A late Stage
and Broad Pipeline
EryDex provides the potential for a safe and effective treatment in a previously untreatable disease, with the potential for disease-modifying properties. Other benefits include: slow release, infrequent dosing, potential avoidance of systemic side effects, and out-patient treatment. EryDex is a novel, proprietary method of encapsulating a potent steroid, dexamethasone sodium phosphate, in red blood cells allowing for its gradual dephosphorylation and release of dexamethasone in a patient’s circulation for up to 30 days, providing the potential for effective treatment and preventing the unacceptable systemic side effects of oral or iv steroid treatment.
Ataxia telangiectasia (AT) is a rare, inherited genetic life-threatening condition for which no effective treatment exists. AT is primarily characterized by cerebellar degeneration, telangiectasia (spider veins), immunodeficiency, cancer susceptibility and radiation sensitivity. Progression of neurodegenerative disease dramatically affects quality of life. The classic form of AT is the severe form, which is usually first detected when patients present with wobbly (ataxic) gait starting at approximately age 2-3 years with frequent falls. In primary school years, walking becomes more difficult and around the beginning of their second decade patients start using a wheelchair. They also are affected by immunological abnormalities leading to immunodeficiency, with frequent infections and increased risk to develop malignancies. Pulmonary failure and cancer are the usual causes of death during the second or third decade of life.
A pivotal Phase 3 study is currently ongoing with EryDex for the treatment of AT patients (clinical trials.gov NCT02770807).
A Novel Approach to Treat Enzyme Deficiency Disorders
- EryDel’s Technology Platform also enables proteins to be encapsulated using the patient’s own red blood cells (RBCs) introducing the potential for a new treatment approach for rare enzyme deficiency disorders – an area of high unmet need.
- Current therapies used to treat enzyme deficiencies often provoke an immune response in the body rendering the enzyme less effective and causing serious side effects
- In contrast, EryDel’s approach circulates the enzyme in the patient’s own RBCs and is expected to protect the enzyme from an immune response while allowing the enzyme to exert its intended therapeutic effect.
- This novel approach has the potential to revolutionize enzyme replacement therapy (ERT).
- EryDel scientists are focused on ERT development programs with the high unmet needs
EE-TP (Mitochondrial NeuroGastrointestinal Encephalomyopathy - MNGIE)
Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) is a rare autosomal recessive inherited disease caused by defects in a gene responsible for the production of thymidine phosphorylase (TP). TP deficiency leads to plasma and tissue accumulation of thymidine and deoxyuridine leading to mitochondrial dysfunction. MNGIE affects mainly the digestive and nervous systems. Our technology is being used to develop and commercialize the RBC encapsulated TP enzyme for the treatment of MNGIE through a partnership with Orphan Technologies.
Phenylketonuria (PKU) is a genetic disorder that increases the levels of a substance called phenylalanine in the blood because of a lack or deficiency of an enzyme (phenylalanine hydroxylase or PAH). If the gene responsible for providing PAH is not functioning due to a mutation, phenylalanine can build up to harmful levels in the body, causing a wide array of symptoms for the affected person, which can range from mild to severe. Ery-PAL consists of recombinant phenylalanine ammonia lyase encapsulated in patient’s erythrocytes for the treatment of PKU.
Ery-uricase (Refractory Gout)
Ery-uricase consists of recombinant uricase enzyme encapsulated in patient’s erythrocytes for the treatment of refractory gout. Refractory gout refers to those patients who have chronic symptoms of active disease despite the use of maximized treatment. People with refractory gout cannot maintain target serum urate levels. Living with refractory gout reduces quality of life, impairs functionality and destroys joints. Refractory gout is characterized by abnormally high serum uric acid levels or continuous manifestations of recurrent flares, chronic arthritis and increased tophi (a deposit of crystalline uric acid and other substances at the surface of joints or in skin or cartilage). Refractory gout continues to be characterized by suboptimal treatment resulting in high morbidity and unacceptable societal costs. Studies have demonstrated that gout patients are at a significantly higher risk for both myocardial infarction and cardiovascular mortality compared with those without gout, a risk not observed among individuals with asymptomatic hyperuricemia.
Ery-GAMT (Guanidino methyltransferase (GAMT) deficiency)
Among cerebral creatine deficiency syndromes, GAMT deficiency can present the most severe symptoms, and is characterized by neurocognitive dysfunction due to creatine deficiency and accumulation of guanidinoacetate in the brain.
GAMT deficiency is an autosomal recessive inherited metabolic disease. The central nervous system is mainly tissue affected, with patients developing neurological symptoms in infancy, in particular intellectual disability/developmental delay and speech acquisition defects. Guanidinoacetate (GAA) accumulation upstream of the GAMT enzymatic block is thought to cause these severe phenotypes ranging from intractable epilepsies to autistic and automutilating behaviors as well as extrapyramidal syndrome.