Denali taps Sirion to expand into CNS gene therapies

Denali CEO Ryan Watts (Denali)

Denali Therapeutics is teaming up with Sirion Biotech to expand into gene therapies. The partners will work to develop adeno-associated virus (AAV) vectors capable of crossing the blood-brain barrier. 

Germany’s Sirion has landed deals with companies including Acucela and Orchard Therapeutics on the strength of its expertise in the manufacturing and delivery of gene therapies. That expertise extends to the design of AAVs, a group of gene therapy delivery vectors that have emerged as the preferred option for drugs designed to trigger the production of therapeutic agents in the brain.

Now, Denali has identified Sirion as the company to support its expansion into gene therapies. Denali will work with Sirion and the German company’s partner at the University Hospital Heidelberg, Dirk Grimm, to develop new and modified AAV capsids. 

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The goal is to develop AAV capsids that get across the blood-brain barrier and to targets in the central nervous system with greater specificity and efficiency than is possible with current vectors. Equipped with such vectors, Denali could develop gene therapies capable of getting the brain to make therapeutic levels of molecules to treat diseases including Alzheimer’s and Parkinson’s.

The potential for gene therapies to modify the brain so that it produces its own treatments, rather than try to get large molecules across the blood-brain barrier, has attracted companies including Novartis and Johnson & Johnson.

Denali, in contrast, has focused on small molecules and biologics since it burst onto the scene led by a team of ex-Genentech staffers in 2015. Since then, Denali has raised big sums of cash from private and public investors, enabling it to advance several candidates into clinical development and build a deep pool of preclinical programs.

The Sirion pact gives Denali the chance to add another modality to its pipeline and thereby increase the likelihood that it will be at the leading edge of efforts to treat neurodegenerative disorders in the coming years. 

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