Eladocagene exuparvovec

Unassigned

New Medicines

Aromatic L-amino acid decarboxylase (AADC) deficiency

Information

Advanced therapy medicinal product (ATMP)
PTC Therapeutics
PTC Therapeutics

Development and Regulatory status

None
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes

Category

Gene therapy correcting the DDC gene defect resulting in a sustained increase in production of AADC in target areas of the CNS thus increasing the production of critical neurotransmitters
A very rare, severe, genetic neurometabolic disorder associated with clinical manifestations related to underproduction of serotonin and dopamine, mainly hypotonia, hypokinesia, ptosis oculogyric crises, and signs of autonomic dysfunction. Prevalence is <1 in 1 million [1].
Aromatic L-amino acid decarboxylase (AADC) deficiency
Intracerebral