Drug Profile

CAPN 3 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics

Alternative Names: Calpain 3 gene therapy - Nationwide Children's Hospital/Sarepta Therapeutics; CAPN-3; LGMD2A Calpain 3

Latest Information Update: 28 Jun 2023

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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 No

28 Jun 2023 No recent reports of development identified for preclinical development in Limb-girdle-muscular-dystrophy-type-2A in USA (Parenteral)
04 Aug 2021 Sarepta Therapeutics executes licensing agreement for CAPN 3 gene therapy
08 May 2019 Nationwide Children’s Hospital and Sarepta Therapeutics enter into an option agreement for CAPN 3 gene therapy for the treatment of Limb girdle muscular dystrophy type 2A

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