Tralesinidase alfa is in clinical development for the treatment of patients with mucopolysaccharidosis type IIIB (MPS IIIB) also known as Sanfilippo syndrome type B. MPS IIIB is a rare and progressive genetic disorder caused by the deficiency of one of the enzymes needed to break down complex sugar molecules called mucopolysaccharides. Clinically, patients have behavioural problems, intellectual deterioration, sleep disorders, hearing impairment, facial dysmorphism, organomegaly, bowel disturbances, mild skeletal changes, and shortened life span. The clinical severity ranges from mild to severe.
Tralesinidase alfa is an investigational enzyme replacement therapy (ERT) designed to replace the faulty enzyme with a healthy one in patients with MPS IIIB. Tralesinidase alfa is delivered directly to the fluid surrounding the brain (cerebrospinal fluid). If licensed, tralesinidase alfa will offer new therapy option for patients with MPS IIIB who currently have no treatment options.
Eladocagene exuparvovec is a type of gene replacement therapy which involves the transfer of the gene encoding the production of the enzyme needed by the brain for the formation of dopamine and serotonin. The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. By increasing production of the AADC-enzyme, this therapy increases dopamine production in the target area of the brain and improves motor and cognitive symptoms in patients. If licensed, eladocagene exuparvovec will provide the first medicinal treatment option for adult and child patients with AADC-deficiency, a disease of very high unmet clinical need.