blood-brain-barrier
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ProJenX
ProJenX is a New York City clinical-stage biotechnology company developing prosetin, a first-in-class, oral, brain-penetrant MAP4K inhibitor for amyotrophic lateral sclerosis (ALS) and other neurodegenerative brain diseases. Spun out of a two-decade collaboration between the nonprofit Project ALS and Columbia University researchers, ProJenX is built on the discovery that inhibiting MAP4 kinases protects motor neurons across multiple patient-derived stem cell models of ALS. The company is running the PRO-101 Phase 1b trial in people living with ALS.
Sangamo Therapeutics, Inc.
Sangamo Therapeutics is a clinical-stage genomic medicine company in Brisbane, California that engineers zinc finger proteins to switch genes on, off, or rewrite them. After three decades pioneering zinc finger technology, it is racing to bring its lead gene therapy ST-920 for Fabry disease to market while building a neurology pipeline aimed at crossing the blood-brain barrier - all under the pressure of a 2026 search for strategic alternatives.