Drug intelligence / Profile preview

JR-478

Development stage: Preclinical
Lead developer: JCR Pharmaceuticals
Modality: Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

JR-478 is a blood-brain barrier (BBB)-penetrating recombinant beta-hexosaminidase A (HexA) enzyme replacement therapy being developed by JCR Pharmaceuticals in partnership with MEDIPAL HOLDINGS. It is designed to treat GM2 gangliosidosis, a group of lysosomal storage disorders including Tay-Sachs and Sandhoff diseases, which are characterized by the accumulation of GM2 gangliosides in the central nervous system due to HexA deficiency. JR-478 utilizes JCR's proprietary J-Brain Cargo technology, which involves a fusion protein that binds to the transferrin receptor to facilitate transport across the BBB, allowing the therapeutic enzyme to reach the brain and degrade accumulated substrates.

Targets

GM2 (Ganglioside GM2)TFRC (Transferrin Receptor)

Beyond the preview

Go deeper on JR-478.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on JR-478.

Explore the full profile