.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.
DUBLIN – Skysona (elivaldogene autotemcel, Lenti-D), Bluebird Bio Inc.’s gene therapy for cerebral adrenoleukodystrophy, received a nod from the EMA’s Committee for Human Medicinal Products (CHMP) during its May meeting this week, paving the way for a formal European authorization in the coming weeks. It will constitute the first approval for the product. An FDA approval is some way behind – the company will not complete its BLA filing with the FDA until around midyear.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: AGTC, Dyne, Genenta, Luye, Metagenomi, Otonomy, Regenxbio, Tessa, Ultragenyx.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: 4D, Affinia, Arcellx, Generation, Homology, Rocket, Sio Gene, Uniqure.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: Antion, Biomarin, Enochian.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: Adaptimmune, Akouos, Beam, Catamaran, Codiak, Dyno, Freeline, M6P, Neurogene, Passage, Phio, Poseida, Precision, Senti, Verve.
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