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BMN 110
BMN 110 is a gene therapy that delivers a functional copy of the HEXA gene to restore hexosaminidase A enzyme activity in patients with Tay-Sachs disease.
BMN 110 is a gene therapy that delivers a functional copy of the HEXA gene to restore hexosaminidase A enzyme activity in patients with Tay-Sachs disease. Used for Tay-Sachs disease (late-infantile or juvenile onset).
At a glance
| Generic name | BMN 110 |
|---|---|
| Also known as | recombinant human N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfatase, N-acetylgalactosamine-6-sulfate, sulfatase, galactose-6-sulfatase |
| Sponsor | BioMarin Pharmaceutical |
| Drug class | Gene therapy (AAV-based) |
| Target | HEXA gene |
| Modality | Small molecule |
| Therapeutic area | Neurology / Rare Genetic Disorder |
| Phase | Phase 3 |
Mechanism of action
Tay-Sachs disease is caused by deficiency of hexosaminidase A (Hex-A), leading to accumulation of GM2 ganglioside in neurons and progressive neurological deterioration. BMN 110 uses an adeno-associated virus (AAV) vector to deliver a functional HEXA gene directly to the central nervous system, enabling production of functional Hex-A enzyme and reducing pathogenic substrate accumulation. This approach aims to halt or slow neurological progression in affected patients.
Approved indications
- Tay-Sachs disease (late-infantile or juvenile onset)
Common side effects
- Immune response to AAV vector
- Liver enzyme elevation
- Thrombocytopenia
Key clinical trials
- A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
- Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE3)
- BMN 110 Phase 3B in Australian Patients (PHASE3)
- Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE2)
- Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (PHASE2)
- Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation (PHASE2)
- A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE1, PHASE2)
- A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE3)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |