Biogen joins race to develop gene editing as a therapy

Biogen has become the latest company to join the race to develop gene editing as a therapy, in a potential $2 billion alliance with the University of Pennsylvania.

At the moment, gene editing is used in drug research to create specially modified cells with disease characteristics that can be used to create drugs. But the holy grail of gene editing is to use the technique as a therapy in itself to cure diseases by correcting genetic defects in live patients.

Companies like AstraZeneca are exploring the technology known as CRISPR/cas9, but Biogen and Penn will be looking at an alternative technique using adeno-associated virus (AAV) vectors to edit genes.

There are advantages and disadvantages to both approaches – CRISPR/cas9 is cheap to use and easily targets rogue DNA sequences. But at the moment it cannot be used as a therapy as it will often target other DNA sequences away from the target site with potentially harmful results.

Biogen’s chosen approach is much more accurate but the small size of AAV vectors limits the amount of genetic material they can carry, meaning they are unable to insert large genes. Research is under way to solve the drawbacks of both techniques.

The latest partnership will focus primarily on approaches targeting the eye, skeletal muscle and central nervous system.

Biogen will work with James Wilson, professor of medicine and paediatrics, and director of Penn’s Gene Therapy Programme and Jean Bennett, professor of ophthalmology and cell and developmental biologay and director of the Center of Ophthalmology and Cell Development Biology and director of the Center for Advanced Retinal and Ocular Therapeutics.

Penn will receive up to $2 billion in research funding, options and milestone payments. Biogen will pay $20 million upfront with an additional $62.5 million to fund R&D costs over the next three to five years in seven preclinical research programmes conducted by the Wilson and Bennett labs

Each programme may trigger milestones ranging from $77.5 million to $137.5 million per product, as well as royalties payable on net sales. Biogen will get an option to license next generation AAV vectors for certain indications from Penn, for Biogen’s use outside of the collaboration.

Biogen is also entering into a licensing agreement with REGENEXBIO for the exclusive rights to use adeno-associated virus serotype 8 (AAV8) or 9 (AAV9) technologies developed in Dr. Wilson’s laboratory, for which REGENEXBIO holds a worldwide patent licence from Penn. The AAV8 and AAV9 technologies will be used in ophthalmic collaboration programmes with Penn.

Bennett said: “Recent advances in gene transfer and editing have now brought us to a promising stage and we believe that this exciting collaboration with Biogen will further fuel our collective ability to translate our research into viable gene therapies.”

Biogen faces slowing growth of its big-selling multiple sclerosis drug, Tecfidera (dimethyl fumarate) and there are concerns about a possible link with the brain infection, progressive multifocal leukoencephalopathy.

It began a cost-cutting programme in October to refocus on core areas such as neurology and autoimmune diseases and earlier this month announced plans to spin off its haemophilia business.

Related stories:

Biogen to spin of haemophilia business

Gene editing to create new generation of targeted drugs

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