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gene therapy
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Solid Biosciences receives FDA rare pediatric disease designation for Friedreich’s Ataxia Gene Therapy SGT-212
The therapy delivers the full-length human frataxin gene using a dual-administration approach that combines direct intradentate nucleus (IDN) injection with intravenous (IV) infusion.
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Regeneron partners with Tessera Therapeutics in USD275M deal to develop gene writing therapy for AATD
Regeneron is investing USD 150 million in equity and upfront payments to acquire half of the global development and commercialization rights for TSRA-196.
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Novartis gains FDA approval for Itvisma gene therapy to treat spinal muscular atrophy
Itvisma works by delivering a functional copy of the survival motor neuron 1 (SMN1) gene through a single, fixed-dose intrathecal injection.