Lonza licenses out AAV tech for gene therapies, signs MEE research pact

Lonza Houston and Massachusetts Eye and Ear (MEE), the world’s largest vision and hearing research center, have inked a new pact that will allow gene therapy players the ability to in-license synthetic adeno-associated viral vectors (AAVs).

Specifically, the deal will see the two allow customers to buy into Anc80 and other Anc-AAVs for their gene therapies. These Anc-AAVs were first developed in the lab of Dr. Luk Vandenberghe, assistant Professor at Harvard Medical School, and Director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear.

The virus itself is small and weak and not thought to cause disease, but can still enter into the body. Viruses are modified in the lab to provide vectors that carry corrected, therapeutic DNA into cells, where it can be integrated into the genome to alter abnormal gene expression and correct genetic disease, and has become a common method of delivery for gene therapies.


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“In this era of personalized medicine, the partnership with Lonza is unique, and potentially very effective,” Dr. Vandenberghe said, “as it brings a highly potent vector technology under one roof with a leading manufacturer of biologics.

“We believe this concept will bring innovative gene therapies to patients in a more efficient and expedient way, and that it will increase access to enabling gene therapy technology to unlock treatment for diseases of unmet need, including those affecting vision and hearing.”

As part of the pact, Lonza has also signed up to create best-in-class, large-scale manufacturing platforms for Anc80, as well as any future vectors coming out of Dr. Vandenberghe’s lab.

Lonza will also under the pact fund research at the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear to help find the next-generation gene transfer reagents.

For its part, Massachusetts Eye and Ear will allow Lonza the “exclusive position to commercially license Anc-AAV,” but will hold on to certain commercial and academic rights--including the ability to sell self-directed gene therapy programs, as well as all rights in the challenging, but potentially lucrative arena of ultra-rare diseases.


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