Afraxis Inc.
This article was originally published in Start Up
Executive Summary
Afraxis Inc. is developing p21-activated kinase (PAK) inhibitors, initially for Fragile X syndrome, a genetic condition involving changes in the X chromosome that can lead to cognitive impairment and autism-like behaviors. The start-up thinks treatment of this orphan indication may be a gateway to developing PAK inhibitors for closely related conditions, such as autism and other central nervous system disorders like Alzheimer's disease and schizophrenia.
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Afraxis Hopes Experimental Fragile X Drug Could Apply To Other CNS Diseases
Biotech believes that inactivation of p21-activated kinase may address underlying cause of Fragile X syndrome.
Ultragenyx Pharmaceutical LLC
Biotech veteran Emil Kakkis founded Ultragenyx Pharmaceutical LLC to develop treatments for rare disorders. First up: a potential therapy for hereditary inclusion body myopathies. There are several variants of this neuromuscular disorder, which causes progressive muscle weakness. By the end of the disease course, patients can be almost quadriplegic in terms of motor function. Ultragenyx is betting it can help restore muscle function in people diagnosed with HIBM by providing a continuous and steady source of sialic acid, likely through thrice-daily oral administration.