Purposeful discovers new treatments and develops pharmaceutical products for rare diseases, by repurposing already marketed drugs. Our vision is to fully employ AI repurposing and revisit rare and common diseases to reverse disease phenotypes.
Rare Diseases
Over 7,000 rare diseases have been identified, 80% of which are genetic. They can affect people at different times in their lives, affect a single system of the body, or display symptoms across multiple organs. Due to this, rare diseases are rarely considered as a group by the medical profession, meaning there are no rare disease specialists. Instead, each condition falls under the care of one or more different specialities.
Fragile X Syndrome
Fragile X syndrome (FXS) is a genetic condition that causes intellectual disability, behavioral and learning challenges, and various physical characteristics. Though FXS occurs in both genders, males are more frequently affected than females, and generally with greater severity. Life expectancy is not affected in people with FXS because there are usually no life-threatening health concerns associated with the condition.
Glioblastoma
A fast-growing type of central nervous system tumor that forms from glial (supportive) tissue of the brain and spinal cord and has cells that look very different from normal cells. Initial signs may include headaches, personality changes, nausea, and symptoms similar to those of a stroke. Symptoms often worsen rapidly and may progress to unconsciousness. About 3 in 100,000 people develop the disease per year.
Psoriasis
Psoriasis is a skin disorder that causes skin cells to multiply up to 10 times faster than normal. This makes the skin build up into bumpy red patches covered with white scales. They can grow anywhere, but most appear on the scalp, elbows, knees, and lower back. Psoriasis can't be passed from person to person.
Duchenne Muscular Dystrophy
Duchenne and Becker MD are rare genetic diseases defined by muscle weakness. Muscles are progressively getting weaker over time and this ultimately affects the ability to breathe as well as the function of the heart, as the heart is a muscle too.
Huntington’s disease
Huntington’s disease is a fatal neurodegenerative genetic disorder that causes the progressive breakdown of nerve cells in the brain. The disease is mostly inherited, with first symptoms usually appearing during their prime working years but can also occur in children and young adults. The worldwide prevalence of HD is 5 to 10 cases per 100,000 persons, but varies greatly geographically.
Acrodysostosis
Acrodysostosis is a rare genetic condition characterized by skeletal malformations, growth delays, short stature and distinctive facial features. A characteristic symptom is unusually small hands and feet with short, stubby fingers and toes.
Partnerships
Collaborations to date
Fraxa & Fraxa-DVI
Purposeful partners with FRAXA and FRAXA-DVI to identify drug repurposing candidates against Fragile X Syndrome.
BIOEMTECH
Purposeful partners with BIOEMTECH to identify drug repurposing candidates against Glioblastoma.
Leo Pharma Ltd
Purposeful collaborated with Leo Pharma Ltd to discover and validate drugs to treat Inflammatory and Dermatology indications.