Therapeutic Areas Overview

iPierian is focused on diseases which have poor in vivo and in vitro models and limited therapeutic treatments to date. iPierian’s proprietary programs are currently centered on three neurological diseases – Alzheimer’s disease, amyotrophic lateral sclerosis, and spinal muscular atrophy. iPierian is generating iPS cells from patients with each of these diseases, differentiating into the disease-relevant cell types, and identifying disease phenotypes. These disease models are being used to identify disease mechanisms and propel drug discovery and development programs. Because disease models are generated directly from the patient, drugs discovered using these models are more likely to translate into efficacious treatments in the clinic.

Alzheimer’s Disease
Alzheimer’s disease (AD) is the most common neurodegenerative disease, affecting five million people in the U.S. alone.  There is no effective intervention to slow, halt or reverse disease progression.  AD is a fatal neurodegenerative disease with progressive memory loss and dementia. There is a progressive loss of neurons in the cortex and hippocampus.  Most AD patients suffer from sporadic disease although known genetic factors contribute to disease in as many as 25-40% of patients.  The most prevalent late onset susceptibility gene, ApoE4, increases the risk of developing AD 6 to 30 fold.  Additionally, several monogenic AD mutations cause AD in < 1% of patients. These monogenic mutations cause early onset AD but faithfully recapitulate most clinical and neuropathological features of late onset sporadic AD.

Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is an adult-onset progressive motor neuron disease that affects approximately 1 in every 50,000 people. Approximately half of all patients die within 3 years of symptom onset, and most of the remaining patients within 5 years. There are, however, significant variations in disease onset, progression, and clinical pathology in patients, making ALS a challenging target for disease modeling and drug discovery.  Thus far only one drug has been approved that may slow the progression of the disease. Although 90% of ALS cases are sporadic, familial forms of ALS have been linked to mutations in a small number of genes.  The primary cellular pathology in ALS is the death of spinal and upper motor neurons with a common pathological feature being proteinaceous aggregates in sporadic and several familial forms of ALS.

Spinal Muscular Atrophy
Spinal muscular atrophy (SMA), a recessive monogenic disease, is a common fatal pediatric genetic disease that affects one in 7,000 newborns. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, causing a deficiency of the survival motor neuron (SMN) protein. The SMN protein is critical to the survival and health of motor neurons, and without this protein, nerve cells may atrophy, shrink and eventually die, resulting in muscle weakness. The mechanisms linking SMN deficiency with dysfunction and loss of functioning motor units are not well understood. Quality of life and survival of SMA patients are improved with aggressive supportive care including optimized respiratory and nutritional care and management of scoliosis and contractures. Several therapeutic compounds for the treatment of SMA are currently being studied but no therapeutic treatment options exist for patients today.