Topic: rare diseases

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Deep Genomics nabs $40M round

Canadian drug development company Deep Genomics has got off a $40 million ound as it looks to double down on its rare genetic disease and AI work.


Alnylam

36. Givlaari

Facing a major challenge to its RNAi therapy Onpattro, Alnylam afforded itself some breathing room with the approval of its second such therapy only a year later.
fda

29. Scenesse

Once the target of multiple buyout attempts, Clinuvel opted to go it alone with its sole drug candidate for rare disease erythropoietic protoporphyria.
Sanofi

2. Cablivi

Cablivi scored an FDA nod in February in combination with plasma exchange and immunosuppression for the treatment of acquired thrombotic thrombocytopenic purpura.