12.07.17
Fortress Biotech has launched Tamid Bio, a subsidiary dedicated to the development of adeno-associated virus (AAV) gene therapies in orphan diseases with unmet medical needs. As part of its formation, Tamid has entered into three exclusive licensing agreements with the University of North Carolina at Chapel Hill for three preclinical AAV gene therapies, developed in the lab of Matthew Hirsch, assistant professor, ophthalmology, at the UNC Gene Therapy Center.
Tamid’s lead program, Tamid-001, targets the ocular manifestations of Mucopolysaccharidosis type I (MPS I), a rare and progressively debilitating disorder, caused by mutations in the IDUA gene, leading to the accumulation of glycosaminoglycans (GAGs) in multiple organs. Existing treatments fail to alleviate the buildup of GAGs in the eye, leaving patients at risk of blindness.
With proof of principle established in the MPS I canine model, Tamid-001 will aim to provide sustained delivery of the missing enzyme, alpha-L-iduronidase, to remove the GAGs already in the eye and prevent future accumulation. Tamid has also in-licensed two earlier-stage assets, developed in Dr. Hirsch’s lab, which will target dysferlinopathies and corneal transplant rejection. Dr. Hirsch will lead preclinical and early clinical research programs at the UNC Gene Therapy Center.
“We are thrilled to be working with Dr. Hirsch to develop novel gene therapies in areas of unmet medical need,” said Lindsay Rosenwald, chairman, president and chief executive officer, Fortress Biotech. “Early pre-clinical data have demonstrated the potential of his AAV technologies to significantly improve treatment options for patients. Fortress looks forward to collaborating with Tamid and Dr. Hirsch to rapidly advance these therapies toward clinical trials.”
Tamid’s lead program, Tamid-001, targets the ocular manifestations of Mucopolysaccharidosis type I (MPS I), a rare and progressively debilitating disorder, caused by mutations in the IDUA gene, leading to the accumulation of glycosaminoglycans (GAGs) in multiple organs. Existing treatments fail to alleviate the buildup of GAGs in the eye, leaving patients at risk of blindness.
With proof of principle established in the MPS I canine model, Tamid-001 will aim to provide sustained delivery of the missing enzyme, alpha-L-iduronidase, to remove the GAGs already in the eye and prevent future accumulation. Tamid has also in-licensed two earlier-stage assets, developed in Dr. Hirsch’s lab, which will target dysferlinopathies and corneal transplant rejection. Dr. Hirsch will lead preclinical and early clinical research programs at the UNC Gene Therapy Center.
“We are thrilled to be working with Dr. Hirsch to develop novel gene therapies in areas of unmet medical need,” said Lindsay Rosenwald, chairman, president and chief executive officer, Fortress Biotech. “Early pre-clinical data have demonstrated the potential of his AAV technologies to significantly improve treatment options for patients. Fortress looks forward to collaborating with Tamid and Dr. Hirsch to rapidly advance these therapies toward clinical trials.”