AskBio subsidiary readies to start clinical trials for Huntington’s Disease gene therapy

By Rachel Arthur

- Last updated on GMT


Related tags AskBio Bayer Gene therapy Huntington’s disease

BrainVectis, a subsidiary of AskBio, has received clearance from the French authorities to start a Phase 1-2 clinical trial for its novel Huntington’s Disease gene therapy, BV-101.

BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein. It was granted orphan drug designation by the European Medicines Agency (EMA) in 2019.

BrainVectis has now received the green light from ANSM (L’Agence nationale de sécurité du médicament et des produits de santé / National Agency for Safety of Medicines and Health Products), France’s governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge. The company can now start recruiting participants with the trial expected to begin in Q4 2022. 

The company believes that, if successful, the novel approach could also have a wider impact on how other neurodegenerative diseases are treated in the future.

Providing neuroprotection

There are currently no approved disease modifying therapies for Huntington’s Disease (HD), a rare inherited neurodegenerative disease that affects around 62,000 people in the EU. The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells. This results in a range of progressive symptoms, leading to complete physical and mental deterioration, with symptoms usually beginning in adults ages 30 to 50, but which can also occur at an earlier age.

In preclinical studies in mice, BV-101 demonstrated the ability to repair the essential cholesterol pathway, provide neuroprotection, and restore physical performance by delivering CYP46A1, a crucial enzyme in the brain which is reduced in people with Huntington’s Disease. 

Based in Paris, the Phase 1/2 BV-101 clinical trial will be an open-label, dose-escalation study to assess the safety, tolerability, and preliminary efficacy of administration of BV-101 in 12-18 adult subjects with early-stage Huntington’s Disease (HD).

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,”​ said Nathalie Cartier-Lacave, MD, founder of BrainVectis and now Vice President, Sector Lead Neurobiology, at AskBio. “If this proves successful, we have the potential to change the course of a devastating disease that causes severe functional and cognitive decline.”

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in October 2020, is a fully integrated gene therapy company with a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications.

Based in Paris, BrainVectis’ focus is on developing gene therapy treatments for neurodegenerative diseases by targeting the cholesterol pathway in the brain, with the INSERM (French National Institute for Health and Medical Research) spin-out having been acquired by AskBio in April that year.

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