cns-diseases
(2)
Atalanta Therapeutics
Atalanta Therapeutics is a Boston-based biotechnology company pioneering RNA interference (RNAi) therapies for diseases of the brain and spinal cord. Its proprietary divalent small interfering RNA (di-siRNA) platform is engineered to silence disease-causing genes durably and broadly across the central nervous system - long the hardest place in the body to reach with RNAi. Founded out of the RNA Therapeutics Institute at UMass Chan Medical School by leaders in the RNAi field, including Nobel laureate Craig Mello, the company is advancing wholly-owned programs in KCNT1-related epilepsy and Huntington's disease toward the clinic, alongside collaborations with Biogen and Genentech.
Jacob Borrajo
Jacob Borrajo is the Founder and CEO of Amber Bio, a Cambridge-based biotech company pioneering a first-of-its-kind RNA writing platform capable of multi-kilobase edits. With a PhD in Biological Engineering from MIT and research training at the Broad Institute, Borrajo has spent his career building at the intersection of CRISPR, machine learning, and synthetic biology. Amber Bio launched in August 2023 with a $26 million seed round co-led by Andreessen Horowitz and Playground Global, with strategic participation from Eli Lilly and the Retinal Degeneration Fund. His platform's key innovation: treating diseases with high allelic diversity using a single therapeutic product, by editing RNA rather than DNA - making gene therapy safer, reversible, and dramatically more scalable.