Our technology

Programmed death-ligand 1 (PD-L1) is a checkpoint molecule involved in the control of reactive T cells. Harnessing the power of the immune-regulatory protein PD-L1 can lead, on one hand, to overcome the onset of autoimmune reactions and to promote immune tolerance, and on the other hand, to significantly impact cancer growth.

 

PD-L1 and autoimmune disorders

Hematopoietic stem cells of patients with autoimmune diseases, including type 1 diabetes, show a decreased expression of PD-L1. The infusion of autologous stem cells pharmacologically or genetically modulated to efficiently express PD-L1 successfully reverse type 1 diabetes in the non-obese diabetic (NOD) mouse model, achieving not only the control of glucose blood levels but also the normalization of pancreatic histology and function.
The use of the same technology in investigational mouse models of multiple sclerosis is demonstrating clinical benefits suggesting that direct delivery of the engineered hematopoietic stem cells into the central nervous system will enhance the benefit of the cell transplant approach potentially reverting the auto-immune reaction causing onset, relapse and progression of the disease.

Programmed death-ligand 1 (PD-L1) is a checkpoint molecule involved in the control of reactive T cells. Harnessing the power of the immune-regulatory protein PD-L1 can lead, on one hand, to overcome the onset of autoimmune reactions and to promote immune tolerance, and on the other hand, to significantly impact cancer growth.

 

PD-L1 and autoimmune disorders

Hematopoietic stem cells of patients with autoimmune diseases, including type 1 diabetes, show a decreased expression of PD-L1. The infusion of autologous stem cells pharmacologically or genetically modulated to efficiently express PD-L1 successfully reverse type 1 diabetes in the non-obese diabetic (NOD) mouse model, achieving not only the control of glucose blood levels but also the normalization of pancreatic histology and function.
The use of the same technology in investigational mouse models of multiple sclerosis is demonstrating clinical benefits suggesting that direct delivery of the engineered hematopoietic stem cells into the central nervous system will enhance the benefit of the cell transplant approach potentially reverting the auto-immune reaction causing onset, relapse and progression of the disease.

Type 1 diabetes

Type 1 diabetes (T1D) represents one of the most aggressive autoimmune disorders in children, characterized by selective and aggressive destruction by autoreactive T cells of insulin-producing β-cells in the pancreas. T1D prevalence is about 10% with more than 20 million patients worldwide. Between 2001 and 2009, there was a 21% increase in the prevalence of T1D in people under age 20 in the US. Moreover, data from large epidemiologic studies worldwide indicate that the incidence of T1D has been increasing by 2–5% worldwide. Complications of poorly managed T1D can severely affect many organs, including heart and circulation, kidney, eye, gastrointestinal tract, central and peripheral nervous system. In addition, sexual dysfunction in diabetics is often a result of physical factors such as nerve damage and poor circulation, and psychological factors such as stress and/or depression caused by the demands of the disease. Injections of insulin – either via subcutaneous injection or insulin pump – are necessary for those living with T1D because it cannot be treated by diet and exercise alone. Insulin dosage is adjusted taking into account food intake, blood glucose levels and physical activity. Actual treatments of diabetes focus on lowering blood sugar or glucose to the near normal range, with an ultimate goal of normalizing glucose being to avoid long-term complications. About 70% of patients achieve a sub-optimal glucose level control. Altheia Science’s novel approach aims to restore a correct expression of PD-L1 on hematopoietic stem cells, enabling the eradication of auto-reactive T cells responsible for the destruction of pancreatic β-cells.

Type 1 diabetes

Type 1 diabetes (T1D) represents one of the most aggressive autoimmune disorders in children, characterized by selective and aggressive destruction by autoreactive T cells of insulin-producing β-cells in the pancreas. T1D prevalence is about 10% with more than 20 million patients worldwide. Between 2001 and 2009, there was a 21% increase in the prevalence of T1D in people under age 20 in the US. Moreover, data from large epidemiologic studies worldwide indicate that the incidence of T1D has been increasing by 2–5% worldwide. Complications of poorly managed T1D can severely affect many organs, including heart and circulation, kidney, eye, gastrointestinal tract, central and peripheral nervous system. In addition, sexual dysfunction in diabetics is often a result of physical factors such as nerve damage and poor circulation, and psychological factors such as stress and/or depression caused by the demands of the disease. Injections of insulin – either via subcutaneous injection or insulin pump – are necessary for those living with T1D because it cannot be treated by diet and exercise alone. Insulin dosage is adjusted taking into account food intake, blood glucose levels and physical activity. Actual treatments of diabetes focus on lowering blood sugar or glucose to the near normal range, with an ultimate goal of normalizing glucose being to avoid long-term complications. About 70% of patients achieve a sub-optimal glucose level control. Altheia Science’s novel approach aims to restore a correct expression of PD-L1 on hematopoietic stem cells, enabling the eradication of auto-reactive T cells responsible for the destruction of pancreatic β-cells.

Multiple sclerosis

Multiple Sclerosis (MS) is a severely debilitating autoimmune disease caused by the degeneration and disruption of the myelin sheaths that surround axons of the brain and spinal cord. Myelin loss is associated to neural inflammation, axonal loss and formation of scar tissue in the white matter, leading to a wide spectrum of neurological symptoms, including fatigue, spasticity, functional loss, and pain.
It has been estimated that about 2.5 million of people are living with MS worldwide, with an estimated 10’000 new cases diagnosed in the US every year. In the majority of cases, the diagnosis occurs between 20 and 50 years of age. The most common symptoms include fatigue, visual disturbances, altered sensation and difficulties with mobility.
Treatments for MS mainly target the management of symptoms and attempt to interrupt the damage to myelin sheets. These treatments mostly consist of anti-inflammatory or immuno-modulatory drugs that, given the chronic nature of the disease, expose the patient to serious side effects, with significant impact on their quality of life.
Clinical patterns of appearance and progression of symptoms significantly vary in MS: the most common form of MS is the relapsing-remitting form (RRMS), characterized by episodes of acute worsening of neurologic deficits, including new symptoms and/or the worsening of pre-existing symptoms, followed by periods of total or partial recovery (remissions).
Infusion of autologous hematopoietic stem cells where the expression of PD-L1 is restored could abort the autoimmune mechanism at the basis of myelin disruption providing a curative treatment to MS patients.

 

Cancer

Cancer is a multi-factorial disease caused by a series of genetic alterations and immune-escape mechanisms resulting in a non-regulated cell growth and proliferation. Tumors have developed multiple immunosuppressive mechanisms to turn down the innate and the effector arms of the immune system, thus compromising most of the immunotherapeutic strategies that have been proposed during the last decade. Programmed cell death-1 (PD-1) plays a major role in tumor immune escape, and the PD-1/PD-L1 interaction enables the tumor to escape the immune response. Targeted modulation of PD-L1 expression might restore an effective immune surveillance of tumors by re-establishing the function of exhausted tumor-specific CD8+ T cells.

Multiple sclerosis

Multiple Sclerosis (MS) is a severely debilitating autoimmune disease caused by the degeneration and disruption of the myelin sheaths that surround axons of the brain and spinal cord. Myelin loss is associated to neural inflammation, axonal loss and formation of scar tissue in the white matter, leading to a wide spectrum of neurological symptoms, including fatigue, spasticity, functional loss, and pain.
It has been estimated that about 2.5 million of people are living with MS worldwide, with an estimated 10’000 new cases diagnosed in the US every year. In the majority of cases, the diagnosis occurs between 20 and 50 years of age. The most common symptoms include fatigue, visual disturbances, altered sensation and difficulties with mobility.
Treatments for MS mainly target the management of symptoms and attempt to interrupt the damage to myelin sheets. These treatments mostly consist of anti-inflammatory or immuno-modulatory drugs that, given the chronic nature of the disease, expose the patient to serious side effects, with significant impact on their quality of life.
Clinical patterns of appearance and progression of symptoms significantly vary in MS: the most common form of MS is the relapsing-remitting form (RRMS), characterized by episodes of acute worsening of neurologic deficits, including new symptoms and/or the worsening of pre-existing symptoms, followed by periods of total or partial recovery (remissions).
Infusion of autologous hematopoietic stem cells where the expression of PD-L1 is restored could abort the autoimmune mechanism at the basis of myelin disruption providing a curative treatment to MS patients.

Cancer

Cancer is a multi-factorial disease caused by a series of genetic alterations and immune-escape mechanisms resulting in a non-regulated cell growth and proliferation. Tumors have developed multiple immunosuppressive mechanisms to turn down the innate and the effector arms of the immune system, thus compromising most of the immunotherapeutic strategies that have been proposed during the last decade. Programmed cell death-1 (PD-1) plays a major role in tumor immune escape, and the PD-1/PD-L1 interaction enables the tumor to escape the immune response. Targeted modulation of PD-L1 expression might restore an effective immune surveillance of tumors by re-establishing the function of exhausted tumor-specific CD8+ T cells.