SIRION Biotech strengthens USA links with new partnership and manufacturing site

Martinsried, Germany: – Viral technology specialist SIRION Biotech has significantly expanded its presence in the USA with an important strategic partnership and new site in Massachusetts.

SIRION has joined forces with California-based Denali Therapeutics, a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases.

It is also inaugurating a business development and liaison center as part of the Cambridge gene therapy and research cluster.

Focusing on CNS conditions

The license and collaboration agreement with Denali aims to develop gene therapies for diseases of the Central Nervous System (CNS) by developing 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 other diseases of the CNS.

The research involves the world-renowned and pioneering scientist in the field of AAV biology, Prof. Dr. Dirk Grimm from the University Hospital Heidelberg (UKHD), who has well-established connections with SIRION.

Combined proprietary technologies

AAV vectors are considered the most promising gene delivery system for therapeutic applications. Denali Therapeutics, SIRION Biotech and Dirk Grimm, will combine 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.

SIRION founder and CEO Dr. Christian Thirion commented: “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.”

New neurodegenerative treatment options

“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, SIRION Vice President for BD & Licensing.

Alexander Schuth, COO of Denali Therapeutics said: “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.”

New Cambridge MA site

At the end of this month, SIRION will also formally inaugurate its new site at Cambridge, Massachusetts, a leading cluster for genetic & cell therapy research, to speed communications between SIRION and its partners in North America. SIRION is being supported in its efforts by CYTO Consulting, who specializes in working with international life science companies to expand commercialization of new technologies into the US market.

The SIRION offices will open with a Bavarian-style reception and dinner on the evening of January 31, featuring addresses from world renowned gene & cell therapy developers and viral vector experts, an open Bavarian buffet and guests representing the range of Cambridge’s innovative gene therapy scene.

About SIRION Biotech

Founded at Martinsried, near Munich, in 2007, SIRION BIOTECH has become Europe’s leading commercial supplier of viral vectors used for genetic research, clinical target validation, gene therapy and vaccination studies.

From the beginning, SIRION’s mission has been to change the paradigm for viral vector supplies, with ability to develop and supply all common viral vector types (adenovirus, lentivirus, AAV, etc.) within a matter of weeks at the concentration titers and quantities needed for preclinical human and animal testing,

The company also offers a full range of virus related services, ranging from particle production to virus driven cell modeling.

SIRION offerings include the transformational RNAiONE™ knockdown validation platform.

SIRION’s core expertise lies in custom virus generation for genetic engineering of mammalian cell systems. With its comprehensive viral vector portfolio, SIRION offers a suitable option for almost any in vitro and in vivo application. SIRION can supply all three of the most commonly used virus systems for gene manipulation: recombinant adenovirus particles (AVs), lentiviruses and adeno-associated viruses (AAVs).

About Denali Therapeutics

Denali is a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases.

Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the blood-brain barrier and guiding development with biomarker monitoring to demonstrate target engagement and select patients. Denali is based in South San Francisco.

For further information, see: www.denalitherapeutics.com.