Scribe’s Gene-Regulating Cholesterol Drug Enters Human Trials as Upsized IPO Raises $128.7 Million

There are already multiple options for lowering cholesterol, but adherence gaps caused by long-term medication or regular injections still leave an unmet need in cardiovascular disease prevention. Scribe Therapeutics is attempting to extend the treatment interval to several years: the company completed an upsized initial public offering, selling 8.58 million shares at $15 each to raise $128.7 million, replenishing funds for its first human trial and subsequent lipid programs.

The offering was larger than the original plan of approximately 7.15 million shares at $13 to $15 per share. Scribe shares trade on Nasdaq under the ticker SCTX; Sanofi separately subscribed for 500,000 shares at the same price, investing $7.5 million. BioPharma Dive noted that this was the first priced IPO by a gene-editing drug developer in more than two years, also indicating that capital markets are willing to assume earlier-stage technology risk.

Scribe’s lead candidate, STX-1150, targets PCSK9. This protein limits the liver’s ability to clear low-density lipoprotein cholesterol (LDL-C), and existing antibodies, RNA interference drugs, and other therapies in development use the same biological mechanism. STX-1150 instead uses lipid nanoparticles to deliver a CRISPR-derived epigenetic regulation tool into liver cells, suppressing PCSK9 gene transcription without rewriting the DNA sequence, with the goal of achieving a lasting effect from a single intravenous administration.

The company has begun its first-in-human trial in Australia and expects to enroll up to 64 adults with elevated LDL-C and an increased risk of atherosclerotic cardiovascular disease. The trial will first assess safety and tolerability while monitoring changes in PCSK9 and LDL-C. Initial data are scheduled for release in the first half of 2027, marking the first critical point for determining whether epigenetic silencing can translate from animal models to humans.

Evidence of efficacy currently still comes primarily from preclinical data submitted by the company. After a single infusion of an STX-1150 prototype in nonhuman primates, the lowest tested dose group showed an average LDL-C reduction of more than 50%, reportedly sustained for two years; however, the studies involved limited sample sizes and were not designed to demonstrate statistical significance. The actual dose, duration, immune response, liver tolerability, and unintended gene regulation in humans all remain to be answered by the trial.

IPO proceeds will also support two earlier-stage programs: STX-1200, which edits LPA to reduce lipoprotein(a), and STX-1400, which targets APOC3 to address diseases involving high triglycerides. The company expects the two programs to begin Phase 1 trials as early as 2027 and 2028, respectively. According to estimates in earlier offering documents, existing resources plus the proceeds could fund operations through the first half of 2029, but would still be insufficient to advance any candidate through regulatory approval and market launch.

For Scribe, the public offering addresses the timing needs of near-term clinical development but does not eliminate the technology’s uncertainty. Epigenetic silencing avoids some concerns associated with permanently rewriting DNA, but the company must still demonstrate that its effects are sufficiently stable and predictable, and that any adverse reactions remain manageable. The early human results in 2027 will determine whether this funding has purchased the next stage of growth or an expensive clinical validation exercise.