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Biogen, UPenn Partner in $2B Gene Therapy Research Deal

Adeno-associated virus

Electrostatic image of adeno-associated virus (National Institute of General Medical Sciences, NIH)

16 May 2016. The biotechnology company Biogen is collaborating with University of Pennsylvania’s medical school on research into gene therapies and editing, with payments to the university totaling as much as $2 billion. The partnership will fund research in the labs of University of Pennsylvania medical professors James Wilson and Jean Bennett, who study gene therapies to treat rare and inherited disorders, as well as eye diseases.

Biogen, in Cambridge, Massachusetts is also licensing technology from biotechnology company Regenxbio Inc., in Rockville, Maryland as a treatment for rare genetic vision disorders. Payment amounts to Regenxbio were not disclosed.

Wilson, a cell and molecular biologist at UPenn, conducts research on the transfer of healthy genes to repair or replace faulty genes associated with genetic diseases, such as cystic fibrosis, inherited muscular disorders, and metabolic diseases. Adeno-associated viruses are among the key technologies investigated in Wilson’s lab for delivering gene therapies. Adeno-associated viruses are benign, naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, and generate a mild immune response.

Bennett, a professor of ophthalmology at UPenn, studies gene therapies as treatments for eye disorders resulting from degeneration of the retina, including retinitis pigmentosa and age-related macular degeneration. Her work includes transfer of genes to specific cells in the retina, which is being extended to address hearing loss.

In the deal with UPenn, Biogen is funding research on gene therapies with adeno-associated viruses, including development of manufacturing methods for gene therapies. The work is also expected to extend to new types of gene therapies, as well as genome editing. Biogen anticipates these therapies will be applied to diseases of the eyes, skeletal muscles, and central nervous system.

The agreement could bring UPenn as much as $2 billion. The deal calls for Biogen to make an initial $20 million payment, with the university receiving another $62.5 million to for research over the next 3 to 5 years in the Wilson and Bennett labs covering 7 preclinical initiatives. Each of the 7 programs will be eligible for milestone payments of $77 million to $137.5 million per product, as well as royalties on future sales. In addition, Biogen receives an option to license new types of adeno-associated viruses for gene therapies targeting diseases outside of those in the UPenn deal.

In the deal with Regenexbio, Biogen receives an exclusive license to two of Regenexbio’s gene-therapy delivery technologies using adeno-associated viruses, originally developed in Wilson’s lab at UPenn. Biogen expects to apply those gene therapies to 2 rare inherited vision disorders. Under the agreement, Regenexbio is receiving an initial payment, and will be eligible for future fees, milestone payments, and royalties on sales.

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