Pharma Focus Asia

SIRION Biotech and Denali Therapeutics Join Forces to Develop Gene Therapies for Diseases of the Central Nervous System

Thursday, January 10, 2019

SIRION Biotech GmbH, a world leader in viral vector-based gene delivery technologies for gene & cell therapy, announced today that it signed a license and collaboration agreement with Denali Therapeutics, a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases, to develop adeno-associated virus (AAV) vectors to enable therapeutics to cross the blood-brain barrier for clinical applications in neurodegenerative diseases including Parkinson’s, Alzheimer’s disease, ALS and certain other diseases of the CNS. Once again SIRION’s strategic alliance partner Prof. Dr. Dirk Grimm from the University Hospital Heidelberg (UKHD) is a key contributor to this highly innovative development. Main terms of the agreement include development expenses, upfront and contingent milestone payments to SIRION Biotech, and additional low-to-mid single digit royalties on net sales resulting from certain future products or therapies.

AAV vectors are considered the most promising gene delivery system for therapeutic applications. Denali Therapeutics, together with SIRION Biotech and Dirk Grimm, a world-renowned and pioneering scientist in the field of AAV biology and application, will combine their proprietary technology platforms to create a next generation of AAV vectors. The goal is to develop new and modified AAV 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. “This ground-breaking collaboration will help Denali Therapeutics to increase the availability of protein therapeutics in the brain, and to quickly enter clinical trials with efficient, safe and scalable therapeutic candidates”, said Dr. Christian Thirion, founder and CEO of the company. “We believe that through our partnership with Denali, CNS-directed AAV-based gene therapies can reach the market in the fastest possible way, providing novel treatment options to many millions of patients worldwide suffering from devastating neurodegenerative diseases such as Parkinson’s, Alzheimer’s, and ALS,” added Dr. Sabine Ott, VP BD & Licensing. Prof. Dirk Grimm notes that "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."

Additionally, Alexander Schuth, COO of Denali Therapeutics stated, “We are excited to partner with SIRION Biotech and combine our expertise around the blood-brain barrier and neurodegenerative diseases with SIRION’s leading expertise on viral vectors for gene therapy to enable new treatments for diseases of the brain. This partnership will add a new therapeutic modality to our portfolio and is complementary to our existing biotherapeutic and small molecule programs.”

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