Rare Disease

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Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Sarepta helps bankroll new gene therapy startup AavantiBio

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences

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Chord gets $16m to challenge Alexion with oral NMOSD drug

Swiss biotech Chord Therapeutics has come out of stealth mode with a $16 million first-round financing that will be used to progress a repurposed drug for rare disease neuromyelitis optica

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EMA starts rapid review of Bluebird’s gene therapy for rare ...

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.<

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Novartis' bid to expand use of SMA gene therapy delayed afte...

Analysts have said the FDA’s request for a new trial will delay Novartis’ intrathecal formulation of its spinal muscular atrophy (SMA) gene therapy in older patients until 2023, potentially

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NICE relents and okays Akcea’s rare disease drug Waylivra fo...

Patients with the rare disease familial chylomicronemia syndrome (FCS) will be able to access treatment with Akcea’s Waylivra via the NHS, after a U-turn by NICE.

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Regeneron’s cholesterol drug evinacumab claims February FDA ...

The FDA has started a speedy review of Regeneron’s ANGPTL3-targeting antibody evinacumab for a rare, inherited disorder that dramatically raises the risk of heart disease.<