C-TX87TransportVehicle platform, biotherapeutics, barrier-crossing therapeutics
0-100 chain-logic scale · 15 dimensions · scored on public evidence
Evaluate DNL952 as a treatment for adult participants with late-onset Pompe disease, focusing on safety, tolerability, pharmacokinetics, and pharmacodynamics.
Phase 1 multicenter, open-label dose-level clinical study in adults with late-onset Pompe disease.
Phase 1 clinical trial published on ClinicalTrials.gov on 2026-05-01.
Evaluate DNL628 in participants with early Alzheimer's disease, including mild cognitive impairment or mild Alzheimer's disease with biomarker evidence of amyloid positivity.
Phase 1b multicenter, randomized, placebo-controlled, double-blind, multiple ascending dose study assessing safety, tolerability, pharmacokinetics, and pharmacodynamics.
Phase 1b clinical trial published on ClinicalTrials.gov on 2026-01-30.
Evaluate BIIB122 in participants with Parkinson's disease carrying pathogenic LRRK2 variants that increase LRRK2 kinase activity.
Phase 2a multicenter, randomized, 12-week double-blind, placebo-controlled, parallel-group study followed by an open-label extension, measuring safety, tolerability, and pharmacodynamic effects.
Phase 2a clinical trial published on ClinicalTrials.gov on 2024-10-24.
Explore inhibition of the integrated stress response downstream of TDP-43 pathology in ALS and other neurological diseases.
CNS-penetrant small-molecule eIF2B activator tested in cellular and mouse ALS models, Phase 1 healthy participant study, Phase 1b ALS participant study, and Phase 1 absorption, metabolism, and excretion study using [14C]-DNL343.
Phase 1 absorption, metabolism, and excretion study published on ClinicalTrials.gov on 2024-03-01; 2025 Nature Communications paper reported Phase 1 and Phase 1b clinical findings.
DNL343 inhibited integrated stress response signaling in ALS models, showed extensive CSF distribution, was generally well tolerated, and reduced integrated stress response biomarkers in peripheral blood mononuclear cells and CSF of ALS participants.
Assess DNL126 in pediatric participants with mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A.
Multicenter, open-label Phase 1/2 study assessing safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical efficacy, with core, open-label extension, and long-term extension periods.
Phase 1/2 clinical trial published on ClinicalTrials.gov on 2023-12-07.
Treat neuronopathic or non-neuronopathic mucopolysaccharidosis type II, also known as Hunter syndrome, using a CNS-penetrant enzyme replacement therapy.
Investigational CNS-penetrant intravenous enzyme replacement therapy evaluated in a Phase 2/3 randomized active-controlled study versus idursulfase and a multiregional open-label extension for long-term safety, tolerability, and efficacy.
Open-label extension trial published on ClinicalTrials.gov on 2023-09-20 following the Phase 2/3 study published on 2022-07-21.
Understand how early long-term anti-amyloid beta immunotherapy affects amyloid plaque pathology, microglial activation, and neurodegeneration-related biomarkers in Alzheimer's disease models.
Long-term chronic anti-Aβ treatment in the APP-SAA triple knock-in mouse model, with CSF proteomics, bulk RNA sequencing, glucose uptake analysis, and microglial response assessment.
Publication described early-intervention anti-Aβ immunotherapy effects in a mouse model of amyloidogenesis.
Long-term anti-Aβ treatment robustly and dose-dependently lowered amyloid plaque pathology, reduced CSF markers including Tau and α-synuclein, attenuated disease-associated microglial and glycolytic gene expression, while microglia around residual plaques remained highly activated.
Enable delivery of biotherapeutics across biological barriers, including delivery to the brain and whole body.
Barrier-crossing therapeutic platform, including antibody transport vehicles targeting blood-brain barrier transferrin receptor and CD98 heavy chain, with biodistribution characterization in mouse and cynomolgus monkey studies.
2025 Nature Communications study characterized TfR and CD98hc antibody transport vehicle biodistribution; 2026 author correction was published.
TfR and CD98hc antibody transport vehicles achieved enhanced brain exposure and parenchymal distribution compared with control IgG and showed distinct whole-body, cellular, and regional CNS biodistribution patterns.
Investigate TREM2 signaling and TREM2 agonism as a way to modulate microglial state, metabolism, and function in Alzheimer's disease-relevant biology.
TREM2 reporter mouse studies, APP transgenic animal microglial profiling, metabolic and lipidomic profiling, phagocytic capacity analysis, and chronic treatment with a TREM2 agonist antibody.
2026 Nature Communications publication on TREM2 expression level and efficacy of TREM2 agonism.
TREM2 expression correlated with microglial immunometabolic pathways, redox and energetics signatures, cholesterol homeostasis, and phagocytic capacity; chronic TREM2 agonist antibody treatment identified a TREM2 expression window with greatest microglial responsiveness.