Eli Lilly and Company / Kelonia Therapeutics, Inc.

Multiple Myeloma

36K US cases/yr · $24.0B Global MM therapeutics market 2024

Disease Overview

Multiple myeloma (MM) is a hematologic malignancy of plasma cells that accumulate in the bone marrow, producing monoclonal immunoglobulin and causing end-organ damage characterized by the CRAB criteria (hypercalcemia, renal insufficiency, anemia, bone lesions). It is the second most common hematologic cancer in the United States. Although recent treatment advances including proteasome inhibitors, immunomodulatory drugs, anti-CD38 monoclonal antibodies, and most recently BCMA-directed CAR-T cell therapies and bispecifics have meaningfully extended survival, myeloma remains incurable for the vast majority of patients. Essentially all patients relapse, and outcomes in triple-class-refractory and penta-refractory disease are poor, with median overall survival measured in months. BCMA (B-cell maturation antigen) is selectively expressed on malignant plasma cells and has emerged as the dominant target across CAR-T, bispecific, and ADC modalities. Ex vivo autologous CAR-T manufacturing remains a bottleneck — vein-to-vein times, apheresis slot scarcity, lymphodepletion-related toxicity, and cost-of-goods restrict access. In vivo CAR-T approaches such as Kelonia's iGPS platform aim to bypass manufacturing by reprogramming T cells directly in the patient.

Competitive Landscape

The myeloma competitive landscape is the most crowded in hematology. Approved BCMA-directed CAR-Ts include Bristol Myers Squibb/2seventy bio's Abecma (idecabtagene vicleucel) and Johnson & Johnson/Legend Biotech's Carvykti (ciltacabtagene autoleucel), both autologous ex vivo products with demonstrated deep responses but significant manufacturing and access constraints. BCMA bispecifics include Pfizer's Elrexfio (elranatamab) and J&J's Tecvayli (teclistamab), alongside the GPRC5D-targeted bispecific Talvey (talquetamab). GSK's Blenrep (belantamab mafodotin), a BCMA ADC, was pulled from the US market in 2022 after the DREAMM-3 miss but returned to the market on the strength of DREAMM-7 and DREAMM-8 combination data. Into this field, multiple in vivo CAR-T programs are emerging including Umoja Biopharma's VivoVec lentiviral platform, Capstan Therapeutics (acquired by AbbVie in 2025) with a targeted lipid nanoparticle (tLNP) approach, Orna Therapeutics (Lilly partner) with circular RNA-LNP, and Interius BioTherapeutics (acquired by Nurix) with an in vivo lentiviral vector. Kelonia's iGPS (in vivo Gene Placement System) is a T-cell-selective lentiviral particle. Competitive positioning across the in vivo cohort hinges on delivery specificity, transgene expression durability, and avoidance of lymphodepletion.