Cell Therapy Landscape Evolves as Major Players Persist Amid Industry Exodus

In the wake of high-profile departures from the cell therapy space, including Takeda and Novo Nordisk, a core group of pharmaceutical giants and emerging biotechs continue to push the boundaries of this innovative treatment modality. Despite recent setbacks, companies like Bristol Myers Squibb, Gilead, and Johnson & Johnson are doubling down on their investments, while smaller players such as Immatics, Kyverna Therapeutics, and Cabaletta Bio are making significant strides in expanding cell therapy applications beyond oncology.

Big Pharma Leads the Charge in CAR T Development

Bristol Myers Squibb (BMS), Gilead, and Johnson & Johnson (J&J) remain at the forefront of CAR T therapy development, each boasting FDA-approved products and robust pipelines. BMS, with its two commercial CAR T assets targeting different proteins (Abecma for BCMA and Breyanzi for CD19), continues to expand its capabilities. The company's recent $1.5 billion acquisition of Orbital Therapeutics aims to advance in vivo cell reprogramming technologies.

Gilead, through its subsidiary Kite, has strengthened its position with strategic partnerships and acquisitions. A recent $120 million deal with Chinese cell therapy specialist Pregene and the $350 million acquisition of Interius BioTherapeutics underscore Gilead's commitment to in vivo CAR T development. The company's lead candidate, anito-cel, partnered with Arcellx, has shown promising results in multiple myeloma trials.

J&J's Carvykti, developed in partnership with Legend Biotech, is rapidly gaining market share in multiple myeloma treatment. With $963 million in worldwide sales last year, Carvykti is supported by strong clinical data, including a recent Phase III study showing 89% of treated patients had no detectable cancer cells after three years.

Expanding Horizons: Cell Therapy Beyond Oncology

While CAR T therapies have primarily focused on blood cancers, companies are now exploring applications in autoimmune diseases. Kyverna Therapeutics is leading this charge with KYV-101, a CAR T candidate targeting CD19 on B cells. Recent Phase II/III data in generalized myasthenia gravis (gMG) showed a 100% response rate, setting what analysts call "a new efficacy standard in the field."

Cabaletta Bio is pursuing a broad autoimmune program with its CABA-201 (rese-cel) therapy. The company presented promising Phase I/II data at the American College of Rheumatology meeting for dermatomyositis, systemic sclerosis, and systemic lupus erythematosus. Cabaletta is now advancing rese-cel into registrational studies for multiple autoimmune indications.

Novel Targets and Approaches in Oncology

Immatics stands out with its focus on the PRAME protein, expressed in over 50 different cancers. The company's lead candidate, anzu-cel, is in a Phase III trial for cutaneous melanoma, with potential FDA submission planned for 2027. Immatics is also exploring combinations with Moderna's mRNA cancer vaccines, highlighting the potential for synergistic approaches in cell therapy.

As the cell therapy landscape continues to evolve, these companies are demonstrating resilience and innovation in the face of industry challenges. Their persistent efforts are expanding the potential applications of cell therapies, offering hope for patients with a wide range of difficult-to-treat conditions.