Keenly-watched neuro drug developer Denali $DNLI is beefing up its arsenal to smuggle drugs across the brain-blood barrier. The San Francisco-based company joined forces with Sirion Biotech to develop adeno-associated virus (AAV) vectors to enable therapeutics to cross the blood-brain barrier and target diseases of the central nervous system, such as Alzheimer’s, Parkinson’s and Lou Gehrig’s disease.
AAVs are small, non-pathogenic viruses with a genome of single stranded DNA, which can insert genetic material at a specific site. AAV vectors are considered a promising gene delivery system for therapies, although there are some risks associated with their use such as the potential toxicity of high-dose AAV studies. Spark Therapeutics’ $ONCE gene therapy for inherited blindness Luxturna uses an AAV vector and was approved by the FDA in late 2017.
Denali, which is run by a crew of former Genentech researchers including and broke the 2017 record for initial market valuation during a biotech IPO, has raised significant capital based on its targeted, genetics-based approach to CNS, a field littered with high-profile failures. In early 2018, fresh off the heels of its $280 million IPO, the company announced an up to $1 billion deal with Japanese drugmaker Takeda. Later in the year, it picked up an early option to secure access to an antibody technology pioneered at F-Star designed to shepherd drugs across the blood-brain barrier.
The financial terms of the deal with Sirion were not disclosed, although the companies said the transaction included development expenses, upfront and contingent milestone payments to Sirion, and additional royalties on net sales if any product resulted from the partnership.
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