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. 

Interdisciplinary Innovation

in Neurological Disease Drug Development

Precision Medicine
Precision Medicine

Genetically defined apoE genotype-associated neuropathology and abnormal lipid metabolism in neurodevelopment

Regenerative Medicine
Regenerative Medicine

Human iPSC-derived GABAergic inhibitory neuron transplantation to replace/restore lost neurons

Pharmaceutical Intervention
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.

Area 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.

Focus Area

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

 

 

The GABAeron Team

 

Sr Management & Advisory Board

Robert Mahley, MD, PhD
Robert Mahley, MD, PhD

Chief Executive Officer & Chief Scientific Officer

Qin Xu, PhD
Qin Xu, PhD

Senior Director of Technology / Discovery

Yadong Huang, MD, PhD
Yadong Huang, MD, PhD

Scientific Co-Founder

Sheng Ding, PhD
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.