Active, Not Recruiting



Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)



AB-1001 is an investigational gene therapy originally developed by BrainVectis, which was acquired by AskBio in 2020. AB-1001 was previously called BV-101. This is Phase I/II dose-finding study to evaluate the safety, tolerability, and the ability to produce the desired result (i.e., efficacy) of a one-time intracerebral injection of AB-1001 (previously BV-101) within the striatum of adults with early manifest Huntington’s disease.

AB-1001 will be given through MRI-guided neurosurgical techniques directed to target tissues in the basal structures of the brain. This approach uses a modified and harmless virus to deliver to brain cells of the caudate and the putamen a copy of the gene encoding the CYP46A1 enzyme. This is a neuron-specific enzyme which helps regulate brain cholesterol synthesis and metabolism, and is deficient in people with HD. By increasing the levels of the enzyme, the therapy is expected to normalize cholesterol metabolism in the brain, which may help slow neurodegeneration.

In two mouse models, this therapy showed the ability to repair the essential cholesterol pathway (which is damaged in HD patients), reduce mutant Huntington protein aggregation,  provide neuroprotection, and restore neuronal function. The main goal of the new trial is to assess the safety of AB-1001 in up to 18 people with early manifest Huntington’s disease and specifically to identify the highest dose that can be given without unacceptable safety issues.

The trial will be in 2 parts with approximately 12-18 participants. The first part aims to find the correct AB-1001 dose and will have two arms (i.e., two groups of people with HD – one receiving a low dose of AB-1001 and other receiving a high-dose of AB-1001). The second part is an expansion of the trial. The exact number of participants will depend on the safety of part 1. This trial is called an open-label, meaning there is no placebo group. The participants will be rigorously monitored for 52 weeks and there is a long follow-up phase of 4 years.

Ages Eligible
for Study:

18 Years to 65 Years   (Adult, Older Adult)

Sexes Eligible
for Study:


Accepts Healthy Volunteers:


  • Documented genetic confirmation of pathological CAG expansion in the huntingtin gene ≥40.
  • Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4, or a DCL of 3 if present with cognitive impairment and clear evidence of disease progression.
  • Striatal MRI volumes per hemisphere: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 (per side) on Screening MRI.
  • All HD concomitant medications stable for at least 30 days prior to screening at the investigator’s discretion.
  • Prior or ongoing medical condition, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, would impact subject’s safety and compliance with the study procedures.
  • Metastatic neoplasms within the five years prior to screening.
  • Presence of clinically relevant immunologic, hematologic, hepatic, cardiac, or renal disease at the time of screening as per investigator’s clinical judgment.
  • Current untreated and unstable depressive disorder or a serious mood disorder requiring hospitalization.
  • History of prior suicide attempt or imminent risk of self-harm based on investigator’s judgment or with a “yes” answer on item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS).
  • Patients with history of confirmed stroke, known intracranial neoplasms, vascular malformations, or intracranial hemorrhage.
  • Subjects not deemed suitable for the surgical procedure as per the Neurosurgeon’s judgment.
  • Any history of gene therapy, cell transplantation or any other experimental brain surgery.
  • Any RNA or DNA targeted HD specific investigational agents such as antisense oligonucleotides within 6 months prior to screening.
  • Subjects unable to tolerate or unwilling to undergo multiple lumbar punctures.
  • Participation in any clinical trial of an approved or non-approved investigational drug or intervention within 12 weeks or 5 half-lives whichever is longer prior to treatment.




Institut du Cerveau (ICM), Hôpital La Pitié Salpêtrière APHP

Address: Paris, Ile-de-France, France, 75013

Contact: Hortense Hurmic. hortense.hurmic@icm-institute.org   


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An inactive substance or treatment that looks the same as, and is given in the same way as, an active drug or intervention/treatment being studied.

The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work. (from HDbuzz.org)

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The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study’s objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.

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Inclusion Criteria

A type of eligibility criteria. These are the reasons that a person is allowed to participate in a clinical study.

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CAG repeat

The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD. (from HDbuzz.org)

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Exclusion Criteria

A type of eligibility criteria. These are reasons that a person is not allowed to participate in a clinical study.