rna-splicing
(2)
Trace Neuroscience
Trace Neuroscience is a South San Francisco biopharmaceutical company developing genomic medicines for neurodegenerative diseases, starting with ALS. Its lead program, TRCN-1023, is an antisense oligonucleotide designed to restore UNC13A protein function - a target genetically validated in roughly 97% of people living with ALS - by correcting the faulty RNA splicing caused by TDP-43 dysfunction. The company launched in November 2024 with a $101 million Series A led by Third Rock Ventures and entered the clinic in 2026.
BlossomHill Therapeutics, Inc.
BlossomHill Therapeutics is a San Diego clinical-stage biopharmaceutical company designing small-molecule precision medicines for cancer and autoimmune disease. Founded in 2020 by drug designer J. Jean Cui and biotech veteran Y. Peter Li - the team behind Turning Point Therapeutics (acquired by Bristol Myers Squibb in 2022) - the company is advancing a wholly-owned pipeline led by BH-30643, a first-in-class macrocyclic OMNI-EGFR inhibitor for EGFR-mutant non-small cell lung cancer, and BH-30236, a CLK inhibitor targeting aberrant RNA splicing in AML and high-risk MDS.