Objective:

To summarize recent developments in cell and gene therapy, including partnerships, advancements in therapies, and research findings.

Approach:

• Bristol Myers and Cellares Partnership: Bristol Myers Squibb ended its partnership with Cellares for the commercial-scale manufacture of CAR T-cell therapy Breyanzi due to unmet manufacturing requirements.
• Sickle Cell Gene Therapy Study: A first-in-human gene therapy study showed a faster process for collecting and modifying blood stem cells for sickle cell disease, with promising results and no unexpected safety events.
• CAR T Cells Against Osteosarcoma: Deleting the Regnase-1 gene from CAR T cells improved tumor control in osteosarcoma models, suggesting a durable immune response.
• CD40 Stimulation in Solid Tumors: Combining CAR T-cell therapy with a CD40 agonist improved tumor control and survival in mouse models of pancreatic and triple-negative breast cancer.
• Bioinspired Nanoparticles for Gene Editing: Lipid nanoparticles delivered a base editor to reduce LDL cholesterol in preclinical models, showing fewer inflammation markers and no liver damage.
• 4basebio and Genezen Collaboration: 4basebio and Genezen expanded their collaboration to provide viral vector developers with access to synthetic, cell-free DNA starting materials.

Key Findings:

• Bristol Myers ended its partnership with Cellares, leading to job cuts.
• Sickle cell gene therapy demonstrated faster collection and modification of stem cells.
• Regnase-1 deletion in CAR T cells improved survival in osteosarcoma models.
• CD40 agonist combined with CAR T therapy enhanced tumor control in solid tumors.
• Bioinspired nanoparticles effectively delivered gene editing tools with minimal side effects.
• 4basebio and Genezen's collaboration aims to streamline viral vector development.

Limitations:

• The findings from preclinical studies may not directly translate to human outcomes.
• Further studies are needed to assess long-term effects and safety in human trials.