In the course of the 2026 BIO Worldwide conference in San Diego, GEN sat down with Aki Ko, CEO of Elixirgen Therapeutics, to debate the corporate’s multi-platform expertise improvement efforts. The corporate, which was based in 2017, is growing what it believes are breakthrough applied sciences that focus on telomere biology issues (TBD) and growing older in addition to mRNA-based therapies.
Its remedy for addressing telomere biology issues, primarily based on its proprietary ZSCAN4 method, is the furthest to the clinic. The biotech firm will even goal different aging-related illnesses with the expertise. In the meantime, its efforts within the mRNA house are at the moment targeted on Duchenne Muscular Dystrophy (DMD), though there are plans to pursue different targets there as effectively.
Ko based the corporate with CSO Minoru Ko, MD, PhD, in 2017. Elixirgen’s 15 workers are primarily based in Baltimore in its workplace house within the Johns Hopkins Medical campus, though there isn’t any affiliation with the college. This location provides some benefits to the corporate, in response to Ko. Particularly, “now we have a moist lab and an animal lab” that has “helped us go from in vivo to in vitro in a short time to check ideas or optimize formulations and issues like that.”
Lately, Elixirgen introduced an possibility settlement with Japan’s Nippon Shinyaku targeted on DMD. Below the phrases of the settlement, Elixirgen shall be accountable for the event of an asset dubbed EXG-7001, a domestically administered, full-length dystrophin mRNA therapeutic that’s at the moment in preclinical improvement for the therapy of DMD.
As a part of the deal, Nippon Shinyaku will present funding for the developmental prices of the remedy. In the meantime, Elixirgen will obtain an upfront cost and is eligible to obtain extra improvement and sales-based milestone funds if the choice had been to be exercised. Additionally, Nippon Shinyaku could acquire unique worldwide rights to commercialize EXG-7001.
“Present approaches for treating DMD deal with delivering or restoring an incomplete dystrophin protein, and there nonetheless stays a major unmet want for a remedy that may efficiently ship a full-length dystrophin protein,” Ko mentioned in feedback in regards to the announcement. “By design, EXG-7001 has the potential to ship the full-length, full dystrophin protein that’s lacking in DMD sufferers, no matter their genetic mutation.”
EXG-7001 leverages certainly one of Elixirgen’s core applied sciences. The corporate has developed a platform for delivering mRNA-based therapies that it claims addresses the most important supply limitations of present strategies. “The important thing options are that it’s a lipid nanoparticle-free, localized mRNA therapeutics platform,” Ko defined to GEN. With this method, “we’re avoiding among the issues of gene therapies and delivering genes systemically by going native” and avoiding liver accumulation, which stays “a giant challenge” for mRNA therapeutics.
The system has two elements. The primary part, referred to as RNA tether, is designed to make sure that the RNA stays within the tissue that’s injected with out migrating to the liver. The second part is the mRNA cargo itself, which the corporate calls Bobcat® mRNA. Although the lead indication for this expertise is DMD, there are different illnesses involving massive genes that the corporate might goal.
“We’re capable of specific the complete size protein as mRNA as a single strand” and “it stays the place you administer it, which is form of uncommon,” Ko mentioned. Combining RNA tether and Bobcat makes it doable to specific massive genes and localize them to focus on tissues even with out accumulation within the liver. Preclinical information has demonstrated its effectiveness in mice with no security considerations related to administration or therapy. “A full size dystrophin being given to form of key muscle tissues might probably change high quality of life,” significantly for the non-ambulatory inhabitants, Ko mentioned.
Past EXG-7001, Elixigen has different candidates in its pipeline which can be a lot nearer to the clinic. Its lead candidate is at the moment in Part I/II testing at Cincinnati Youngsters’s Hospital Medical Heart. That is an ex vivo cell remedy primarily based on the corporate’s ZSCAN4 expertise, which is designed to increase the telomeres of stem cells in “a managed means” utilizing a telomerase-independent mechanism. EXG-34217 is comprised of autologous CD34+ hematopoietic stem cells which were handled ex vivo with EXG-001, a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector encoding human ZSCAN4.
The options of that expertise had been recognized by the corporate’s CSO and his staff whereas he labored on the Nationwide Institutes of Well being’s Nationwide Institute on Getting older. In 2024, the U.S. Meals and Drug Administration granted Uncommon Pediatric Illness Designation to the therapy, dubbed EXG-34217, for the therapy of sufferers with dyskeratosis congenita and associated telomere biology issues.
“Telomeres clearly have a relationship with growing older, and there are in actual fact genetic illnesses related to quick telomeres and telomerase mutations,” CEO Ko advised GEN at BIO. Folks with TBDs are “born with shorter telomeres usually, but in addition have a mutation of their telomerase so they aren’t essentially sustaining them both.” The result’s a sort of untimely growing older, so situations like bone marrow failure and cytopenia occur earlier within the lifetime of the affected person. In reality, “bone marrow failure is likely one of the largest points” affecting each adults and younger kids, CEO Ko mentioned.
One therapy possibility in these circumstances is allogeneic hematopoietic stem cell transplantation (HSCT), he continued. Nevertheless, individuals with quick telomeres have extra fragile genomes which can be much less proof against chemotherapy and radiotherapy and are at higher danger of most cancers even after HSCT therapy. In a great situation, it might be doable to postpone or keep away from HSCT for these sufferers, and the corporate’s ZSCAN4-based remedy might make it doable to do this.
The therapy is at the moment being examined in grownup and pediatric sufferers in Cincinnati. “We began in adults as a result of that is first-in-human,” however the illness can also be very extreme in kids, Ko mentioned. “Our goal in the end is to verify as many individuals with TBDs can get this in the event that they want it.” Early medical outcomes printed in 2025 in a paper in NEJM Proof present sturdy telomere extension total with no treatment-related security considerations noticed over a 24-month and 5-month interval after infusion. The trial has been occurring for a while, and “now we have lots of longer-term information now” and are “trying towards potential accelerated approval.”
However concentrating on TBDs is only one indication. “Brief telomeres manifest in many alternative methods,” Ko mentioned. Different potential targets for the corporate’s expertise are aging-related illnesses, together with issues like idiopathic pulmonary fibrosis.
To this point, Elixirgen has raised roughly $34 million from current buyers.
