ANB-004

Originator investigational product
List of clinical studies

ANB-004 is an originator investigational gene therapy product for human use, which was developed for the treatment of spinal muscular atrophy.

ANB-004 is based on a viral vector used to deliver the healthy SMN1 gene to motor neuron cells. A viral vector is a specially designed virus that cannot cause disease but allows delivery of the necessary genes into cells1.

SMA is a genetic disease (that is, due to a change in chromosomes or genes) associated with dysfunction of the spinal cord cells responsible for movement and muscle tone (motor neurons). In SMA, a gene called SMN1 has abnormalities that result in the absence or severe deficiency of a certain protein (abbreviated as SMN) necessary for the normal function of motor neurons.

One of the most modern ways of treating SMA is to deliver a healthy gene to the patient’s body, where it enters the cells and can start the process of production of the necessary protein. This will promote the production of SMN protein, which, in turn, theoretically can improve the course of SMA2.

Preclinical trials in animals confirmed the safety of ANB-004 and demonstrated its ability to induce the production of SMN protein.

The investigational product has not been previously used for the treatment of humans, so its side effects are currently unknown.

In the clinical trial, the investigational product ANB-004 is administered intravenously. The research physician closely monitors patients’ health throughout their participation in the study and will take all necessary actions to treat adverse events if they occur.

  1. Deverman BE, Ravina BM, Bankiewicz KS, Paul SM, Sah DWY. Gene therapy for neurological disorders: progress and prospects. Nature Reviews Drug Discovery. 2018;17(9):641-59.
  2. Jablonka, S., Hennlein, L. & Sendtner, M. Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders. Neurol. Res. Pract. 4, 2 (2022).

This section of the website contains information about the clinical trial of an unapproved medicinal product.