Our development pipeline

Chondrosarcoma

Cholangiocarcinoma

Glioma

Glioma

Non-small Cell Lung Cancer

Non-small Cell Lung Cancer

Non-small Cell Lung Cancer

Gastric cancer

Solid tumors

Solid tumors

Acute Myeloid Leukemia

Acute Myeloid Leukemia

Acute Myeloid Leukemia

Hematological malignancies

Hematological malignancies

Fragile X syndrome is a genetic disorder caused by a mutation in the FMR1 gene, leading to a deficiency of the fragile X mental retardation protein (FMRP). This protein is crucial for normal brain development and function. The absence of FMRP disrupts synaptic plasticity, which is essential for learning and memory, resulting in cognitive impairments and behavioral challenges.​

Patients with Fragile X syndrome often exhibit a range of symptoms, including intellectual disability, anxiety, and social difficulties. The condition can also manifest itself through physical features such as an elongated face and enlarged ears.

KCNT1-related Developmental and Epileptic Encephalopathy (DEE) are a group of severe neurological disorders characterized by very early-onset seizures and significant developmental delays. KCNT1-DEE is caused by genetic mutations in the KCNT1 gene that disrupt normal brain development and function. The seizures are most often refractory to standard anti-epileptic medication, complicating treatment.

Patients with KCNT1-DEE experience a range of symptoms, including severe cognitive impairments, motor deficits, and behavioral issues. The impact on family dynamics and overall quality of life is profound.

More details about ASO technology

Movement disorders are a diverse group of neurological conditions characterized by abnormal movements that significantly impact daily functioning. These disorders are often due to genetic mutations, neurodegenerative processes, or environmental factors affecting the brain’s motor control pathways. Common examples include sustained muscle contractions and abnormal postures, as  irregular, rapid movements difficult to control.

Patients with rare movement disorders often face challenges in mobility, communication, and social interactions. The unpredictability of symptoms is often associated with emotional distress and a reduced quality of life.

Heart Failure

Heart Failure

Hypertension

Hypertension

T2 diabetes

Hypertension

T2 diabetes