Tag: Healthcare provider

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Elixirgen Builds Rare Disease Pipeline Around Telomere Biology Disorders and DMD

During the 2026 BIO International convention in San Diego, GEN sat down with Aki Ko, CEO of Elixirgen Therapeutics, to discuss the company’s multi-platform technology development efforts. The company, which was founded in 2017, is developing what it believes are breakthrough technologies that target telomere biology disorders (TBD) and aging as well as mRNA-based therapies.

Its therapy for addressing telomere biology disorders, based on its proprietary ZSCAN4 approach, is the furthest to the clinic. The biotech company will also target other aging-related diseases with the technology. Meanwhile, its efforts in the mRNA space are currently focused on Duchenne Muscular Dystrophy (DMD), although there are plans to pursue other targets there as well. 

Ko founded the company with CSO Minoru Ko, MD, PhD, in 2017. Elixirgen’s 15 employees are based in Baltimore in its office space in the Johns Hopkins Medical campus, although there is no affiliation with the university. This location offers some advantages to the company, according to Ko. Specifically, “we have a wet lab and an animal lab” that has “helped us go from in vivo to in vitro very quickly to test concepts or optimize formulations and things like that.” 

Recently, Elixirgen announced an option agreement with Japan’s Nippon Shinyaku focused on DMD. Under the terms of the agreement, Elixirgen will be responsible for the development of an asset dubbed EXG-7001, a locally administered, full-length dystrophin mRNA therapeutic that is currently in preclinical development for the treatment of DMD.

As part of the deal, Nippon Shinyaku will provide funding for the developmental costs of the therapy. Meanwhile, Elixirgen will receive an upfront payment and is eligible to receive additional development and sales-based milestone payments if the option were to be exercised. Also, Nippon Shinyaku may obtain exclusive worldwide rights to commercialize EXG-7001.

“Current approaches for treating DMD focus on delivering or restoring an incomplete dystrophin protein, and there still remains a significant unmet need for a therapy that can successfully deliver a full-length dystrophin protein,” Ko said in comments about the announcement. “By design, EXG-7001 has the potential to deliver the full-length, complete dystrophin protein that is missing in DMD patients, regardless of their genetic mutation.”

EXG-7001 leverages one of Elixirgen’s core technologies. The company has developed a platform for delivering mRNA-based therapies that it claims addresses the major delivery limitations of current methods. “The key features are that it is a lipid nanoparticle-free, localized mRNA therapeutics platform,” Ko explained to GEN. With this approach, “we’re avoiding some of the complications of gene therapies and delivering genes systemically by going local” and avoiding liver accumulation, which remains “a big issue” for mRNA therapeutics. 

The system has two components. The first component, called RNA tether, is designed to ensure that the RNA stays in the tissue that is injected without migrating to the liver. The second component is the mRNA cargo itself, which the company calls Bobcat® mRNA. Though the lead indication for this technology is DMD, there are other diseases involving large genes that the company could target. 

“We’re able to express the full length protein as mRNA as a single strand” and “it stays where you administer it, which is kind of unusual,” Ko said. Combining RNA tether and Bobcat makes it possible to express large genes and localize them to target tissues even without accumulation in the liver. Preclinical data has demonstrated its effectiveness in mice with no safety concerns associated with administration or treatment. “A full length dystrophin being given to kind of key muscles could potentially change quality of life,” particularly for the non-ambulatory population, Ko said. 

Beyond EXG-7001, Elixigen has other candidates in its pipeline that are much closer to the clinic. Its lead candidate is currently in Phase I/II testing at Cincinnati Children’s Hospital Medical Center. This is an ex vivo cell therapy based on the company’s ZSCAN4 technology, which is designed to extend the telomeres of stem cells in “a controlled way” using a telomerase-independent mechanism. EXG-34217 is comprised of autologous CD34+ hematopoietic stem cells that have been treated ex vivo with EXG-001, a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector encoding human ZSCAN4.

The features of that technology were identified by the company’s CSO and his team while he worked at the National Institutes of Health’s National Institute on Aging. In 2024, the U.S. Food and Drug Administration granted Rare Pediatric Disease Designation to the treatment, dubbed EXG-34217, for the treatment of patients with dyskeratosis congenita and related telomere biology disorders. 

“Telomeres obviously have a relationship with aging, and there are in fact genetic diseases associated with short telomeres and telomerase mutations,” CEO Ko told GEN at BIO. People with TBDs are “born with shorter telomeres typically, but also have a mutation in their telomerase so they are not necessarily maintaining them either.” The result is a type of premature aging, so conditions like bone marrow failure and cytopenia happen earlier in the life of the patient. In fact, “bone marrow failure is one of the largest issues” affecting both adults and young children, CEO Ko said. 

One treatment option in these cases is allogeneic hematopoietic stem cell transplantation (HSCT), he continued. However, people with short telomeres have more fragile genomes that are less resistant to chemotherapy and radiotherapy and are at greater risk of cancer even after HSCT treatment. In an ideal scenario, it would be possible to postpone or avoid HSCT for these patients, and the company’s ZSCAN4-based therapy could make it possible to do that. 

The treatment is currently being tested in adult and pediatric patients in Cincinnati. “We started in adults because this is first-in-human,” but the disease is also very severe in children, Ko said. “Our target ultimately is to make sure as many people with TBDs can get this if they need it.” Early clinical results published in 2025 in a paper in NEJM Evidence show durable telomere extension overall with no treatment-related safety concerns observed over a 24-month and 5-month period after infusion. The trial has been going on for some time, and “we have a lot of longer-term data now” and are “looking toward potential accelerated approval.”

But targeting TBDs is just one indication. “Short telomeres manifest in many different ways,” Ko said. Other potential targets for the company’s technology are aging-related diseases, including things like idiopathic pulmonary fibrosis. 

To date, Elixirgen has raised roughly $34 million from existing investors.

The post Elixirgen Builds Rare Disease Pipeline Around Telomere Biology Disorders and DMD appeared first on GEN – Genetic Engineering and Biotechnology News.

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Effects of Virtual Reality on Postoperative Pain Management Following Minimally Invasive Gynecologic Surgery: Randomized Controlled Trial

<strong>Background:</strong> Postoperative pain and anxiety remain common concerns after minimally invasive gynecologic surgery despite advances in surgical techniques and analgesic strategies. Virtual reality (VR) has been investigated as a potential nonpharmacological intervention for pain management; however, evidence in gynecologic postoperative settings is limited. <strong>Objective:</strong> This study aims to evaluate the efficacy and safety of VR technology compared with standard postoperative analgesia for pain and anxiety management in patients undergoing minimally invasive gynecologic surgery. <strong>Methods:</strong> This randomized controlled trial was conducted at Sun Yat-sen Memorial Hospital of Sun Yat-sen University in China. A total of 131 patients undergoing laparoscopy or combined hysteroscopy for benign gynecologic diseases were randomly assigned in a 1:1 ratio to either a VR group (n=68) or a control group (n=63). All patients received a standardized general anesthesia protocol intraoperatively. The control group received conventional analgesic therapy after surgery, and the VR group received a 20-minute VR intervention 6 hours postoperatively. The pain and anxiety levels were evaluated using a visual analog scale at 6 and 7 hours postoperatively. The primary outcome was the change in pain scores between 6 and 7 hours. Secondary outcomes included maximum pain score, anxiety score changes, length of hospital stay, hospitalization costs, and occurrence of adverse events. Analyses were performed according to the intention-to-treat principle. <strong>Results:</strong> There was no statistically significant difference in the primary outcome between the VR and control groups (mean difference 0.169, 95% CI −0.271 to 0.608; <i>P</i>=.45). Similarly, no significant differences were observed in the maximum pain score (mean difference 0.839, 95% CI −0.101 to 1.779; <i>P</i>=.08), and no improvement was observed in the anxiety score (mean difference 0.042, 95% CI −0.365 to 0.449; <i>P</i>=.84). No significant differences were found in length of hospital stay, hospitalization costs, or incidence of adverse events, including dizziness, nausea, and vomiting (all <i>P</i>&gt;.05). <strong>Conclusions:</strong> A single 20-minute VR intervention did not provide additional analgesic or anxiolytic benefit compared with standard postoperative care after minimally invasive gynecologic surgery. VR was well tolerated, and its role in postoperative recovery requires further investigation. <strong>Trial Registration:</strong> Chinese Clinical Trial Registry ChiCTR2400091244; https://tinyurl.com/4b92a9td
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Developing forensic patient-oriented research guidelines: a rapid review using an integrated knowledge translation approach

This paper reports findings from a rapid literature review that informed new guidelines for conducting patient-oriented research in forensic mental health settings. The project adopted an integrated knowledge translation approach at a mental health hospital in Ontario, Canada, engaging a project team that included current forensic patients, hospital staff, and members of an international community of practice. Sources were identified through nine academic databases and targeted grey literature searches, screened independently by two reviewers and extracted using a structured template guided by an a priori framework developed with patients and staff at a knowledge exchange event. Findings were iteratively refined through a patient advisory group, an implementation study, ethnographic observations, and related integrated knowledge translation activities conducted alongside the review. Together, 31 academic and grey literature sources informed a framework organized around five core dimensions: 1) Resourcing, orientation, and training; 2) Confidentiality, consent, and compensation; 3) Relationships, shared understanding, and support; 4) Levels of engagement; and 5) Evaluation and sustainability. Guided by cross-cutting principles common among participatory mental health research, such as dignity, trust, respect, and a commitment to redressing power and attending to forms of epistemic injustice, the guidelines respond to distinctive constraints of forensic environments while highlighting opportunities to promote authentic co-production and sustain patient involvement in research. Recommendations include dedicated resources and capacity-building for patients; relational, ongoing consent practices co-developed with patients; flexible patient researcher roles with fair, paid compensation; and sustained institutional support for participatory practices. We call on forensic hospitals and secure settings to adapt and evaluate these guidelines and to invest in expanding patient leadership to advance the field.
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Smoking as a correlate of suicidal behavior and self-harm in adolescents with depressive disorders

Suicidal behavior and self-harm are major public health concerns among adolescents, particularly those with depressive disorders. While smoking has been linked to suicidality in general populations, its independent role in both suicidal behavior and self-harm within well-characterized clinical samples of youth with depressive disorders remains understudied. This study consecutively enrolled 2,343 adolescents (aged 12–18 years) diagnosed with unipolar depression, bipolar disorder, or depressive episode according to DSM-5 criteria at Nanhai Public Health Hospital of Foshan City and The Third People’s Hospital of Foshan between January 2025 and December 2025. Suicidal behavior (≥1 suicide attempt) and self-harm (intentional self-injury or poisoning regardless of intent) were assessed via clinical interviews. Multivariable binary logistic regression models were constructed to identify factors independently associated with each outcome, adjusting for age, sex, education, income, parental education, psychiatric diagnosis, family history of mental illness, physical disease, and smoking status. Among 2,343 participants (mean age 14.99 ± 1.65 years; 77.8% female), the prevalence of self-harm was 76.0% and suicidal behavior was 44.2%. In fully adjusted models, smoking status was the only variable significantly associated with suicidal behavior after adjustment for measured covariates: current smokers (OR = 2.74, 95% CI: 1.81–4.22, P<0.001) and past quitters (OR = 2.32, 95% CI: 1.66–3.28, P<0.001) had higher odds compared to never smokers. For self-harm, current smoking was significantly associated with increased risk (OR = 2.31, 95% CI: 1.33–4.37, P = 0.006). Education level showed a borderline association with self-harm, with each additional year of schooling corresponding to a 10% lower odds (OR = 0.90, 95% CI: 0.81–1.00, P = 0.050); however, this finding should be interpreted cautiously given the exploratory nature of the analysis. No other demographic or clinical variables, including age, sex, or psychiatric diagnosis, were independently associated with either outcome. Smoking was strongly associated with both suicidal behavior and self−harm after adjustment for measured demographic and clinical covariates. These findings underscore the importance of assessing tobacco use as a potential clinical marker of vulnerability in youth mental health settings.
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Use of a Conversational Agent for Training Mental Health Professionals in Suicide Safety Planning: Pilot Feasibility and Acceptability Study

Background: Safety planning is recognized as one of the most effective interventions for reducing suicidal behaviors. The quality of safety plans strongly depends on professional training, and traditional methods, such as role-playing, are time-consuming and offer limited opportunities for repetition across diverse patient profiles. Generative artificial intelligence (GenAI) may provide innovative solutions by offering accessible, flexible, and realistic training environments. Objective: This pilot study aimed to evaluate the acceptability and feasibility of a GenAI-based simulator designed to train mental health professionals in safety planning. Methods: Twenty nurses and nursing assistants from psychiatric units in a French university hospital participated in a pre-post, single-session evaluation. After self-rating their ability, competence, and willingness to manage patients experiencing suicidal ideation, participants interacted individually with the text-based simulator for 20 minutes to perform a safety plan with a chatbot, then completed postsimulation acceptability items, and open-ended feedback. Composite scores were computed: acceptability (eg, helpfulness; 0‐40), realism (eg, looking like real interaction with patient; 0‐20), and challenge (eg, emotional challenge; 0‐30). Pre-post changes were tested (Wilcoxon signed-rank test), and age-group comparisons were performed. Results: Acceptability was high (mean 31.9/40, SD 5.3; median 32, IQR 7), realism moderate-to-high (mean 15.1/20, SD 4.1; median 15, IQR 5.25), and challenge manageable (mean 17.0/30, SD 8; median 18, IQR 12.5). Participants rated usefulness (mean 7.65/10, SD 1.57; median 8, IQR 1.57), perceived learning (mean 7.6/10, SD 1.79; median 8, IQR 2), recommendation to use the chatbot for training (mean 8.3/10, SD 1.59; median 9, IQR 2.25), and feedback quality (mean 8.35/10, SD 1.27; median 8.5, IQR 1.25) favorably. Willingness to actively manage patients experiencing suicidal ideation significantly increased postsimulation (.03). Younger participants reported higher acceptability (.04) and realism (.03). Participants reported minimal concerns regarding the simulator’s use. Conclusions: This pilot study demonstrates that a GenAI-based simulator for safety planning is feasible and highly acceptable among experienced mental health professionals. The findings are promising and warrant larger, controlled trials to assess impacts on training effectiveness and patient outcomes.
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