Biogen, Penn $2 Billion alliance to Squeeze Out Gene Therapy’s Futuristic Role


A $2 billion alliance between the U.S. based Biogen and University of Pennsylvania (Penn) speaks volume about the futuristic application of the investigational technique, gene therapy in the medical world in place of drugs and surgery.

biogen pharma official logoBiogen and Penn collaborated to take the gene therapy and gene editing technologiesuniversity of Pennsylvania logo to a higher level by its multiple gene therapy programs. The collaboration has chalked out multiple objectives to address:-

  • The primary objective is the development of therapeutic approaches for eye, skeletal muscle and the central nervous system (CNS).
  • Validation of next-generation gene transfer technology by utilizing adeno-associated virus (AAV) gene delivery vectors.
  • To understand the expansive use of genome editing technology and to create a potential therapeutic platform for wide range of disease, for the insertion, deletion or replacement of DNA in genome of an organism.

Jean Bennett james wilson penn univerisityBiogen teamed up with two renowned gene therapy experts from Perelman School of Medicine at the University of Pennsylvania, James Wilson, M.D., Ph.D., professor of medicine and pediatrics, and director of Penn’s gene therapy program and Jean Bennett, M.D., Ph.D., professor of ophthalmology and cell and developmental biology and director of the Center for Advanced Retinal and Ocular Therapeutics.

We are committed to advancing gene therapy, and our collaboration with the University of Pennsylvania has significantly strengthened both our leadership and overall capabilities in the field,” said Olivier Danos, Ph.D., senior vice president, Cell & Gene Therapy at Biogen. “Joining forces with gene therapy pioneers, James Wilson and Jean Bennett, further enables Biogen to approach the technology from a powerful perspective. By exploring next-generation delivery in various tissues such as the retina, skeletal muscle and CNS, we will explore the potential for extending gene therapy beyond disorders linked to single gene mutations and into a broader spectrum of complex diseases, including devastating neurological conditions that affect a multitude of patients throughout the world”.

Under the $2 billion deal, Penn will receive an upfront payment of $20 million plus additional fund of $62.5 million for research and development over the period of 3 to 5 years in seven distinct preclinical research and development program conducted by the laboratories of Dr. Wilson and Dr. Bennett.

A potential milestone ranging from $77.5 million to $137.5 million per product is expected from each program plus royalties payable on net sales of products.

Biogen also gets the option to licence the next generation AAV vectors from Penn for some indications which could be used by them outside the collaboration.

In terms of role of both the partners in the collaboration, Penn will combine its extensive gene therapy resources and expertise for the development of therapeutic candidates using both existing as well as newly developed AAV vectors under different collaboration programs and will also help in the development of new manufacturing approaches required to support the commercialization of gene therapy products.

On the other hand, Biogen will help in the advancement of collaboration program into the clinic and further approval. The further strengthening of therapeutic area, target identification expertise and drug development capabilities will help Biogen to support the collaboration program.

Biogen views the development of gene editing technology as a natural extension of its current efforts to develop gene therapies employing viral and non-viral vectors,” noted Dr. Danos. “The successful development of gene editing technology for the treatment of inherited or acquired diseases requires expertise not only in a broad range of biological disciplines, such as translational development and viral and non-viral delivery, but also in a variety of aspects of conventional pharmaceutical product development, including manufacturing and clinical development”.

Apart from this agreement, Biogen is also collaborating with REGENXBIO, which is a leading U.S. based biotechnology company, focussed in development, commercialization and licensing of the AAV gene therapy. REGENXBIO have NAV® Technology Platform which is a proprietary AAV gene delivery platform. It has provided more than 100 novel AAV vectors each enhancing the gene expression for a particular target. Biogen is entering in the licence agreement with REGENXBIO to get the exclusive rights for using adeno-associated virus serotype 8 (AAV8) or 9 (AAV9) technologies which was developed in Dr. Wilson’s laboratory and REGENEXBIO holds a worldwide patent license from Penn. Under the ophthalmic collaboration programs with Penn, AAV8 and AAV9 technologies will be utilized.

The collaboration is expected to develop AAV gene replacement programs and utilize next-generation AAV vectors and genome editing techniques to explore new therapeutic targets.

Biogen believes that these AAV gene transfer capabilities are important for future gene therapy products as well as for application of the genome editing technology for various disease indications.

Featured image credit: Four colorful gears — one green, red, blue and gold — turn in unison to symbolize synergy, cooperation, partnership and collaboration in working together to meet a goal or overcome a challenge © iqoncept (Stock Photo ID: 23779796)

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