CHAPEL HILL – The University of North Carolina, a powerhouse in gene therapy research, has signed exclusive licensing agreements with a New York City biotechnology company for three preclinical gene therapies that could treat an eye disease, certain muscular dystrophies and rejection of corneal transplants.

UNC signed the agreements with Tamid Bio, a newly formed subsidiary of Fortress Biotech, a publicly traded biopharmaceutical company that acquires, develops and commercializes novel pharmaceutical and biotechnology products. Terms were not disclosed.

Fortress said in a news release that Tamid will focus on developing adeno-associated virus (AAV) gene therapies in rare diseases with unmet medical needs.

Tamid acquired the rights to three AAV gene therapies developed in the lab of Matthew Hirsch, Ph.D., assistant professor of ophthalmology at the UNC Gene Therapy Center.

One of the therapies, Tamid-001, the company’s lead program, targets mucopolysaccharidosis type I (MPS I), a rare and progressively debilitating genetic disorder. MPS I leads to a buildup of glycosaminoglycans (GAGs), a family of carbohydrates, in multiple organs. In the eye, the buildup can cause blindness.

Tamid-001 will aim to provide sustained delivery of a missing enzyme to remove GAGs in the eye and prevent future accumulation. Existing treatments fail to alleviate the buildup of GAGs.

Proof of principle for Tamid-001 has been established in canine models of the disease, Fortress said.

Tamid also in-licensed two earlier-stage gene therapies that will target corneal transplant rejection and dysferlinopathies, a group of muscular dystrophies caused by mutations in the gene for dysferlin, a protein involved in skeletal muscle repair.

AAV gene therapies use a small, benign virus to ferry corrective DNA into cells so faulty genes that cause disease can be made functional.

Hirsch will lead preclinical and early clinical research programs for Tamid’s three potential AAV therapies 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 A. Rosenwald, M.D., Fortress Biotech’s chairman, president and chief executive officer. “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.”

UNC’s Gene Therapy Center became a wellspring of gene therapy technologies under the directorship of Richard “Jude” Samulski, Ph.D., a pioneer in AAV biology. The Center has partnerships with at least 17 rare disease foundations, eight other universities and biotech companies.

Samulski was recruited to UNC from the University of Pittsburgh with the help of a $430,000 Faculty Recruitment Grant from the North Carolina Biotechnology Center in 1993.

In 2003 Samulski and entrepreneur Sheila Mikhail created Asklepios BioPharmaceutical, or AskBio, a gene-delivery company that has collaborated with major pharmaceutical companies to advance gene therapy treatments for various diseases.

AskBio also spun off three gene therapy companies of its own: NanoCor Therapeutics, which partnered with Medtronic to develop treatments for heart failure; Chatham Therapeutics, which was sold to Baxter for $70 million to develop hemophilia treatments; and Bamboo Therapeutics, which was sold to Pfizer for $150 million up front, plus up to $495 million more in potential milestone payments, to develop cures for rare neuromuscular diseases and muscular dystrophy.

(C) N.C. Biotechnology Center