News uniQure falls on long-term Huntington's gene therapy data uniQure's latest data drop for its gene therapy for Huntington's disease, under review in the US and UK, has spooked investors.
News Beacon's XLRP gene therapy set for filing after phase 3 win Beacon is preparing to file for approval of its gene therapy for X-linked retinitis pigmentosa, after reporting encouraging phase 3 data.
News Ultragenyx gets FDA nod for first Sanfilippo A gene therapy Patients in the US with Sanfilippo syndrome type A have their first FDA-approved therapy, Ultragenyx Pharma's $3.9m one-shot gene therapy Fayuvi.
News Novartis' new SMA gene therapy cleared in EU Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.
News Vertex gets paediatric FDA okay for Casgevy gene therapy For the first time, children in the US as young as two with blood disorders sickle cell disease and beta thalassaemia are eligible for gene therapy.
News uniQure sets course to US filing for Huntington gene therapy Shares in uniQure have rocketed this morning, as it emerges that the FDA will not require it to run a new trial of Huntington's gene therapy AMT-130.
News Iambic prices its IPO, seeking to raise up to $160m AI-powered drug discovery company Iambic Therapeutics has announced the terms of its Nasdaq IPO, pitching at a valuation of more than $800m.
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