ProSavin (OXB-101) / Oxford Biomedica - LARVOL DELTA

Gene therapy for Parkinson's disease: current landscape, translational challenges, and future directions. (PubMed, Expert Rev Neurother) - "The authors summarize clinical translation with emphasis on trials targeting dopamine synthesis (AAV2 AADC, ProSavin/AXO Lenti PD), neurotrophic factors (AAV2 GDNF, AAV2 NRTN), pathogenic variants (AAV9 GBA1/PR001; LRRK2 RNAi; emerging CRISPR/PINK1 strategies), and circuit modulation (AAV GAD), correlating mechanistic ration. Persistent challenges include limited vector biodistribution, reduced retrograde transport in advanced disease, immune variability, and surgical infrastructure requirements. Overcoming these via engineered capsids, delivery optimization, and validated biomarkers will enable precision, stage‑specific interventions."

Journal • Review • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease • GBA1 • LRRK2

The Next Frontier in Parkinson's Disease Treatment: A Narrative Review of Innovative Neurosurgical and Gene Therapy Approaches. (PubMed, Health Sci Rep) - "Gene therapy strategies like AAV2-hAADC and ProSavin have shown early-phase safety and efficacy, but are still in early clinical stages...The range of PD treatment options is growing because of the newer neurosurgical and gene therapy approaches. The current results are promising, but larger-scale, controlled trials are required to show long-term safety and efficacy."

Journal • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Deep brain stimulation is well tolerated and effective following ProSavin® gene therapy for Parkinson's disease. (PubMed, Neurotherapeutics) - "The decrease in axial scores from UPDRS III MEDSOFF/STIMON at 12 months post-DBS compared with baseline DBS was significantly greater in the GT/DBS group than in the DBS group. DBS of the STN was as effective and well-tolerated in GT/DBS PD patients as in the DBS group, even when administered later in their disease course."

Journal • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

A Review of Genetic and Gene Therapy for Parkinson's Disease. (PubMed, Cureus) - "With these developments, a drug named prosavin is under clinical phase 1 trial...This GDNF infusion technique works with a tetracycline-controlled transactivator. Gene therapy introduction into the treatment of PD would be beneficial as there would be lesser adverse effects seen as linked with conventional treatment involving levodopa, MAO-B inhibitors, and anticholinergics, among a few. This article discusses the genetic basis and genetic model of therapy for PD."

Gene therapy • Journal • Review • CNS Disorders • Gene Therapies • Movement Disorders • Parkinson's Disease

Long Term Safety and Efficacy Study of ProSavin in Parkinson's Disease (clinicaltrials.gov) - P1/2 | N=15 | Terminated | Sponsor: Axovant Sciences Ltd. | Trial completion date: Aug 2022 ➔ Apr 2022 | Active, not recruiting ➔ Terminated | Trial primary completion date: Aug 2021 ➔ Nov 2021; As a result of termination of development of Prosavin, it is no longer possible for Sio to either commence or to continue any related clinical trials.

Trial completion date • Trial primary completion date • Trial termination • CNS Disorders • Movement Disorders • Parkinson's Disease