Drug intelligence / Profile preview

OXB-102

Development stage
Discontinued
Lead developer
Oxford BioMedica
Modality
Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intraputaminal
01

Overview

OXB-102 is an investigational gene therapy developed for the treatment of Parkinson's disease. It utilizes a lentiviral vector to deliver three genes—tyrosine hydroxylase (TH), cyclohydrolase 1 (CH1), and aromatic L-amino acid decarboxylase (AADC)—which are essential for endogenous dopamine synthesis. The therapy is administered via direct bilateral infusion into the putamen during surgery, aiming to provide continuous dopamine production in targeted brain regions. Preclinical studies demonstrated that OXB-102 achieves higher dopamine yield and improved motor function compared to its predecessor ProSavin, with favorable safety and tolerability profiles in non-human primates. Clinical trials showed promising improvements in motor symptoms among Parkinson’s patients; however, development was discontinued after Phase I/II trials[1][2][3][5][6][7][8].

Brand names
AXO-Lenti-PD
Other names
ProSavin
02

Targets

DDC (Aromatic L-amino acid decarboxylase)GCH1 (GTP cyclohydrolase 1)

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