Drug Profile

Research programme: haemophilia A and B gene therapy - Biogen/The San Raffaele Telethon Institute for Gene Therapy

Latest Information Update: 22 Aug 2023

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At a glance

Originator The San Raffaele Telethon Institute for Gene Therapy
Developer Bioverativ; The San Raffaele Telethon Institute for Gene Therapy
Class Gene therapies
Mechanism of Action Blood coagulation factor replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

22 Aug 2023 Discontinued - Preclinical for Haemophilia A in Italy (Parenteral)
22 Aug 2023 Discontinued - Preclinical for Haemophilia A in USA (Parenteral)
22 Aug 2023 Discontinued - Preclinical for Haemophilia B in Italy (Parenteral)

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