This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter.
Scientists long believed the human genome consisted mostly of useless DNA. These sequences, after all, aren’t translated into proteins, making them appear as bits of genetic material with no biological purpose. They were often referred to as “junk.”
Accumulating academic research over the last decade or so has proven otherwise. That “junk” is transcribed into so-called long non-coding RNAs, which are key cogs of the molecular machinery that switches genes on or off. Mining them for drug targets might yield a way to control those switches and, in the process, help treat an array of diseases.
The promise has tantalized venture capitalists for awhile now, leading to the formation of several companies startups — among them Rome Therapeutics and NextRNA Therapeutics — to sift through this “dark genome” for different types of drugs. While research remains early and hasn’t led to an approved medicine, investment has continued, with a fresh financing serving as the latest example.
Biotechnology startup Haya Therapeutics disclosed Thursday that it raised $65 million in Series A funding to probe for drug targets in this large portion of the genome.
The financing will propel Haya’s lead program, for an inherited heart condition called non-obstructive hypertrophic cardiomyopathy, into clinical testing. It will also support earlier programs in development for other conditions, including pulmonary fibrosis and obesity, as well as an expansion of the company’s research efforts.
Sofinnova Partners and Earlybird Venture Capital led the round, which involved Eli Lilly, Alexandria Venture Investments and eight other investment firms. The funding follows a $25 million seed round raised by the firm in 2021 and an obesity-focused partnership with Lilly last September.
Haya is using computing tools to build an internal “atlas” of the dark genome that can help it identify drug targets as well as “RNA-guided therapeutics” that can impact them. In a statement, co-founder and CEO Samir Ounzain claimed Haya’s treatments should “reprogram disease-driving cell states into healthy ones.”
Haya’s first test of that theory is a drug aimed at a long non-coding RNA dubbed “Wisper” that’s overexpressed in certain cardiovascular conditions — among them hypertrophic cardiomyopathy.