amyotrophic-lateral-sclerosis
(2)
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.
Trace Neuroscience
Trace Neuroscience is a South San Francisco biopharmaceutical company developing genomic medicines for neurodegenerative diseases, starting with ALS. Its lead program, TRCN-1023, is an antisense oligonucleotide designed to restore UNC13A protein function - a target genetically validated in roughly 97% of people living with ALS - by correcting the faulty RNA splicing caused by TDP-43 dysfunction. The company launched in November 2024 with a $101 million Series A led by Third Rock Ventures and entered the clinic in 2026.