Kelonia Therapeutics Announces Late-Breaking Oral Presentation Showcasing First-in-Human Data for KLN-1010, an In Vivo BCMA CAR-T Gene Therapy, at ASH 2025
Kelonia Therapeutics, Inc., a clinical-stage biotechnology company advancing a next-generation approach to genetic medicines through in vivo gene delivery, announced today that early clinical findings from its ongoing inMMyCAR Phase 1 study will be highlighted in a late-breaking oral presentation at the American Society of Hematology (ASH) 2025 Annual Meeting, scheduled to take place in Orlando, Florida. This milestone marks the first public disclosure of human data for KLN-1010, Kelonia’s pioneering in vivo gene therapy designed to generate anti-BCMA chimeric antigen receptor (CAR) T cells directly inside a patient’s body.
The inMMyCAR program represents a significant turning point in the field of cellular immunotherapy. For the past decade, CAR-T therapies have delivered transformative responses in patients with a range of hematologic malignancies—most notably multiple myeloma—but their widespread adoption has been constrained by access barriers such as complex manufacturing, time-consuming cell engineering processes, and the need for lymphodepleting chemotherapy. KLN-1010 has been engineered to overcome these limitations by enabling the patient’s own immune cells to become CAR-T cells following a single infusion of an in vivo gene delivery vector. If successful, this model could dramatically expand patient access by eliminating several of the most challenging steps associated with traditional CAR-T treatment.
Overview of Early Clinical Results
The late-breaking abstract to be presented at ASH highlights data from the first three patients treated with KLN-1010 in the inMMyCAR Phase 1 dose-escalation study. Each of these individuals had relapsed and refractory multiple myeloma—a population that typically has limited remaining therapeutic options and generally experiences diminishing duration of response with each successive line of therapy.
Across all three patients, KLN-1010 demonstrated a consistent pattern of deep clinical response. Notably, each patient achieved minimal residual disease (MRD) negativity by month one following the single infusion. MRD-negative status is widely recognized as one of the strongest correlates of durable benefit in multiple myeloma, and achieving this level of disease clearance within weeks is an encouraging indicator of therapeutic potency. In the patient with the longest follow-up, the MRD-negative response was maintained through at least three months, supporting the potential for sustained disease control.
Beyond these clinical outcomes, the biological activity observed in the study underscores the novelty and promise of Kelonia’s platform. Following administration of KLN-1010, investigators recorded measurable CAR-T cell expansion within the patients, followed by the generation and persistence of memory-phenotype CAR-T cells. Importantly, these events occurred without the preparative lymphodepleting chemotherapy that is currently required for all approved ex vivo CAR-T therapies. Moreover, KLN-1010 bypasses the need for apheresis—a procedure in which immune cells must be extracted from the patient—and eliminates all ex vivo cell manufacturing, which is typically a multi-week, resource-intensive process.
Collectively, these findings point to a potentially groundbreaking shift in the delivery of CAR-T therapy, suggesting that the power of engineered cell therapies may soon be accessible through streamlined, outpatient-friendly treatment approaches.
Company Commentary on Clinical Progress
Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia Therapeutics, emphasized the significance of these early observations. “KLN-1010 is beginning to show the extraordinary clinical outcomes that may be possible with in vivo CAR-T therapy—early, deep responses from a single infusion without the barriers that limit access to traditional CAR-T treatments,” Friedman stated.
He noted that these first-in-human results not only highlight the therapeutic potential of KLN-1010 but also serve as strong validation for Kelonia Therapeutics proprietary in vivo gene placement system, the iGPS® platform. According to Friedman, the Kelonia Therapeutics platform has already supported the development of multiple partnered programs and is the foundation of Kelonia’s internal pipeline, demonstrating broad utility across therapeutic areas. “These initial data point to a potentially powerful medicine while also providing clear clinical validation of our iGPS platform,” he added. “We are encouraged by these first-in-human results and look forward to sharing additional study insights with the scientific and medical communities at the ASH Annual Meeting.”
Clinical Perspective from Study Leadership
Simon Harrison, MBBS, MRCP(UK), FRCPath(UK), FRACP, Ph.D., Director of the Centre of Excellence in Cellular Immunotherapy at the Peter MacCallum Cancer Centre and lead author of the abstract, also reflected on the significance of the preliminary data. He emphasized two key patterns emerging from the study: rapid MRD negativity and the sustained presence of memory-phenotype CAR-T cells.
“In these early patients, we are seeing both rapid MRD-negative responses and persistent memory-phenotype CAR-T cells, a combination that has been strongly prognostic for durable clinical benefit with existing CAR-T approaches,” Harrison said. He highlighted that the ability to achieve these outcomes without lymphodepleting Kelonia Therapeutics chemotherapy or CAR-T cell manufacturing underscores KLN-1010’s potential to deliver the same or greater therapeutic value with significantly reduced treatment burden.
Harrison noted that the approach taken by Kelonia could help reshape the standard of care for relapsed and refractory multiple myeloma by bringing CAR-T therapy within reach of patient populations that previously had limited access due to logistical or clinical constraints. He added that widespread deployment of an in vivo gene therapy approach may dramatically increase the number of patients who are able to benefit from CAR-T technology, including those who are too frail to undergo cell collection or intensive conditioning regimens.
The Potential Impact of In Vivo CAR-T Therapy
As multiple myeloma remains an incurable cancer, with most patients eventually relapsing despite advances in therapy, innovation in treatment modalities continues to be a critical area of need. Traditional CAR-T therapies targeting BCMA have delivered unprecedented response rates; however, challenges remain, including variable access, time constraints related to manufacturing, toxicities associated with conditioning chemotherapy, and the risk of treatment delays for patients with progressive disease.
The in vivo CAR-T model employed by KLN-1010 could mitigate several of these limitations. By enabling the patient’s T cells to be modified directly within the body, treatment may be administered more rapidly, potentially within days of clinical decision-making. The Kelonia Therapeutics removal of apheresis and ex vivo manufacturing also reduces procedural complexity and could lower overall treatment costs.
Furthermore, eliminating or reducing the need for lymphodepleting chemotherapy may improve tolerability and expand eligibility to patients who are currently not considered candidates for traditional CAR-T regimens. As the therapy matures and more trial data become available, KLN-1010 could represent a new frontier in CAR-T therapy—creating a model that is more scalable, accessible, and patient-friendly.
Presentation Details at ASH 2025
The preliminary findings from the inMMyCAR study will be presented during the Late-Breaking Abstracts Session at the ASH Annual Meeting. Full details are as follows:
Minimal residual disease (MRD)-negative outcomes following a novel, in vivo gene therapy generating anti–B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR)-T cells in patients with relapsed and refractory multiple myeloma (RRMM): Preliminary results from inMMyCAR, the first-in-human Phase 1 study of KLN-1010
Date: Tuesday, December 9, 2025
Time: 7:30 AM – 9:00 AM EST
Location: West Hall D2, Orange County Convention Center, Orlando
Session: Late-Breaking Abstracts Session
The presentation will provide additional details on safety, pharmacodynamics, CAR-T cell kinetics, and clinical outcomes for the initial patient cohort.
inMMyCAR is a Phase 1, open-label, dose-escalation clinical trial designed to assess the safety, tolerability, pharmacology and preliminary efficacy of a single dose of KLN-1010 in up to 40 patients. The primary endpoints are incidence and severity of treatment-emergent adverse events (TEAEs), including dose limiting toxicities (DLTs), and to establish the recommended Phase 2 dose of KLN-1010. KLN-1010 has been granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA). This Phase 1 clinical trial marks the first time KLN‑1010 will be evaluated in humans. Additional information and study site information may be found on clinicaltrials.gov (NCT07075185).
Multiple myeloma is a hematologic malignancy characterized by the proliferation of plasma cells in the bone marrow, leading to bone destruction, anemia, renal dysfunction, and immunosuppression. It is driven by complex genetic and epigenetic alterations that promote malignant cell survival and resistance to apoptosis. Relapsed and refractory multiple myeloma is characterized by clonal evolution, drug resistance, and increased disease heterogeneity, heightening the need for accessible, personalized therapeutic strategies.
KLN‑1010 is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting a protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR‑T treatments, KLN‑1010 is administered to patients via direct transfusion and is designed to generate durable CAR‑T cells inside the body after a single dose, potentially eliminating the need for long wait times to receive treatment. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and preconditioning chemotherapy.
Kelonia is a clinical-stage company pioneering a new wave of genetic medicines using its in vivo gene placement system (iGPS®). The company’s elegant, cutting-edge in vivo gene delivery technology uses an advanced lentiviral vector particle harboring envelope modification to improve in vivo gene transfer efficiency and tropism molecules to facilitate tissue-specific delivery. Kelonia is building a pipeline of genetic medicines across a range of diseases, with the bold goal of making CAR-T cell therapies accessible to every patient in need, when and where they need them. Its lead candidate, KLN-1010, is an in vivo anti-BCMA CAR-T therapy for multiple myeloma being evaluated in a Phase 1 clinical trial. Learn more about Kelonia at https://www.keloniatx.com/ and follow us on LinkedIn and X. Kelonia, iGPS, and inMMyCAR are trademarks of Kelonia Therapeutics, Inc.
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