The Jackson Laboratory

The authors tested two different base editors in mice — one called ABE8e and a modified version called ABE8e-V106W — and found that both efficiently corrected the PEX1 mutation, but ABE8e-V106W was significantly better tolerated in the animals and made fewer off-target edits. When Kiran Musunuru of the University of Pennsylvania Perelman School of Medicine, one of baby KJ’s lead doctors, asked Liu which deaminase he and his team should use to correct KJ’s disease-causing mutation, Liu recommended ABE8e-V106W based on the data reported in today’s publication. “These observations, well before the data was published, suggested that ABE8e-V106W is an adenine base editor that sits in the Goldilocks zone, where it offers both high on-target activity that can efficiently correct a pathogenic mutation, and relatively low off-target activity that reduces the frequency of unwanted edits,” said Liu , Richard Merkin Professor, and director of the Merkin Institute for Transformative Technologies in Healthcare at Broad the Dudley Cabot Professor of the Natural Sciences in the Faculty of Arts and Sciences at Harvard University, and Howard Hughes Medical Institute investigator. “It’s easy to assume that rescuing this quite-rare peroxisome disorder in mice wouldn’t have anything to do with saving a human baby with a very different life-threatening disease. But, it actually had a lot to do with it. It goes to show how science builds on itself, and how sharing the latest unpublished findings at a key moment can inform important decisions in the cutting-edge treatment of patients,” Liu added. The findings pave a path toward treating the root genetic cause of Zellweger spectrum disorder, a metabolic disease that affects about 1 in 50,000 to 90,000 births in North America. “Correcting the most common PEX1 mutation is just the beginning of building meaningful treatment strategies for patients with Zellweger spectrum disorder, who currently have no options that treat the cause of the disease,” said lead author Xin “Daniel” Gao , now an Assistant Professor at the University of Pittsburgh, who led the project as a postdoctoral fellow in Liu’s lab. “More broadly, this work lays the foundation for using precision gene editing to develop better treatments for other rare peroxisomal diseases caused by mutations in PEX genes.” “A great deal of work went into the mouse modeling for this project. Ultimately, we developed a model that faithfully reproduces the disease and its long-chain fatty acid biomarkers, giving us strong confidence that these findings will translate into the clinic. This collaboration between David’s and my labs represents one of many joint efforts and serves as a cornerstone for future therapeutic advances emerging from the Center for Genetic Surgery,” said Lutz , co-senior author of the study and vice president of the Rare Disease Translational Center at Jackson Laboratory. Translational potential In their study, the researchers administered base editors to neonatal and older mice with the PEX1 variant, using two adeno-associated viruses, which delivered the base editors to the livers of the animals. The team revealed that the base editor corrected the PEX1 gene in roughly 60 percent of liver cells, high enough to restore peroxisome and liver function and lower the buildup of toxic metabolites throughout the body. The scientists also showed that the editing efficiency increased over the duration of the study — a lower dose of base editors was able to achieve the same level of editing over time as the higher dose the team tested. According to Liu, more research is needed to translate this work in mice into a potential treatment for humans. For example, the authors showed that the base editing system could be reformulated to be delivered using lipid nanoparticles, which have been used in several treatments for humans including KJ’s. However, further optimization of nanoparticle delivery of the base editor to treat Zellweger spectrum disorder (ZSD) remains to be done. One of Liu’s ultimate goals is to make personalized gene editing treatments accessible to patients on a broader scale through the new Center for Genetic Surgery (CGS) that he co-leads at Broad. “Currently, candidate CGS programs are ones for which a base or prime editor has been shown to correct a pathogenic mutation back to a healthy sequence, resulting in at least partial rescue of the disease in an animal model,” Liu said. “Demonstrating benefit to animals provides the foundation to move the study towards a clinical trial that we hope will eventually benefit patients with few treatment options.” “There is an urgent unmet need to develop and implement targeted therapies that address the root causes of thousands of genetic disorders. I am honored that our work on a common form of ZSD may have broader impact across other genetic diseases,” said Hacia , co-senior author and medical geneticist at the Keck School of Medicine of University of Southern California. “Many children with ZSD develop life-threatening liver disease, leading to frequent hospitalizations and a shortened lifespan. We are driven by the hope of changing that reality and look forward to advancing this work into clinical trials to address liver disease in ZSD and related pathologies in other affected organs.” The study’s co-first authors are Xin “Daniel” Gao of Broad and Maximiliano Presa of Jackson Laboratory, and other co-authors include Nancy Braverman of McGill University and Guangping Gao of University of Massachusetts Chan Medical School. Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. 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NEW YORK, April 14, 2026 (GLOBE NEWSWIRE) -- Neion Bio, a biotechnology company revolutionizing the production of biologic medicines, today announced the appointment of James Kehler, VMD, PhD, as Head of Avian Sciences. Dr. Kehler joins Neion Bio from Colossal Biosciences, where he served as Vice President of BioSynergy overseeing multiple programs at the intersection of genome engineering and animal science. His appointment further strengthens Neion’s leadership bench in advanced genome engineering, following the earlier recruitment of founding geneticist and Chief Scientific Officer Dr. Sven Bocklandt, who also joined Neion from Colossal Biosciences. “James has a rare ability to translate cutting-edge genome engineering into real-world preclinical and translational execution,” said Dimi Kellari, co-founder and CEO of Neion Bio. “As we scale our platform toward commercial programs, building a leadership team with deep experience at the frontier of genetic engineering is a deliberate priority.” Dr. Kehler brings more than two decades of experience developing advanced gene-edited animal models and enabling preclinical studies for leading biotechnology and pharmaceutical organizations. His pioneering work in cloning mammals from blood was recognized as one of TIME magazine’s Best Inventions of 2025. Earlier in his career, Dr. Kehler worked alongside Dr. Bocklandt to develop the world’s first gene-edited hypoallergenic cat. He received his VMD and PhD in Cell and Molecular Biology from the University of Pennsylvania and completed post-doctoral training at the National Institutes of Health, including research appointments at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Institute of Neurological Disorders and Stroke (NINDS), and the National Cancer Institute (NCI). Dr. Kehler has held leadership roles at several notable biotechnology organizations, including Stemgent (now part of REPROCELL), the New York Stem Cell Foundation (now part of The Jackson Laboratory), MTI-GlobalStem (now part of Thermo Fisher Scientific), and Mirimus. In these roles, he led the development of advanced gene-edited models to enable translational and preclinical programs across multiple therapeutic areas. “Very few areas of science have advanced as rapidly as genome engineering over the past decade,” said Dr. Kehler. “I’m thrilled to join Neion Bio at this stage of both the field and the company’s development to apply frontier genetics to an industrial application with the potential to meaningfully expand innovation and access to medicines. Neion has a unique opportunity to translate scientific progress into durable technology that will have lasting impact.” Dr. Kehler’s appointment reinforces Neion Bio’s focus on advancing its genome engineering platform toward industrial-scale production of biologic medicines. His addition further strengthens the company’s ability to translate scientific innovation into scalable, cost-efficient manufacturing solutions for the biotech industry. About Neion Bio Neion Bio is pioneering a new era in biomanufacturing, using frontier genetic engineering to harness nature’s most prolific molecular factory – the egg – to produce biological medicines with unprecedented efficiency, scalability, and environmental sustainability. Founded in 2024, Neion aims to dramatically lower the cost of biologics manufacturing to spur biotech innovation and democratize global access to life-changing therapies. Neion’s Raptor™ platform enables localized, resilient domestic manufacturing of critical medicines, strengthening national security while unlocking significant commercial opportunities. Led by a multidisciplinary team of scientists and industry veterans with deep expertise in genetic engineering, biologics manufacturing, and commercialization, Neion is building a platform designed to reset the boundaries of what is possible in biomanufacturing. The company aims to become the partner of choice for upstream biologics manufacturing and deliver transformational advantages for the biotech industry and patients worldwide. Neion Bio Contacts: info@neionbio.com Photos accompanying this announcement are available at https://www.globenewswire.com/NewsRoom/AttachmentNg/e37518bc-d9ef-45ce-99a7-35e82f143444 https://www.globenewswire.com/NewsRoom/AttachmentNg/4f6216ab-2904-4d09-b30a-41ec33b0e03b

Holstein will lead external enterprise engagement strategy to advance JAX's next-generation biomedical research platform and global impact BAR HARBOR, Maine, April 13, 2026 /PRNewswire/ -- The Jackson Laboratory (JAX), a global leader in genetics and genomic medicine, today announced the appointment of Steven Holstein to senior vice president of external affairs. Holstein joins JAX at a pivotal moment as the organization continues to advance a new, integrated, platform-driven approach to biomedical research to accelerate the development of breakthrough therapies. As a member of the executive team, Holstein will lead a coordinated enterprise strategy across advancement, communications, marketing, and government and external affairs. His role will focus on aligning partnerships, capital, and institutional engagement to expand JAX's scientific platform's reach across its growing network of research partners in the United States and internationally, reinforcing its position as a central node in the global biomedical ecosystem. "JAX is helping drive an inflection point in biomedical research, redefining how breakthrough discoveries translate into life-changing real-world therapies," said Lon Cardon, Ph.D., FMedSci, president and CEO of JAX. "Realizing the full impact of our vision requires aligning partnerships, capital, and global engagement at scale, and Steven brings the leadership to serve as a force multiplier, connecting our science to the relationships and resources that will accelerate that impact." This appointment comes as JAX expands its role as a global platform for biomedical discovery, connecting its integrated research capabilities with leading academic institutions, industry partners, and philanthropic organizations worldwide. Recent collaborations, including its partnership with GSK and its acquisition of the New York Stem Cell Foundation, reflect a broader effort to extend JAX's capabilities beyond its own laboratories and into the wider scientific ecosystem. Across these initiatives, JAX is enabling discovery, validation, and translation at scale, helping researchers and partners around the world move more quickly from insight to impact. "JAX plays a unique role in the biomedical ecosystem by developing and integrating the models, data, and platforms that enable more predictive, scalable science," said Holstein. "The opportunity now is to connect that scientific foundation more directly to the partnerships, capital, and global engagement required to accelerate discovery and deliver real-world impact, positioning JAX as a catalyst and convener for the broader research community." Holstein brings more than two decades of leadership experience spanning financial services, healthcare, and life sciences. He previously held senior roles at MassMutual, Guardian, and Fidelity Investments, where he led enterprise marketing, distribution, and growth strategies. Most recently, he served as managing director and head of business development at Outcome Capital, advising founders, boards, and investors on capital formation and strategic partnerships to advance life science and health care innovation. Throughout his career, Holstein has led integrated efforts that connect institutional strategy with external stakeholders, aligning marketing, business development, and executive engagement to drive measurable growth and long-term value. About The Jackson Laboratory The Jackson Laboratory (JAX) is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center. JAX leverages a unique combination of research, education, and resources to achieve its bold mission: to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health. Established in Bar Harbor, Maine in 1929, JAX is a global organization with nearly 3,000 employees worldwide and campuses and facilities in Maine, Connecticut, California, Florida, New York, and Japan. For more information, please visit . SOURCE The Jackson Laboratory 21% more press release views with Request a Demo