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aptaTargets is a clinical-stage biopharmaceutical company specializing in the development of innovative therapies based on aptamer technology.

The company’s main drug is ApTOLL, an aptamer (single-stranded DNA molecule) with an immunomodulatory and anti-inflammatory effect. The benefit of ApTOLL over other treatments is that its action focuses on blocking the activation of TLR4, a receptor located on the surface of immune cells and responsible for initiating the inflammatory cascade. By blocking TLR4, ApTOLL modulates the unwanted immune response.

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ApTOLL is being investigated in five indications: acute ischemic stroke (clinical phase IIa), COVID-19 (clinical phase Ib), intracerebral hemorrhage, myocardial infarction, and multiple sclerosis (preclinical phase). It should be noted that ApTOLL has shown an excellent safety profile in preclinical and Phase Ia clinical studies, with 46 healthy volunteers, and Phase Ib, with 32 patients, presented at the Stroke International Conference 2022.

Within the framework of the OLIGOFASTX consortium, aptaTargets has started the development of a new aptamer for the treatment of Guillain-Barré syndrome, a rare disease that affects the peripheral nervous system, producing muscle weakness. There is currently no curative therapy for Guillain-Barré syndrome. There is currently no curative therapy for Guillain-Barré syndrome.

Founded in 2014, aptaTargets is based in Madrid (Spain) and has obtained an investment of more than 5 million euros led by Inveready and the Center for Industrial Technological Development (CDTI) of the Ministry of Science and Innovation through the Innvierte program.


Guillain-Barré syndrome (GBS) is a neurological disorder in which the body’s immune system attacks the peripheral nervous system causing various symptoms such as numbness, tingling and muscle weakness that can progress to paralysis. GBS is currently the most common cause of acute flaccid paralysis and is a major health problem worldwide.

During the acute phase of GBS, symptoms progress until reaching clinical stability (4 weeks). From that moment begins a recovery period that can last more than a year. In the most severe cases, GBS can be fatal.

GBS is considered a rare disease with an incidence that varies from 0.9 to 1.9 cases per 100,000 inhabitants per year, mainly in adult men. The most common subtypes are acute inflammatory demyelinating polyneuropathy (AIDP) and acute motor axonal neuropathy (AMAN).

Currently, there is no curative treatment for GBS, only two therapies that try to reduce the severity of the disease and speed up the patient’s recovery based on the removal of antibodies from the blood (plasmapheresis) or the administration of antibodies intravenously to block the antibodies that cause the disease.

The prognosis is variable, with 5% of patients dying despite treatment and 20% remaining with significant aftermath, compromising their autonomy. On the other hand, a very high proportion of patients with an apparent good prognosis remain with persistent symptoms or aftermath that include weakness, pain, or fatigue, the severity of which correlates directly with the degree of neural loss that occurs in the acute phase. Therefore, there is a great path for developing treatments for GBS that can significantly improve the prognosis of the patient.

Several mechanisms are involved in the pathogenesis of the disease, such as humoral and cellular immune responses, autoantibodies and complement, and activated macrophages and lymphocytes. However, the exact role and temporal sequence in which these immunopathogenic mechanisms appear remain to be elucidated.

In most patients, the disease is preceded by an infectious event that triggers the immune response and triggers the acute onset of neurological symptoms manifesting as limb weakness and areflexia.

This direct link to a previous infection suggests that the driver of the autoimmunity developed in the disease is due to molecular mimicry between the antigens of the infectious agent and the peripheral nerve leading to post-infectious inflammation that enhances nerve damage or blocks nerve conduction.