Genetic Health
Our compounds are designed to address the underlying cause of severe diseases.
Afro Therapeutic research platform, has broad therapeutic potential. Our ongoing research programs currently focus on two severe diseases.
Our lead clinical program is focused on Dravet syndrome, a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. We are also examining a preclinical target for Autosomal Dominant Optic Atrophy (ADOA) a severe, progressive optic nerve disorder.
Dravet Syndrome
Dravet Syndrome is a rare and severe form of epilepsy that typically begins in infancy, often characterized by prolonged and frequent seizures that may be triggered by factors such as fever or changes in temperature. The condition is caused by mutations in the SCN1A gene, which is essential for the proper functioning of sodium channels in the brain. As children with Dravet Syndrome grow, they may experience a range of challenges, including developmental delays, cognitive impairments, and behavioral issues.
Autosomal Dominant Optic Atrophy (ADOA)
Autosomal Dominant Optic Atrophy (ADOA) is a genetic disorder characterized by progressive vision loss due to the degeneration of the optic nerve, which transmits visual information from the eye to the brain. The condition is inherited in an autosomal dominant manner, meaning that only one copy of the mutated gene from an affected parent can cause the disorder in their offspring.
Rett Syndrome
Rett Syndrome is a rare neurodevelopmental disorder that primarily affects girls and is characterized by a period of normal development followed by a loss of purposeful hand skills, speech, and motor function. The disorder is typically caused by mutations in the MECP2 gene, which is essential for normal brain development and function. Symptoms usually appear between 6 months and 2 years of age, after a period of typical growth and development.
SYNGAP1
SYNGAP1 is a gene located on chromosome 6 that encodes a protein involved in synaptic function, which is crucial for communication between neurons in the brain. Mutations in the SYNGAP1 gene are associated with a range of neurodevelopmental disorders, particularly SYNGAP1-related intellectual disability and epilepsy.
