The Munich-based vector specialist Sirion, the scientist Dirk Grimm from Heidelberg University Hospital and the US biotech company Denali want to jointly develop new gene therapies for neurodegenerative diseases such as Alzheimer’s disease. Sirion waving milestone payments and royalties.
Sirion Biotech GmbH has been known as a reliable partner for more than ten years when it comes to products and services in the field of cell and gene therapies. Two years ago, sales growth of 140% to EUR 4 million and the associated achievement of profitability were celebrated. With the current agreement, which was concluded at the end of 2018, Sirion is now likely to venture into other spheres financially. According to Sirion, this is the company’s most important cooperation to date.
Concrete amounts were not mentioned. What is certain, however, is that the Munich-based company will be reimbursed for its development expenses by Denali Therapeutics Inc., which is listed on the US stock exchange Nasdaq, and will receive advance payments and payments after certain development phases have been reached. In addition, there is a revenue share in the low to mid single-digit percentage range should products from this cooperation reach the market. No information was given on the minimum or maximum number of development programs. If the development of an Alzheimer’s therapy, for example, actually succeeds, small amounts will quickly add up to huge sums.
Denali is knowledgeable about neurodegenerative diseases. Three low-molecular drug candidates are in early clinical development, and other “small molecules” and some biopharmaceuticals are in the pipeline. A program is running with partner Sanofi (France) in the indications amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and multiple sclerosis, and Denali was able to win Takeda (Japan) as a partner for another program.
However, gene therapies are new territory for Denali. This is where Sirion and Dirk Grimm contribute their knowledge. Sirion has been working on adeno-associated viruses (AAV) as vectors for gene therapy for years. Their advantage over other vector technologies: AAV vectors cross the blood-brain barrier more easily and can therefore be administered systemically. The goal of the partnership is to develop novel AAV capsids that have a safer product profile, including better specificity and efficiency than currently available alternatives. Not much was said about the molecular target structures addressed and the type of gene therapy. According to Sirion boss Christian Thirion, it is about certain protein therapeutics whose blueprint is to be smuggled into the brain with the vectors.