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Say It With Your Eyes
Tobii Dynavox turns eye movements into speech for people who have lost their voice. Here is how a Pittsburgh-and-Stockholm company built the tools that let hundreds of thousands of people be heard.
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.
Prosetta Biosciences Inc
Prosetta Biosciences is a San Francisco biotech using a proprietary cell-free protein synthesis and assembly (CFPSA) platform to discover small-molecule 'assembly modulators' - drugs that disrupt the multi-protein complexes behind viral infection, cancer, and neurodegeneration. Founded by UCSF physician-scientist Vishwanath Lingappa, the company is advancing candidates against ALS/FTD, Alzheimer's, pan-cancer targets, and antivirals.
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.
Unlearn.AI
Unlearn.AI is a San Francisco biotech-AI company that builds disease-specific machine learning models to generate digital twins of clinical trial participants - longitudinal predictions of how each patient would have fared on placebo. Folded into a statistical method called PROCOVA and a trial design called TwinRCT, the approach lets sponsors shrink control arms by up to a third, so more patients receive the experimental drug while trials reach answers faster. Founded in 2017 by physicists Charles Fisher, Aaron Smith and Jon Walsh, the company has raised about $130M and earned a first-of-its-kind EMA qualification for an AI-based method to reduce sample size in pivotal trials.
Augustine Therapeutics
Augustine Therapeutics is a Belgian clinical-stage biotech spun out of VIB-KU Leuven that is building a next-generation class of selective HDAC6 inhibitors for chronic diseases. Using a proprietary non-hydroxamate, non-hydrazide chemotype, the company designs molecules that block HDAC6's harmful catalytic activity while sparing its beneficial functions - aiming to avoid the toxicity that stalled earlier inhibitors. Its lead candidate, AGT-100216, is the first selective HDAC6 inhibitor to enter the clinic for Charcot-Marie-Tooth disease, with a pipeline extending into neurodegenerative and cardio-metabolic indications.
Nura Bio
Nura Bio is a clinical-stage biopharmaceutical company in South San Francisco developing brain-penetrant, small-molecule neuroprotective medicines. Its work centers on SARM1, an injury-activated NAD hydrolase that triggers axon degeneration, an early and common event across many neurological diseases. The lead candidate, NB-4746, is an oral, brain-penetrant SARM1 inhibitor that completed Phase 1 in healthy volunteers and shows protection in preclinical models of ALS, MS, traumatic brain injury and chemotherapy-induced peripheral neuropathy. The company has raised more than $140 million in Series A financing led by The Column Group.
Verge Genomics
Verge Genomics is a South San Francisco biotech that builds drugs from human biology instead of mouse models. By assembling one of the largest proprietary multi-omics datasets drawn directly from human brain and tissue samples, then mining it with machine learning, its CONVERGE platform identifies disease targets and drug candidates for neurodegenerative diseases like ALS, ALS-FTD and Parkinson's. Founded in 2015 by Alice Zhang and Jason Chen, the company famously moved its first AI-discovered drug from research to the clinic in four years. After that ALS candidate failed its early trial, Verge has refocused on its core asset, the platform, repositioning to supply other drug developers with human-grounded target data.
Bloom Science, Inc.
Bloom Science is a San Diego clinical-stage biotech translating the biology of the ketogenic diet into oral live biotherapeutics that act through the gut-immune-brain axis. Built on microbiome research licensed from UCLA and powered by its IrisRx discovery platform, the company is developing BL-001, a once-daily bacterial therapy being studied in obesity and in drug-resistant seizures from Dravet syndrome, with a pipeline reaching toward ALS and neurodegenerative disease.
Leal Therapeutics
Leal Therapeutics is a Worcester, Massachusetts biotech founded in 2021 by repeat CNS entrepreneur Asa Abeliovich, the scientist behind Prevail Therapeutics (acquired by Eli Lilly). Leal builds first-in-class neuro-metabolic medicines on a single idea: many brain diseases share a broken metabolism, and correcting those imbalances can treat conditions from schizophrenia to ALS. Backed by roughly $114M in total funding, its pipeline includes LTX-001, a brain-penetrant oral glutaminase inhibitor in the clinic, and LTX-002, an antisense oligonucleotide for ALS.
insitro
insitro is a machine-learning-driven drug discovery and development company that uses high-content human data, cellular models and AI to find better targets and design better medicines. Founded in 2018 by Daphne Koller, the company runs its own therapeutic pipeline and partners with large pharma on programs in ALS, metabolic disease and oncology.