Ultragenyx Entered into an Exclusive License Agreement with Abeona for ABO-102 (UX111) to Treat Sanfilippo Syndrome Type A
Shots:
- Abeona is eligible to receive commercial milestones along with royalties of ~10% on net sales. Ultragenyx obtains global rights for ABO-102
- The company presented the interim results from the (Transpher A) trial at ASGCT 2022 which showed the neurocognitive development in children aged 2yrs. or with DQ > 60 along with continued or stabilized cognitive function & behavioral progress, increase in cortical gray matter, total cerebral & amygdala volumes
- Additionally, reduction in liver volume, CSF fluid & plasma heparan sulfate, was well-tolerated with no TRAEs & SAEs, ABO-102 has received RMAT, FTD, ODD & RPD designation from the US FDA while PRIME & Orphan medicinal product designations from the EMA
Ref: Ultragenyx | Image: Ultragenyx
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