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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Denali join hands with Sirion to develop CNS-directed AAV-based gene therapies

Denali Therapeutics, a biopharmaceutical company developing drug candidates for neurodegenerative diseases has entered into a licence agreement with Sirion Biotech to advance gene therapies for central nervous system disorders.

Delivery of biomolecules to cross the blood brain barrier is one of the greatest challenges in developing therapies for central nervous system disorders. Denali Therapeutics is specialized in engineering biomolecules to solve this problem. Through the collaboration with Sirion, the company aims to expand its portfolio to adeno-associated viral (AAV) vectors, to enable these vectors to cross the blood brain barrier and deliver therapeutics genes for neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease.

Prof. Dirk Grimm of University of Heidelberg, Sirion’s strategic alliance partner will also be a key contributor for developing these next generation AAV vectors. The collaboration aims to develop new viral capsids exhibiting a safe product profile with improved specificity and high efficiency for therapeutic drug delivery to the brain at therapeutic levels to effectively treat patients.

Under the terms of the agreement, Denali will pay development expenses, milestone payments and additional royalties to Sirion Biotech.

Prof. Dirk Grimm commented: “By contributing and harnessing our unique and proprietary expertise in the engineering and high-throughput in vivo screening of AAV capsid libraries, we will significantly accelerate this joint endeavour and increase our chances to realize its pivotal aims.”

Source: SIRION Biotech and Denali Therapeutics Join Forces to Develop Gene Therapies for Diseases of the Central Nervous System; Press Release

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