Future Targets

Retrotope believes that its proprietary approach to down-regulating lipid peroxidation (LPO) through the administration of its novel, isotopically stabilized PUFAs has broad application across a range of degenerative diseases.  As such, the company has identified multiple additional indications for which it may pursue programs to supplement its current pipeline.  Indications of particular interest include:

Huntington’s disease (HD):

HD is a neurodegenerative disease that is most often inherited from a parent who has the disease-causing genetic mutation.  The disease is progressive with initial symptoms typically involving subtle challenges with mood and mental capacity.  Over time, HD causes deterioration of an individual’s physical, mental and emotional abilities.  The disease typically strikes people between the ages of 30 and 50, though there is a juvenile form of the disease (juvenile HD) that can impact children and young adults.

It is estimated that there are approximately 41,000 Americans living with symptomatic HD, and an additional 200,000 who are at-risk of inheriting the disease.  While there is currently no cure or treatment capable of slowing or reversing the progression of the disease, there are treatments and interventions that can help to manage HD symptoms.

 Spinocerebellar ataxia (SCA):

SCA refers to a group of hereditary, progressive, neurodegenerative ataxias caused by degeneration of the portions of the brain responsible for movement control.  While there are many different types of SCA, each caused by a different genetic mutation, all are characterized by challenges with movement that progressively worsen.  The most commonly seen SCA symptoms include uncoordinated walk (gait), poor hand-eye coordination, and abnormal speech (dysarthria).

It is estimated that approximately 150,000 Americans are living with SCA at any given time and it can affect people of any age.  The disease is often fatal and there is currently no cure of treatment for the disease.  Current therapeutic approaches are aimed at managing symptoms of the various forms of SCA.

Multiple system atrophy (MSA):

MSA is a rare, neurodegenerative disease that affects involuntary biological functions such as breathing, balance/coordination, blood pressure, bladder function and motor control.  Previously referred to as Shy-Drager syndrome, The disease results in the deterioration of the portions of the brain responsible for regulating these autonomic body functions.  MSA results in symptoms that are similar to those seen in patients with Parkinson’s disease, including slow movement, rigid muscles and poor balance.  These symptoms typically present in adulthood, often between the ages of 50 and 70.

MSA is estimated to affect approximately 14,000 Americans with an average of 1,900 new cases diagnosed in the U.S. each year.  There is currently no cure or treatment for MSA and current therapeutic approaches are designed to manage the symptoms of the disease.