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Pharma & Biotech

Is JCR unlocking the CNS for lysosomal storage diseases before gene therapy gets there?

by Pallavi Madhiraju
February 5, 2026
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Find out how JCR Pharmaceuticals is redefining lysosomal disease therapy with brain-targeted delivery platforms.

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Latest news and features

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POPULAR NEWS

Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

Why Mirum Pharmaceuticals and Incyte’s zilurgisertib data could reset expectations in FOP treatment

June 15, 2026

Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

Niagen wins twin rare-disease designations, but can NB4168 turn nicotinamide riboside evidence into a medicine?

July 14, 2026

Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

Gilead and Merck pipeline update reveals promise in HIV and pressure in oncology

June 9, 2026

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AstraZeneca clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immunotherapy Inc. Johnson & Johnson medical devices non-small cell lung cancer oncology precision oncology U.S. Food and Drug Administration United States Food and Drug Administration

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