Lilly-partnered start-up raises $65 million for RNA-targeting drugs

A start-up targeting regulatory RNA is one step closer to getting a drug candidate into human trials with the help of $65 million in series A financing.Haya Therapeutics announced the new fundraising round on Thursday. CEO Samir Ounzain says he and his team will use the money to advance their lead candidate, called HTX-001, into clinical studies. HTX-001 is an antisense oligonucleotide that targets a long noncoding RNA, or lncRNA, that regulates fibroblast activity in the heart. Such RNA molecules form the backbone of Haya’s science. lncRNAs are not translated into proteins but instead regulate biological processes like fibrosis. They are sometimes referred to as a component of the regulatory genome, or dark genome.“Now is the moment where we hope to be able to demonstrate this in humans, which will be important for the whole field in the regulatory genome—the dark genome—but also in cardiology, in the heart failure space,” Ounzain says.Previous attempts to develop new drugs for heart failure have “unfortunately been plagued with failures,” Ounzain says; targeting proteins, a predominant approach, was not fruitful . Ounzain and his team believe that going after lncRNAs instead will allow them to effectively reprogram fibroblasts. Preclinical studies have already shown promise, he says.Second behind HTX-001 in Haya’s pipeline is a potential drug for idiopathic pulmonary fibrosis, a fibrotic disorder involving the lungs. Other early-stage drug candidates are designed to target fibroblasts in solid tumors, including those of head and neck, breast, and pancreatic cancers.Apart from a $20 million seed round in