Drug Profile

Research programme: oligonucleotide based RNA base editing therapeutics - Edigene

Latest Information Update: 28 Jun 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
Available For Licensing Yes

Research Cancer; Eye disorders; Musculoskeletal disorders; Neurological disorders
No development reported Mucopolysaccharidosis I

28 Jun 2024 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-I in China (Parenteral)
08 Nov 2022 Early research in Eye disorders in China (Parenteral), prior to November 2022
09 Nov 2021 EdiGene enters into a research collaboration with the University of Wisconsin–Madison for the development of LEAPER™ RNA editing technology into in vivo therapies

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