Drug Profile

Research programme: gene therapies - Aavatar Therapeutics

Latest Information Update: 19 Feb 2026

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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

Preclinical Dry macular degeneration; Globoid cell leukodystrophy; Lysosomal storage diseases; Mucolipidoses

13 Feb 2026 Preclinical trials in Dry macular degeneration in South Korea (Unspecified) before January 2026 (Aavatar Therapeutics pipeline, February 2026)
13 Feb 2026 Preclinical trials in Globoid cell leukodystrophy in South Korea (Parenteral) before January 2026 (Aavatar Therapeutics pipeline, February 2026)
13 Feb 2026 Preclinical trials in Lysosomal storage disease in South Korea (Parenteral) before January 2026 (Aavatar Therapeutics pipeline, February 2026)

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