Objective:

To report on recent advancements in cell and gene therapies, including the approval of Tregzi and other notable studies.

Approach:

• FDA Approval of Tregzi: The FDA approved Tregzi, the first Treg-based immunotherapy for adults with blood cancers undergoing allogeneic hematopoietic stem cell transplantation.
• Clinical Trial Support: The approval is supported by the PRECISION-T trial, which involved 187 adults and demonstrated improved chronic graft-versus-host disease-free survival.
• Epigenetic Therapy for FSHD: Epicrispr Biotechnologies reported early evidence that EPI-321 may increase lean muscle volume in patients with facioscapulohumeral muscular dystrophy.
• CAR T Therapy for Rare Blood Cancers: A new CAR T-cell therapy targets mutant calreticulin in myeloproliferative neoplasms, showing selective killing of disease-driving cells.
• CAR T Therapy for Glioblastoma: A CAR T-cell therapy targeting GPNMB has shown strong activity in preclinical models of glioblastoma, attacking both tumor cells and supportive immune cells.
• Controlled CRISPR Systems: Researchers developed small-molecule-controlled CRISPR systems that allow for on-demand genome editing in human cells and mouse models.

Key Findings:

• 78% of patients treated with Tregzi were alive without moderate or severe chronic GVHD at one year.
• EPI-321 showed an average increase of approximately 0.8 pounds of muscle mass in early trial patients.
• CAR T cells targeting mutCALR selectively killed disease-driving cells while sparing healthy cells.
• Anti-GPNMB CAR T cells controlled glioblastoma tumors and showed prolonged responses in preclinical models.
• The PRINCE system allowed for controlled genome editing with minimal background activity.

Interpretation:

Conclusion: