Drug Profile

Research programme: gene therapies - CSL Behring/Seattle Childrens Research Institute

Latest Information Update: 28 Jul 2024

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At a glance

Orphan Drug Status

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

No
New Molecular Entity Yes

28 Jul 2024 No recent reports of development identified for research development in Agammaglobulinaemia in USA (Parenteral)
28 Jul 2024 No recent reports of development identified for research development in Wiskott-Aldrich-syndrome in USA (Parenteral)
02 Jun 2020 Seattle Children's Research Institute and CSL Behring agree to co-develop stem cell gene therapies for Wiskott-Aldrich syndrome and Agammaglobulinaemia

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