Companies in #Lysosomal Storage Disorders

Denali Therapeutics (NASDAQ: DNLI) is a biotechnology company headquartered in South San Francisco, California, developing a broad portfolio of therapeutics for neurodegenerative diseases through its proprietary blood-brain barrier (BBB) delivery platform (the ATV/Otv platform, including Enzyme Transport Vehicle and Oligonucleotide Transport Vehicle technologies). Denali's clinical and preclinical pipeline targets Parkinson's disease (BIIB122/DNL151, partnered with Biogen), Hunter syndrome (DNL310, tividenofusp alfa), ALS (DNL343), and multiple other lysosomal storage diseases and neurodegenerative indications. The company was founded in 2015 by a team that included former Genentech scientists.

SonoThera is a biotechnology company developing non-viral, ultrasound-based gene therapies. Its platform uses microbubble-mediated focused ultrasound to deliver DNA, RNA, and other nucleic acid payloads directly to target tissues, eliminating the immunogenicity and packaging-size limitations of viral vectors.