Drug Profile

Research programme: AAV-based gene therapies - Lacerta Therapeutics

Latest Information Update: 28 Mar 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

No development reported Aromatic amino acid decarboxylase deficiency; Glioblastoma; Glycogen storage disease type II; Mucopolysaccharidosis III; Neurodegenerative disorders; Spinocerebellar degeneration

28 Mar 2023 No recent reports of development identified for research development in Glioblastoma in USA
28 Mar 2023 No recent reports of development identified for research development in Spinocerebellar-degeneration in USA
28 Feb 2023 No recent reports of development identified for research development in Aromatic-amino-acid-decarboxylase-deficiency in USA

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