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JR-403

Amyotrophic Lateral Sclerosis (ALS)

PreclinicalResearch

Key Facts

Indication

Amyotrophic Lateral Sclerosis (ALS)

Phase

Preclinical

Status

Research

Company

JCR Pharmaceuticals

About JCR Pharmaceuticals

JCR Pharmaceuticals has established itself as a premier global specialty pharmaceutical company focused on high-unmet-need rare diseases, particularly lysosomal storage disorders. Its strategic advantage is anchored in the proprietary J-Brain Cargo® platform, which enables targeted delivery of biologics across the blood-brain barrier—a key differentiator in treating neurological manifestations. The company has successfully commercialized products in multiple international markets and maintains a robust clinical pipeline, driving its significant market valuation. JCR's fully integrated model, from R&D to global commercialization, combined with a strong venture spirit, positions it for sustained growth in the orphan drug space.

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Therapeutic Areas

Lysosomal Storage Disorders Neurological Diseases Rare Genetic Diseases

Other Amyotrophic Lateral Sclerosis (ALS) Drugs

Drug	Company	Phase	Status
NPT520-34	Neuropore Therapies	Pre-clinical/Phase 1	Partnered (Biogen), Active
CNM-Au8	Clene	Phase 2/3	Active
Masitinib	AB Science	Phase 3	Active
SLS-005	Seelos Therapeutics	Platform Trial	Active (HEALEY ALS Platform Trial – Regimen E)
nAV-101	nVector	Pre-clinical	Active
ALTA-808	Alteron Therapeutics	Preclinical	IND-enabling studies launching
GM6	Genervon Biopharmaceuticals	Phase 2	Active
Lou Gehrig’s disease	Genclis	Discovery	Active
Treg-directed therapy for ALS	Iltoo Pharma	Pre-clinical	Active
NG CELL	NeuroGenesis Bio	Phase 2	Active
NP001	Neuvivo	Phase 3	Active
BREN02 / hEN1	BrainEver	Pre-clinical	Active