Mesoblast acquires chimeric antigen receptor (CAR) platform technology for precision-enhanced cell products

Mesoblast Limited (Nasdaq: MESO; ASX: MSB) said it has acquired an exclusive worldwide license to a patented chimeric antigen receptor (CAR) technology platform aimed at enhancing its therapeutic mesenchymal lineage stromal cell (MSC) products. The company plans to incorporate engineered CARs to improve target specificity and augment MSCs’ inherent immunomodulation and tissue-regeneration properties.

CAR-MSC technology to enhance targeting and potency

Mesoblast’s MSC platforms are designed to treat tissue-specific inflammatory diseases, leveraging the cells’ homing capabilities and immunomodulatory effects. The company said genetically engineering CAR constructs into MSCs is intended to substantially enhance targeted homing to inflamed tissue, with the goal of increasing potency.

Mesoblast said foundational CAR-MSC work was developed by investigators at Mayo Clinic and published in Nature Biomedical Engineering. The investigators identified CAR-MSCs with potential for enhanced tissue-specific targeting in inflammatory and autoimmune diseases, including inflamed bowel. Mesoblast said this creates an immediate opportunity to develop products with greater potency for ulcerative colitis or Crohn’s disease.

The company also said it plans to use CAR-MSCs engineered to express CD19 on their surface to induce remission in lupus nephritis and other B-cell autoimmune diseases, where durable, effective and safe immunomodulation is described as highly desirable.

Exclusive license terms and support from Mayo Clinic

Mesoblast obtained worldwide exclusive rights to the CAR-MSC intellectual property developed at Mayo Clinic through the acquisition of a startup formed specifically to advance the technology. As part of the exclusive license, Mayo Clinic will provide in-kind support to further advance the technology and resulting products, including GMP manufacturing activities.

Mesoblast said the acquisition was completed through the issuance of ASX ordinary shares.

Company context: MSC platform and existing product

Mesoblast described itself as a developer of allogeneic (off-the-shelf) cellular medicines for severe and life-threatening inflammatory conditions. It said its proprietary mesenchymal lineage cell therapy technology platform responds to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple immune-system effector pathways, reducing damaging inflammatory processes.

The company highlighted Ryoncil (remestemcel-L-rknd) for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older, describing it as the first FDA-approved MSC therapy. It noted that full prescribing information is available at www.ryoncil.com.

Pipeline and partnerships

Mesoblast said it is developing additional cell therapies across distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell platforms. It cited ongoing development of Ryoncil for additional inflammatory diseases, including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. It also said rexlemestrocel-L is being developed for heart failure and chronic low back pain.

The company said it has commercial partnerships in Japan, Europe and China.

Intellectual property and manufacturing capabilities

Mesoblast said it holds a global intellectual property portfolio with more than 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, manufacturing methods and indications, with protection extending through at least 2044 in major markets.

On manufacturing, the company said its proprietary processes produce industrial-scale, cryopreserved, off-the-shelf cellular medicines with defined pharmaceutical release criteria intended to be available to patients worldwide.

Forward-looking statements

The company included standard forward-looking statement language, noting that the press release contains statements about future events and financial performance that involve risks and uncertainties. It said actual results may differ materially from those expressed or implied by forward-looking statements and that it does not undertake obligations to publicly update or revise such statements.