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Elpida Therapeutics: Accelerating gene therapies for ultra‑rare neurodegenerative diseases

Research area:

Rare disease

Disease:

Spastic Paraplegia type 50 (SPG50), Neuronal ceroid lipofuscinosis 7 (CLN7 Batten disease), Charcot–Marie–Tooth disease type 4J (CMT4J).

Intervention:

Therapeutics – ATMP

Phase:

Funding awarded to

Elpida Therapeutics