{"id":1913,"date":"2026-07-02T18:14:00","date_gmt":"2026-07-02T18:14:00","guid":{"rendered":"https:\/\/futurenews24.com\/index.php\/2026\/07\/02\/elixirgen-builds-rare-disease-pipeline-around-telomere-biology-disorders-and-dmd\/"},"modified":"2026-07-05T15:59:12","modified_gmt":"2026-07-05T15:59:12","slug":"elixirgen-builds-rare-disease-pipeline-around-telomere-biology-disorders-and-dmd","status":"publish","type":"post","link":"https:\/\/futurenews24.com\/index.php\/2026\/07\/02\/elixirgen-builds-rare-disease-pipeline-around-telomere-biology-disorders-and-dmd\/","title":{"rendered":"Elixirgen Builds Uncommon Illness Pipeline Round Telomere Biology Issues and DMD"},"content":{"rendered":"


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In the course of the 2026 BIO Worldwide conference in San Diego, <\/span>GEN <\/span>sat down with Aki Ko, CEO of Elixirgen Therapeutics, to debate the corporate\u2019s 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. <\/span><\/p>\n

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.\u00a0<\/span><\/p>\n

Ko based the corporate with CSO Minoru Ko, MD, PhD, in 2017. <\/span>Elixirgen\u2019s 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, \u201cnow we have a moist lab and an animal lab\u201d that has \u201chelped us go from in vivo to in vitro in a short time to check ideas or optimize formulations and issues like that.\u201d\u00a0<\/span><\/p>\n

Lately, Elixirgen introduced an possibility settlement with Japan\u2019s 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.<\/span><\/p>\n

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.<\/span><\/p>\n

\u201cPresent 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,\u201d Ko mentioned in feedback in regards to the announcement. \u201cBy 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.\u201d<\/span><\/p>\n

EXG-7001 leverages certainly one of Elixirgen\u2019s 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. \u201cThe important thing options are that it’s a lipid nanoparticle-free, localized mRNA therapeutics platform,\u201d Ko defined to <\/span>GEN<\/span>. With this method, \u201cwe\u2019re avoiding among the issues of gene therapies and delivering genes systemically by going native\u201d and avoiding liver accumulation, which stays \u201ca giant challenge\u201d for mRNA therapeutics.\u00a0<\/span><\/p>\n

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<\/span>\u00ae<\/span> mRNA. Although the lead indication for this expertise is DMD, there are different illnesses involving massive genes that the corporate might goal.\u00a0<\/span><\/p>\n

\u201cWe\u2019re capable of specific the complete size protein as mRNA as a single strand\u201d and \u201cit stays the place you administer it, which is form of uncommon,\u201d 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. \u201cA full size dystrophin being given to form of key muscle tissues might probably change high quality of life,\u201d significantly for the non-ambulatory inhabitants, Ko mentioned.\u00a0<\/span><\/p>\n

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\u2019s Hospital Medical Heart. That is an ex vivo cell remedy primarily based on the corporate\u2019s ZSCAN4 expertise, which is designed to increase the telomeres of stem cells in \u201ca managed means\u201d 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.<\/span><\/p>\n

The options of that expertise had been recognized by the corporate\u2019s CSO and his staff whereas he labored on the Nationwide Institutes of Well being\u2019s 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.\u00a0<\/span><\/p>\n

\u201cTelomeres clearly have a relationship with growing older, and there are in actual fact genetic illnesses related to quick telomeres and telomerase mutations,\u201d CEO Ko advised <\/span>GEN<\/span> at BIO. Folks with TBDs are \u201cborn with shorter telomeres usually, but in addition have a mutation of their telomerase so they aren’t essentially sustaining them both.\u201d 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, \u201cbone marrow failure is likely one of the largest points\u201d affecting each adults and younger kids, CEO Ko mentioned.\u00a0<\/span><\/p>\n

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\u2019s ZSCAN4-based remedy might make it doable to do this.\u00a0<\/span><\/p>\n

The therapy is at the moment being examined in grownup and pediatric sufferers in Cincinnati. \u201cWe began in adults as a result of that is first-in-human,\u201d however the illness can also be very extreme in kids, Ko mentioned. \u201cOur goal in the end is to verify as many individuals with TBDs can get this in the event that they want it.\u201d Early medical outcomes printed in 2025 <\/span>in a paper in <\/span>NEJM Proof<\/span> 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 \u201cnow we have lots of longer-term information now\u201d and are \u201ctrying towards potential accelerated approval.\u201d<\/span><\/p>\n

However concentrating on TBDs is only one indication. \u201cBrief telomeres manifest in many alternative methods,\u201d Ko mentioned. Different potential targets for the corporate\u2019s expertise are aging-related illnesses, together with issues like idiopathic pulmonary fibrosis.\u00a0<\/span><\/p>\n

To this point, Elixirgen has raised roughly $34 million from current buyers.<\/span><\/p>\n<\/p><\/div>\n


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Source link <\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

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\u2019s 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 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