Phase I study for human monoclonal antibody for Lyme disease demonstrates safety, tolerability, pharmacokinetics. By Jim Fessenden March 31, 2026 A Phase I clinical trial of a human monoclonal antibody discovered and developed at UMass Chan Medical School for the prevention of Lyme disease in the U.S. was well tolerated and showed lasting serum concentrations in participants, according to data presented by Mark Klempner, MD, at the World Vaccine Congress 2026 in Washington D.C. TNX-4800 (formerly known as mAb 2217LS), a long-lasting borreliacidal (or bactericidal) was licensed to Tonix Pharmaceuticals Holding Corp., a commercial biotechnology company in Berkeley Heights, N.J. An adaptive Phase 2 field study is expected to begin in the first half of 2027, pending FDA clearance. "Our study demonstrated potentially protective blood levels of TNX-4800 at two days, with protective blood levels sustained for at least four months due to its extended half-life design," said Dr. Klempner, professor of medicine. "Additionally, with its differentiated mechanism of action, TNX-4800 has the potential to provide passive immunity by directly supplying neutralizing antibodies, bypassing the need for a vaccine to induce a patient's immune system to generate its own antibodies, which can be associated with other issues. We look forward to further clinical investigation of TNX-4800 as we strive to overcome this major public health challenge." Lyme disease, the most common tick-born illness in the Northern hemisphere, is transmitted to humans through the bite of infected deer ticks (Ixodes scapularis). According to the Centers for Disease Control and Prevention estimates, there are more than 450,000 cases annually in the U.S. That number is expected to rise as global changes in climate are expanding habitat range for ticks and other disease vectors. Typical symptoms of Lyme disease include fever, headache, fatigue and a characteristic skin rash called erythema migrans. If left untreated, infection can spread to the joints, heart and nervous system. While most cases of Lyme disease can be treated successfully with antibiotics, the only way to prevent infection is to avoid tick bites by using insect repellent and removing ticks promptly. "TNX-4800 is expected to provide a preventative option to the 87 million people in the United States who are at high risk of contracting the disease because they live, work or vacation in a tick-endemic area," said Seth Lederman, MD, chief executive officer of Tonix Pharmaceuticals. "As a monoclonal antibody, we believe TNX-4800 offers significant advantages over vaccines in development. Lyme disease vaccines that elicit antibodies to OspA [a protein found on the outer membrane of the bacterium that causes Lyme disease] currently in development take more than six months to offer protection and require complex immunization schedules. A previously approved anti-OspA vaccine was withdrawn due to poor uptake, potentially relating to its complex immunization schedule," said Dr. Lederman. Lyme is not caused by the tick directly. It is caused by the bacterium Borrelia burgdorferi present in the gut of infected ticks. When an infected tick bites someone and begins to feed on their blood, Lyme-causing bacteria can slowly travel from the tick's gut to its salivary glands and then transfer to the site of the bite. TNX-4800 blocks the maturation of Borrelia burgdorferi in the mid-gut of infected deer ticks. This inactivates the bacteria in the tick before it ever reaches the skin at the site of the tick bite. Intended to be administered in the early spring before ticks are at their most active, TNX-4800 is expected to provide almost-immediate protection from Borrelia burgdorferi bacteria-caused Lyme disease. A single injection is designed to maintain protective antibody serum concentration, with expected duration of protection approximately four months, providing pre-exposure protection against Lyme without relying on the recipient's immune system to generate antibodies. The primary objective of the Phase 1 study was to evaluate the safety and tolerability of a subcutaneous injection of TNX-4800 when administered to healthy male and female subjects 19 to 65 years old. The secondary objective was to evaluate pharmacokinetics when administered to healthy subjects. A total of 44 subjects were enrolled in the study, with 41 completing it. Results showed no significant clinical or laboratory safety signals with most adverse events mild or moderate. Pending FDA clearance, Tonix plans to initiate a randomized, double-blind, placebo-controlled, adaptive Phase 2 trial in the first half of 2027.

UMass Chan Medical School, Caring Cross and Trenchant BioSystems form collaborative research and development alliance to treat sickle cell disease (SCD) patients with gene-edited HSCs utilizing new cell processing technologies. March 30, 2026 Alliance focused on providing affordable, autologous cell and gene therapies (CGT) manufactured at Place-of-Care Worcester, MA, Gaithersburg, MD, San Diego, CA - The Department of Genetic & Cellular Medicine at UMass Chan Medical School, the academic department focused on advancing gene and cell therapies to treat rare inherited diseases, Caring Cross, which focuses on equal access to advanced medicines for all patients in need, and Trenchant BioSystems, the CGT manufacturing platform developer for gene-modified cells, today announce a collaborative research alliance for the development and manufacture of an accessible and commercially viable hematopoietic stem cell (HSC) manufacturing platform for diseases like SCD. The alliance combines Trenchant BioSystems' disruptive technology for automating patient-specific CGT processes, UMass Chan's expertise on blood stem cell processes and Caring Cross's expertise in increasing patient access. The collaboration will develop a gene-modified stem cell manufacturing process with Trenchant BioSystems' AutoCell automated CGT manufacturing platform that is designed to be scalable and operate at place-of-care in an ISO class 7 environment, to increase efficiencies and decrease costs. A key reason Trenchant BioSystems' automated CGT manufacturing platform was selected is its use of a microbubble separation approach as an alternative to immunomagnetic bead-based separation for stem cell gene therapies. In addition, AutoCell has a small footprint and therefore significantly fewer facility requirements, important factors for lowering the cost of these therapies. In the first phase of the collaboration, UMass Chan researchers will work with Trenchant BioSystems to start evaluating blood products to separate stem cells and build the automated gene transfer genetic engineering platform with lentiviral vectors from Caring Cross. In the next post-validation phase, Caring Cross will evaluate the system and process for simplicity and cost before offering it as a potential alternative to its collaborators worldwide. UMass Chan and Caring Cross will conduct IND-enabling preclinical studies to launch a Phase I/II clinical trial of autologous gene-modified HSCs for patients with SCD or beta-thalassemia. The alliance currently plans to hold an INTERACT meeting with the US FDA in Q1 2027 and launch the clinical trial at the start of the second half of 2027. Jennifer E. Adair, PhD, Vice Chair and Professor of Genetic & Cellular Medicine and Director of the Horae Gene Therapy Center at UMass Chan Medical School, said, "Disruptive technologies such as the AutoCell platform that empower us to tap into the immense unexplored runway between current state of the art ex vivo and in vivo blood cell gene therapies stand to significantly expand and improve access to these transformative medicines." "Caring Cross is dedicated to ensuring the global affordability of advanced therapies, and a key driver for this is the adoption of cell processing platforms that effectively lower barriers to patient care," said Dr. Boro Dropulic, Executive Director of Caring Cross. "The Trenchant AutoCell platform has the potential to lower cost, increase scalability, and expand the accessibility of place-of-care manufacturing." "Current cell and gene therapy manufacturing practices are too time consuming and costly to actually deliver CGT therapies to large-scale patient populations. Trenchant BioSystems has now released internal and independent data that confirms that its AutoCell platform is integral to the solution to these challenges," said Jon Ellis, CEO, Trenchant BioSystems. "This alliance will allow Trenchant to contribute to the first autologous CGT clinical treatment of SCD patients that is simple, fast, automated, and affordable, thus scalable globally." About UMass Chan Medical School UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine, the Morningside Graduate School of Biomedical Sciences, the Tan Chingfen Graduate School of Nursing, ForHealth Consulting at UMass Chan Medical School, MassBiologics, and a thriving Nobel-Prize-winning biomedical research enterprise. UMass Chan is advancing together to improve the health and wellness of its diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. It is ranked among the best medical schools in the nation for primary care education and biomedical research by U.S. News & World Report. Learn more at www.umassmed.edu. About Caring Cross Caring Cross is a 501(c)(3) nonprofit dedicated to accelerating the development of and ensuring global access to advanced therapies. To achieve its mission, Caring Cross develops technologies and therapeutic candidates that improve the accessibility and affordability of advanced medicines like CAR-T therapy and stem cell gene therapy. Lentiviral vectors used to manufacture Caring Cross advanced therapy medicinal products are manufactured by Vector BioMed, a for-profit contract development and manufacturing organization (CDMO) specializing in rapid manufacturing solutions to provide the industry with affordable, high-quality GMP lentiviral vectors. For more information on Caring Cross, visit https://caringcross.org. About Trenchant BioSystems Trenchant BioSystems is a disruptive technology firm reimagining cell and gene therapy manufacturing to be scalable, efficient, rapid and affordable through the development of truly automated, end to end manufacturing platforms. Trenchant has developed technology that will reduce the production times for CAR-Ts to 2.5 days. Its manufacturing platform will also reduce CAR-T manufacturing cost significantly. Trenchant was launched in 2022, and is based in San Diego, CA, US. It was founded by chairman and CTO Philip Coelho, CEO Jon Ellis, and Bill Busa, PhD. To discover more about Trenchant, visit https://www.trenchantbio.com.

UMass Chan receives $800K for ALS research. March 12, 2026 As universities throughout the nation struggle to access federal funding, UMass Chan Medical School in Worcester has received hundreds of thousands of dollars from a nonprofit dedicated to supporting the school's ALS research. The Angel Fund for ALS Research, based in Wakefield, awarded UMass Chan $800,000 to fund the research of Dr. Robert Brown, the university's chair in neuroscience, and Daryl Bosco, chair of research for the department of neurology, according to a Thursday press release. Both professors lead their own labs at UMass Chan focused on research of neuromuscular and neurodegenerative diseases, particularly ALS. Brown's lab researches gene defects that help explain the development of neuromuscular diseases, while Bosco's lab studies the pathogenic mechanisms associated with neurodegenerative diseases, according to the university's website. Brown, who identified the first gene linked to hereditary ALS, credits The Angel Fund for helping to advance the university's ALS research program. Since its establishment in 1997, the fund has awarded more than $10 million to the Worcester school. "This has made a decisive difference in the breadth of our program, which almost exclusively focus on investigations related to therapy development. Some of our most innovative programs have been possible because of the generosity of The Angel Fund," Brown said in the release. The Angel Fund's sole mission is to fund ALS research and clinical trials at UMass Chan, with the ultimate goal of eradicating the disease. In fiscal year 2024, the nonprofit generated $895,977 in revenue and held $3.17 million in assets, according to nonprofit financial tracker Candid. UMass Chan's most recent funding comes after the university received a $35-million donation for its research of neurodegenerative and genetic diseases, particularly ALS, from the Florida-based Paul J. DiMare Foundation in February 2025. The gift marked the third-largest donation in the history of the entire UMass system, prompting UMass Chan to rename its 350,000-square-foot research building the Paul J. DiMare Center. With 1,359 full-time students enrolled in fall 2024, UMass Chan is the 11th-largest higher education institution in Central Massachusetts, according to data provided to WBJ's Research Department. In June, the university's Chancellor Dr. Michael Collins announced he would retire from the role at the end of the 2026 academic year. Collins is the school's longest-serving chancellor, having assumed the role in an interim capacity in 2007 and being appointed to the permanent role in 2008. Mica Kanner-Mascolo is a staff writer at Worcester Business Journal, who primarily covers the healthcare, manufacturing, and higher education industries. - Digital Partners -