Tamid-001

Tamid-001 is an **adeno-associated virus (AAV)-based gene therapy** being developed to treat Mucopolysaccharidosis type I (MPS I), specifically targeting the ocular manifestations of this rare disease. MPS I is caused by mutations in the IDUA gene, leading to accumulation of glycosaminoglycans (GAGs) in multiple organs. Existing treatments fail to alleviate GAG buildup in the eye, leaving patients at risk of blindness. Tamid-001 aims to provide sustained delivery of the missing enzyme, **alpha-L-iduronidase**, to remove GAGs already present in the eye and prevent future accumulation. The therapy was developed in the laboratory of Dr. Matthew Hirsch at the UNC Gene Therapy Center and is being advanced by Tamid Bio, a subsidiary of Fortress Biotech formed specifically to develop AAV gene therapies for orphan diseases. Proof of principle has been established in the MPS I canine model.