AskBio Initiates Phase II REGENERATE-PD Trial for Parkinson's Gene Therapy
On June 26, 2024, Asklepios BioPharmaceutical (AskBio), a subsidiary of Bayer, launched the recruitment process for its Phase II REGENERATE-PD clinical trial to evaluate AB-1005, a gene therapy for moderate-stage Parkinson's disease. This therapy, derived from the AAV2 glial cell line, is designed to deliver the neurotrophic factor GDNF. The double-blind, randomized trial will compare the effects of a single dose of AB-1005 infused into the putamen with a control procedure involving bilateral partial burr holes. With a goal to enroll 87 participants aged 45 to 75 in the UK, US, and Europe, the study aims to determine the safety and efficacy of this innovative therapy.
Encouraged by the positive results from the Phase Ib study, which revealed no serious adverse events associated with AB-1005, Krystof Bankiewicz, AskBio's scientific chair, expressed optimism about the potential impact of this therapy on Parkinson's patients. Moreover, AskBio is also conducting a Phase I trial in the US for patients with the parkinsonian subtype of multiple system atrophy (MSA-P) to test the safety, tolerability, and efficacy of AB-1005 for this condition.
Key Takeaways
- The Phase II REGENERATE-PD trial assesses AB-1005 for moderate Parkinson's disease using gene therapy.
- AB-1005, a neurotrophic factor gene therapy derived from AAV2-GDNF, will be evaluated in the trial.
- The trial involves 87 participants across the UK, US, and Europe and offers either active treatment or a control procedure.
- The Phase Ib results proved AB-1005 to be well-tolerated without any serious adverse events.
Analysis
The commencement of the Phase II REGENERATE-PD trial by AskBio signifies a significant advancement in gene therapy for Parkinson's disease, with potential impacts on patients, healthcare systems, and biotech investments. The trial's global reach implies broad regulatory and market implications. Positive outcomes could expedite approval processes and market entry, benefiting both patients and investors. Conversely, setbacks could lead to delays in treatment availability and diminish investor confidence. The trial's emphasis on safety and efficacy, following promising Phase Ib results, underscores the cautious optimism surrounding gene therapies for neurological conditions. This development could influence future research and funding in the realm of neurodegenerative diseases.
Did You Know?
- AAV2 glial cell line: AAV2, or Adeno-Associated Virus type 2, is a small virus utilized in gene therapy due to its ability to deliver genetic material into cells, without causing disease. Glial cells in the nervous system support neurons. In this context, the AAV2 glial cell line serves as a vector to deliver the gene for GDNF, a neurotrophic factor, to the brain cells of Parkinson's patients.
- GDNF (Glial Cell Line-Derived Neurotrophic Factor): GDNF is a protein that supports the survival and function of dopamine-producing neurons, which are significantly affected in Parkinson's disease. By delivering GDNF directly to these cells, the therapy aims to slow down or reverse the disease's progression.
- Bilateral partial burr holes: In the clinical trial, this surgical procedure involves drilling small holes into both sides of the skull. This minimally invasive control intervention, without the active gene therapy treatment, is used to compare outcomes with those receiving the actual treatment.