Scenic Biotech develops genomics and immunotherapy technologies to identify and utilize genetic suppressors for therapeutic applications. Their platform targets disease pathways in neuro- and metabolic disorders, enabling the creation of disease-modifying therapies that offer new treatment options for patients.
Funding
$31M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
BVBFounders
Product
Problem
Many genetic disorders lack effective treatments because therapies often focus solely on the primary disease-causing mutation, neglecting other factors that can modify disease progression. This approach overlooks the potential of targeting alternative pathways in the genome to neutralize the impact of the primary mutation.
Solution
Scenic Biotech is developing modifier therapies, a novel approach to treating genetic disorders by targeting modifier genes that can rebalance health and neutralize the impact of disease-causing mutations. The company's Cell-Seq platform identifies these "hidden disease protection factors," enabling the development of small molecule programs that address neuro- and metabolic diseases. By identifying and drugging untapped modifiers of disease, Scenic Biotech aims to create first-in-class therapies that offer new treatment options for patients with devastating conditions. Their lead program focuses on PLA2G15, a novel lysosomal modifier with potential for treating lysosomal storage disorders and neurodegenerative diseases.
Target Audience
The primary target audience includes patients with neuro- and metabolic genetic disorders, as well as pharmaceutical companies seeking innovative targets and development partnerships.
Features
- Cell-Seq platform for identifying and validating modifier genes as therapeutic targets
- Focus on small molecule inhibitors targeting novel disease modifiers
- Lead program targeting PLA2G15 for lysosomal storage disorders and neurodegenerative diseases, currently in IND-enabling studies
- Pipeline programs targeting GALK for Galactosemia, as well as treatments for X-ALD and mitochondrial disease
- Discovery programs for HSAN1 (inherited peripheral neuropathy) and MacTel2 (eye disease)
- Expertise in designing brain-penetrating compounds