Restoring Neurons. Rebuilding Lives.
We build on the promise of combining precision medicine with regenerative medicine and pharmaceutical intervention – building towards a new first-in-class IND candidate for neuron replacement in patients with Alzheimer’s disease and other neurological disorders and for the protection by small molecule drugs in the neurodevelopmental disorder Smith-Lemli-Opitz syndrome.
Interdisciplinary Innovation
in Neurological Disease Drug Development
Precision Medicine
Genetically defined apoE genotype-associated neuropathology and abnormal lipid metabolism in neurodevelopment
Regenerative Medicine
Human iPSC-derived GABAergic inhibitory neuron transplantation to replace/restore lost neurons
Pharmaceutical Intervention
Small molecules designed to modulate sterol (cholesterol) biology in aberrant neurodevelopmental disorder
Transplanted human neurons reside in the hippocampus of an apoE4 mouse brain.
Areas of Research
Causative Role of ApoE4 in Neuropathology
ApoE4 is the strongest genetic risk factor for many neurodegenerative diseases, including Alzheimer’s disease (AD).
Individuals carry one of three variants of the apoE gene. Carriers with the apoE4 allele have an increased risk of developing AD at an earlier age and with an increased rate of progression. GABAeron aims to treat apoE4 carriers with neurodegenerative diseases with human iPSC-derived GABAergic inhibitory interneurons to restore lost/injured hippocampal neurons as a novel first-in-class treatment.
Modulation of Abnormal Sterol (Cholesterol) Biosynthesis in Neurodevelopment
Enzyme mutations in the sterol biosynthetic pathway block normal cholesterol production, cause a buildup of neurotoxic intermediates that could impair neurodevelopment, and are associated with a number of physical developmental defects. GABAeron has identified several small molecules that can modulate key steps in the sterol biosynthetic pathway and could be useful, especially in Smith-Lemli-Opitz syndrome.
Focus Areas
Alzheimer’s Disease
& Other Neurodegenerative Diseases
A prominent feature of Alzheimer’s disease (AD) is impaired GABAergic inhibitory interneuron function and an associated network hyperactivity. The loss of both the neurotransmitter GABA and the GABAergic interneurons is associated with neurological disorders, including AD and epilepsy, and psychiatric disorders, including schizophrenia and depression.
GABAergic inhibitory interneurons in the hippocampus are highly vulnerable to apoE4-mediated toxicity. Previous studies in mice have demonstrated that the transplantation of GABAergic progenitors into the hippocampus restored the impaired learning and memory in apoE4 mice. The goal of GABAeron is to use cell replacement as a treatment for neurological disorders by selectively producing human iPSC-derived GABAergic interneurons and transplanting them into the dentate gyrus of the hippocampus
Smith-Lemli-Opitz Syndrome (SLOS)
The Smith-Lemli-Opitz syndrome (SLOS) is a genetic disorder (autosomal recessive) resulting in defective cholesterol synthesis and is associated with abnormalities in neurodevelopment, cognition, and behavior along with various physical malformations. GABAeron scientists have identified small molecules that modulate the activity of several enzymes in the cholesterol biosynthesis pathway and may correct the defective cholesterol biosynthesis in cultured neurons and other cells from patients with SLOS. The goal is to develop a drug that would both reduce the 7-dehydrocholesterol and increase cholesterol.
The GABAeron Team
Sr Management & Advisory Board
Robert Mahley, MD, PhD
Chief Executive Officer & Chief Scientific Officer
Qin Xu, PhD
Senior Director of Technology / Discovery
Yadong Huang, MD, PhD
Scientific Co-Founder
Sheng Ding, PhD
Scientific Co-Founder
Careers at GABAeron
We are looking for colleagues who are team players, always learning, dedicated to conducting rigorous science, and passionate about improving the lives of patients with neurodegenerative and neurodevelopmental disorders. GABAeron has positions open for those looking to join a focused organization with opportunities to guide early lab discoveries towards meaningful disease-modifying therapeutics.
