Clinical pipeline
From hypothesis to clinical evidence
GeNeuro's pipeline spans five disease areas — Multiple Sclerosis, Psychosis, ALS, Type 1 Diabetes, and Long Covid Brain Fog — with programs ranging from preclinical discovery to Phase II clinical trials. Each program is anchored in the same core HERV biology, validated by decades of academic research and our own clinical data.
5
Disease areas
4
Phase II programs
1
Phase I program
Pipeline overview
Development stages
ALS
HERV-K Env
Multiple Sclerosis
HERV-W Env
Type 1 Diabetes
HERV-W Env
Long Covid — Brain Fog
HERV-W Env
Psychosis
HERV-W Env
Program details
Each program, in depth
ALS
GNK-301
Target: HERV-K Env
Overview
HERV-K envelope protein is demonstrated to cause motor neuron degeneration in animal subjects and in human motor neurons in culture. Under a Cooperative Research and Development Agreement (CRADA) with the NIH/NINDS, GeNeuro's antibody GNK-301 was shown to prevent motor neuron degeneration in mouse brains and in human brain cells in culture — establishing proof of concept for HERV-K targeted therapy in ALS.
Key highlights
- HERV-K Env demonstrated to cause motor neuron degeneration in vivo and in vitro
- GNK-301 prevents motor neuron degeneration in mouse brains and human neuronal cultures
- Developed under CRADA with the NIH/NINDS (Avindra Nath, MD)
- Human-grade GNK-301 now in production; First in Human expected July 2027
Multiple Sclerosis
Temelimab (GNbAC1)
Target: HERV-W Env
Overview
Temelimab is a humanized monoclonal antibody that neutralizes the HERV-W envelope protein. In MS patients, HERV-W Env drives microglial activation and myelin destruction. Phase II data demonstrate significant reductions in neuroinflammatory markers and early neuroprotective signals.
Key highlights
- Phase IIb CHANGE-MS trial completed
- Significant reduction in cortical lesion volume
- Favorable safety and tolerability profile
- Neuroprotective effect on grey matter
Type 1 Diabetes
GNbAC1
Target: HERV-W Env
Overview
HERV-W Env protein triggers autoimmune destruction of pancreatic beta cells. Preclinical studies demonstrate that neutralizing HERV-W Env reduces insulitis and preserves beta cell mass, positioning temelimab as a potential disease-modifying therapy for T1D.
Key highlights
- HERV-W Env detected in T1D patient samples
- Beta cell preservation shown in preclinical models
- IND-enabling studies in planning
Long Covid — Brain Fog
GNbAC1
Target: HERV-W Env
Overview
SARS-CoV-2 infection is a potent trigger for HERV-W reactivation. In Long Covid patients, sustained HERV-W Env expression drives persistent neuroinflammation manifesting as cognitive impairment and memory loss. Phase II results demonstrate a statistically significant reduction in memory alterations with temelimab compared to placebo.
Key highlights
- Phase II trial completed
- Statistically significant reduction in memory alterations vs. placebo
- SARS-CoV-2 confirmed as HERV-W reactivation trigger
- Neuroinflammatory mechanism consistent with MS pathway
Psychosis
Temelimab (GNbAC1)
Target: HERV-W Env
Overview
HERV-W Env expression is elevated in the brains of first-episode psychosis and schizophrenia patients and correlates with inflammatory markers. A Phase I study demonstrated that temelimab is safe and well-tolerated in this population, supporting further clinical development.
Key highlights
- Phase I safety study completed
- Well-tolerated in psychosis patients
- HERV-W Env confirmed as biomarker
Learn more
The science behind every program
Each program is grounded in GeNeuro's HERV hypothesis — decades of peer-reviewed research linking endogenous retroviral proteins to the onset and progression of serious disease.