How former $RARE employees, academics, patient groups and VCs are targeting autism, epilepsy and other genetic brain disorders
The company has 10 employees.At the center of the disorder is a gene called"WW domain containing oxidoreductase," or WWOX, that encodes for a protein that plays a role in brain development. When that protein is deficient, it causes WOREE syndrome, or"WWOX-related epileptic encephalopathy."Children diagnosed with the extremely rare mutation can develop seizures that can't be treated by drugs — what's known as refractory epilepsy.
With its MZ-9138 program, Mahzi looks to change that prognosis for children with WOREE as well as those with a slightly less severe form which presents at around 10-12 months of age. Mahzi is making the vector, an adeno-associated virus, and will start investigational new drug studies once it is ready, Weiss said. It could be in the clinic in 2024.
Mahzi's patient foundation and academic collaborations also are clear with the other two programs it has disclosed on its website.
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