Mechanism-First Drug Discovery for Neurological Disease
We start with human genetics and validated biology, then engineer precision RNA therapeutics and medical devices to intervene at the root cause of disease.
Genetic Therapeutics Pipeline
Our RNA therapeutic programs are built on allele-selective targeting, silencing disease-causing mutations while preserving normal gene function. Each program is designed for IND-enabling execution at $250K, leveraging our Baja Bio computational design engine.
Genetic Targeting
We design therapeutics that directly target the genetics of disease. By addressing root cause rather than downstream symptoms, our programs offer the potential for disease modification rather than management.
Computational Design Engine
Our Baja Bio platform screens thousands of candidate sequences in silico, delivering 20+ optimized compounds per year. This compresses the hit-to-lead timeline from years to months.
IND-Enabling Execution
We run lean, milestone-driven programs designed to reach IND filing for $250K by leveraging established CRO networks and academic partnerships.
Therapeutic Programs
ALS, Various targets for genetic and sporadic forms
Building on the development of BB-fALS, designed and delivered to the clinic by the Baja Bio founding team in under seven months, Baja Bio is pursuing multiple new allele-selective programs targeting the genetic and sporadic drivers of ALS with the same speed and precision.
Tauopathy (Alzheimer’s / FTD)
BBI-TAU targets pathological tau isoforms implicated in Alzheimer’s disease and frontotemporal dementia. Our allele-selective approach aims to reduce toxic tau aggregation while preserving normal microtubule-associated tau function.
Hereditary Sensory Neuropathy (HSN1) & Pediatric ALS
Dominant gain-of-function mutations in SPTLC2 cause hereditary sensory neuropathy type 1 by shifting sphingolipid synthesis toward neurotoxic deoxysphingolipids. BBI-SPT selectively silences the mutant allele to restore normal sphingolipid homeostasis.
Brain Cancers (e.g. Oligodendroglioma & other CNS tumors)
A portfolio of programs targeting the genetic drivers of CNS tumors, including IDH1/IDH2 mutations and 1p/19q co-deletions in oligodendroglioma, and EGFR amplification, PTEN loss, and TERT promoter mutations in glioblastoma.
Biomarker Diagnostics Pipeline
Baja Bio is developing blood-based biomarker algorithms for neurological disease detection, enabling earlier diagnosis and patient stratification to support both clinical care and therapeutic development.
ALS-SLI™
ALS Serum Lipid IndexALS-SLI™ is an experimental blood test being developed to help detect ALS (Lou Gehrig’s disease) — a serious illness that is currently hard to diagnose and often takes a year or more to confirm. The test looks at a small set of fats in the blood (a type called sphingolipids) that tend to be higher in people with ALS. It measures four of these specific fats and combines them into a single score that estimates how likely it is that a blood sample came from someone with ALS, sorted into low, medium, or high likelihood. The goal is a simple, low-cost blood test that could help doctors identify ALS earlier and more easily than today’s methods. In repeated cross-validation on the derivation cohort, the algorithm achieves AUC 0.84 ± 0.09.
Patent Pending — U.S. Patent Application No. 64/128,748
Neurological Medical Devices
Alongside our therapeutic programs, Baja Bio is developing medical devices that address critical unmet needs in drug preparation and CNS delivery, designed to work in concert with our therapeutic pipeline.
BajaSonora™
Sterile Compounding · Pharmacy Compounding for Intrathecal AdministrationAn automated sterile compounding and syringe-filling platform for pharmacy preparation of intrathecal therapeutics, enabling repeatable, pharmacy-grade syringe preparation from lyophilized GMP drug substance.
Intrathecal Delivery Device
Drug Delivery · Pediatric CNS Drug DeliveryA minimally invasive intrathecal delivery device designed specifically for pediatric patients. The device enables precise CNS delivery of oligonucleotide therapeutics with a form factor optimized for the anatomical and clinical constraints of pediatric neurology.
Our Device Strategy
Pharmacy-Ready Automation
BajaSonora™ bridges the gap between GMP drug substance and patient-ready syringes, automating the compounding workflow with validated controls for concentration accuracy, fill weight, sterile fluid-path integrity, and aseptic compatibility.
Minimally Invasive Pediatric Access
Our intrathecal delivery device is purpose-built for pediatric patients, where standard adult delivery approaches are often unsuitable. The design prioritizes minimal invasiveness, procedural simplicity, and safe CNS access.
Pipeline Integration
Both devices are designed to complement our therapeutic programs, BajaSonora™ supports the clinical preparation of our RNA therapeutics, while the intrathecal device enables direct CNS delivery for our pediatric ALS and HSN1 programs.
Partner With Us
We are actively seeking research collaborations, licensing partners, and co-development opportunities across our therapeutics and device pipeline.