Drug Profile

JLK 247

Alternative Names: JLK-247; Mucopolysaccharidosis IIIC gene therapy - Phoenix Nest

Latest Information Update: 11 Aug 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.

Yes - Mucopolysaccharidosis III
New Molecular Entity No

11 Aug 2026 JLK-247 is still in preclinical trials for Mucopolysaccharidosis III in USA (Phoenix Nest pipeline, August 2026)
11 Aug 2026 Preclinical trials in Retinitis pigmentosa in USA (Ophthalmic) prior to August 2026 (Phoenix Nest pipeline, August 2026)
05 Aug 2026 Phoenix Nest receives SBIR grant from National Institutes of Health (NIH) and National Institute of Neurological Disorders and Stroke (NINDS) for JLK 247 development in Mucopolysaccharidosis III

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