HERVS are likely to play a major role in a variety of other neurodegenerative and autoimmune diseases. 

Type 1 Diabetes

GeNeuro has conducted a clinical trial in Type 1 Diabetes, showing safety of its approach on an adult population of patients who were insulin-dependent, and observing positive effects on pharmacodynamic markers such as a reduction in the number of hypoglycemic episodes. This trial opens the door for further exploration of this approach in a pediatric population with recent onset, where stopping the degeneration of insulin-producing cells is still a critical unmet medical need.

Type 1 Diabetes (T1D) is a chronic disease resulting from the autoimmune destruction of the insulin-producing beta cells in the pancreas. As a consequence, the pancreas is no longer able to produce insulin in response to increasing glucose levels in blood and T1D patients become hyperglycemic. Lifelong insulin replacement therapy is the only option for these patients to control their glycemia, as there is currently no cure for T1D. Despite improvements in insulin replacement therapy in the past decades, normoglycemia is rarely achieved by T1D patients. The absence of proper glycemia control is associated with mortality and severe complications, including diabetic retinopathy, nephropathy, foot ulceration and diabetic neuropathy.

GeNeuro has shown, using immunodetection methods, that pHERV-W-Env protein is present in the pancreas of T1D patients. Immune cells of these patients also express pHERV-W-Env mRNA. Transgenic mice expressing pHERV-W-env have been shown to display abnormalities in glucose regulation, including hyperglycemia and reduced levels of insulin. In vitro experiments on human pancreatic beta cells have demonstrated a toxic effect of pHERV-W-Env, which inhibits insulin secretion in response to glucose. This pathogenic effect can be blocked with temelimab. Overall, GeNeuro has shown (1) significant expression of pHERV-W-Env in T1D patients, (2) the pathogenic effect of pHERV-W-Env in diabetic models, and (3) the positive effect of temelimab on diabetic models.

Inflammatory Psychosis

GeNeuro has also collaborated with a pre-eminent consortium of academic institutes, under the auspices of Fondation FondaMental, establishing a clear link between HERV proteins and psychotic disorders. These results open a new path for treating inflammatory psychosis by developing a drug candidate that neutralizes causal factors arising from human endogenous retroviruses.

Chronic Inflammatory Demyelinating Polyneuropathy- CIDP

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare autoimmune disorder of the peripheral nervous system (PNS). CIDP is related to multifocal inflammation and demyelinating lesions of the proximal PNS. Its clinical presentation is heterogeneous and its diagnosis is challenging because of its unknown etiology and the lack of specific biomarkers. Existing CIDP therapies are intravenous human immunoglobulins (IVIG), corticosteroids and plasma exchange. Long-term therapy is often limited by side effects and one-third of patients are refractory to existing treatments. This illustrates a critical unmet medical need for new treatments of CIDP and diagnostic biomarkers in this indication. Several studies have confirmed that pHERV-W-Env is found in half of CIDP patients and that this protein is expressed in Schwann cells in CIDP lesions. The effects of pHERV-W Env- expression were studied in vitro on cultured human Schwann cells. The cells expressing pHERV-W Env presented a strong and significant increase in proinflammatory cytokines transcript levels, such as IL-6 and CXCL10.

In February 2018, GeNeuro received Orphan Drug Designation for temelimab by the US-FDA.