This biopharmaceutical company develops therapies for neurological diseases using stem cells and small molecules. Their approach combines precision medicine, using genetic information for patient stratification, with regenerative medicine, using human iPSC-derived cell products, to restore and preserve brain functions.
Funding
$3M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Current treatments for neurodegenerative diseases like Alzheimer's primarily address symptoms, failing to restore lost neuronal function. The loss of GABAergic interneurons, particularly in the hippocampus, contributes significantly to cognitive decline and network hyperactivity in these conditions. Addressing the underlying neuronal loss and dysfunction is critical for developing disease-modifying therapies.
Solution
GABAeron is developing cell replacement therapies using human induced pluripotent stem cell (iPSC)-derived GABAergic interneurons to restore neuronal function in patients with neurodegenerative and neurodevelopmental disorders. Their approach focuses on replacing lost or injured neurons in the hippocampus, particularly in individuals carrying the apoE4 allele, a major genetic risk factor for Alzheimer's disease. By transplanting these iPSC-derived neurons, GABAeron aims to re-establish inhibitory neurotransmission, reduce network hyperactivity, and improve cognitive function. The company is also exploring small molecule interventions to modulate sterol biology and correct apoE4-related neuropathology.
Target Audience
The primary target population includes patients with Alzheimer's disease and other neurodegenerative disorders, particularly those carrying the apoE4 allele, as well as individuals with Down's Syndrome and Epilepsy.
Features
- Development of human iPSC-derived GABAergic interneuron progenitors for transplantation.
- Focus on apoE4 carriers, a genetically defined subpopulation at high risk for Alzheimer's disease.
- Preclinical studies demonstrating restoration of learning and memory in apoE4 knock-in mice following transplantation of GABAergic progenitors.
- Small molecule programs targeting apoE4 structure and function to prevent neurotoxic fragment generation.
- In vitro assays using human iPSC-derived neurons for screening and validating therapeutic candidates.
- Pipeline targeting Alzheimer's disease, Down's Syndrome, and Epilepsy.