Asklepios BioPharmaceutical (AskBio) subsidiary BrainVectis has obtained clearance from the French National Agency for Safety of Medicines and Health Products (ANSM) to carry out the Phase I/II clinical trial of its new gene therapy, BV-101, for treating Huntington’s disease (HD). 

The authorisation, along with the French Ethics Committee in charge’s approval for the trial protocol, will aid in commencing subject recruitment in the trial.

The dose-escalation, open-label trial will evaluate the safety, tolerability, and initial efficacy of BV-101 in adult patients with early-stage HD.

Set to commence in Paris in the fourth quarter of this year, the trial anticipates enrolling 12 to 18 subjects.

A new adeno-associated virus (AAV) gene therapy vector, BV-101 addresses diseased neurons’ metabolic dysfunction and aids in clearing mutant huntingtin proteins at the same time. 

It is given through magnetic resonance imaging (MRI)-guided neurosurgical approaches to act on tissues in the brain’s basal structures. 

The gene therapy showed the potential for repairing the vital cholesterol pathway, providing neuroprotection, and restoring physical performance by delivering CYP46A1, in preclinical research in mice.

An enzyme in the brain, CYP46A1 is found to be low in HD patients. 

BrainVectis founder Nathalie Cartier-Lacave said: “Unlike other attempts to treat Huntington’s Disease, BV-101 aims to restore cholesterol metabolism, reduce mutant huntingtin, and to improve neuronal function. 

“Importantly, BV-101 does not affect the levels of normal huntingtin protein in cells. 

“If this proves successful, we have the potential to change the course of a devastating disease that causes severe functional and cognitive decline.”

There are currently no approved disease-modifying therapies for HD that exist.

Anomalous repeating mutations in the huntingtin gene cause abnormal protein build-up in nerve cells, resulting in this rare, inherited, neurodegenerative ailment.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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