For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.
Gene therapy’s one-and-done mindset proved “a boon and a bane,” said Avak Kahvejian, general partner at Flagship Pioneering. “You have one shot to get it right, is what that really means,” and his firm founded Ring Therapeutics Inc. to design redosable drugs in the space.
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 in draft guidance – although experience from Novartis AG’s pricey Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy shows this could change.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.
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