antisense-oligonucleotides
(3)
Creyon Bio
Creyon Bio is a San Diego biotech reinventing how RNA-targeted medicines are designed. Founded in 2019 by former Ionis Pharmaceuticals scientists, the company pairs machine learning with quantum-level molecular modeling in its NucleIQ platform - an AI-Powered Oligo Engineering Engine - to predict the safety, efficacy, and delivery of oligonucleotide-based medicines before they ever reach a lab bench. The goal: replace slow, expensive trial-and-error with computational design rules that make precision therapeutics engineerable 'on demand,' for both rare and common diseases across neuromuscular, CNS, and immunologic areas.
HAYA Therapeutics
HAYA Therapeutics is a clinical-stage precision medicines company spun out of Lausanne University Hospital (CHUV) in 2019. It develops RNA-guided programmable therapeutics that target long non-coding RNAs in the so-called 'dark genome' - the roughly 98% of human DNA that does not code for proteins - to reprogram disease-driving cell states. Its lead candidate, HTX-001, is an antisense oligonucleotide targeting the cardiac lncRNA Wisper for heart failure, with a broader pipeline spanning fibrosis, metabolic disease and cancer. The company is headquartered in Lausanne, Switzerland, with US laboratory operations in San Diego, and raised a $65M Series A in May 2025.
Leal Therapeutics
Leal Therapeutics is a Worcester, Massachusetts biotech founded in 2021 by repeat CNS entrepreneur Asa Abeliovich, the scientist behind Prevail Therapeutics (acquired by Eli Lilly). Leal builds first-in-class neuro-metabolic medicines on a single idea: many brain diseases share a broken metabolism, and correcting those imbalances can treat conditions from schizophrenia to ALS. Backed by roughly $114M in total funding, its pipeline includes LTX-001, a brain-penetrant oral glutaminase inhibitor in the clinic, and LTX-002, an antisense oligonucleotide for ALS.