TX200 / Sangamo Therap - LARVOL DELTA

Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term) (clinicaltrials.gov) - P=N/A | N=11 | Active, not recruiting | Sponsor: Sangamo Therapeutics | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplant Rejection • Transplantation

STEADFAST Study Update: A Phase 1/2 Clinical Trial of Regulatory T Cells Expressing a Chimeric Antigen Receptor Directed Towards HLA-A2 in Renal Transplantation (WTC 2025) - "By end Jan 2025, 3 of 5 subjects in DL2 or above achieved complete tapering of mycophenolate mofetil (MMF) to tacrolimus monotherapy compared to none of the control subjects; it is too early to assess the success of the other 2 subjects. These data demonstrate that TX200 has a favorable safety profile, with preliminary biomarker data consistent with the proof of mechanism and establishment of tolerance in the renal graft. The study completes 4Q2025."

Clinical • P1/2 data • Nephrology • Transplantation

Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term) (clinicaltrials.gov) - P=N/A | N=11 | Enrolling by invitation | Sponsor: Sangamo Therapeutics | N=21 ➔ 11

Enrollment change • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplant Rejection • Transplantation

STEADFAST: Safety & Tolerability Study of Chimeric Antigen Receptor T-Reg Cell Therapy in Living Donor Renal Transplant Recipients (clinicaltrials.gov) - P1/2 | N=22 | Active, not recruiting | Sponsor: Sangamo Therapeutics | Trial completion date: Mar 2026 ➔ Oct 2025 | Trial primary completion date: Nov 2024 ➔ Jul 2024

Trial completion date • Trial primary completion date • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplant Rejection • Transplantation

Safety & Tolerability Study of Chimeric Antigen Receptor T-Reg Cell Therapy in Living Donor Renal Transplant Recipients (clinicaltrials.gov) - P1/2 | N=22 | Active, not recruiting | Sponsor: Sangamo Therapeutics | Recruiting ➔ Active, not recruiting | N=42 ➔ 22

Enrollment change • Enrollment closed • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplant Rejection • Transplantation

Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term) (clinicaltrials.gov) - P=N/A | N=21 | Enrolling by invitation | Sponsor: Sangamo Therapeutics

New trial • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplant Rejection • Transplantation

Sangamo Therapeutics Announces Strategic Update and Reports Preliminary First Quarter 2023 Financial Results (Businesswire) - "Dosed third patient in cohort 1 in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor...Plan to share initial data from cohort 1 by the end of 2023; Dosing of patients to support primary analysis is complete in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A. A pivotal readout is expected in mid-2024, with Pfizer anticipating BLA and MAA submissions in the second half of 2024."

BLA • European regulatory • P1/2 data • Genetic Disorders • Immunology

Long-Term Follow-Up of Patients who have received an Autologous Antigen-Specific Chimeric Antigen Receptor T Regulatory Cell Therapy (CAR- Treg therapy, TX200-TR101) in a prior clinical study. (clinicaltrialsregister.eu) - P2 | N=15 | Ongoing | Sponsor: Sangamo Therapeutics France SAS

New P2 trial • Chronic Kidney Disease • Nephrology • Renal Disease • Transplant Rejection • Transplantation

Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation. (PubMed, Gene Ther) - "Concomitant tacrolimus did not impair TX200-TR101 Treg survival or their ability to inhibit peripheral blood mononuclear cell (PBMC) engraftment. These data demonstrate that TX200-TR101 is specific, stable, efficacious, and safe in preclinical models, and provide the basis for a first-in-human study."

Journal • Preclinical • Graft versus Host Disease • Immunology • Solid Organ Transplantation • Transplant Rejection • Transplantation • HLA-A

Study Design: Human Leukocyte Antigen Class I Molecule A02-Chimeric Antigen Receptor Regulatory T Cells in Renal Transplantation. (PubMed, Kidney Int Rep) - "The STEADFAST study represents the next frontier in adoptive cell therapies. TX200-TR101 holds great potential to prevent immune-mediated graft rejection and induce immunologic tolerance after HLA-A02-mismatched renal transplantation."

Journal • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Solid Organ Transplantation • Transplant Rejection • Transplantation • HLA-A

DESIGN OF STEADFAST STUDY EVALUATING TX200-TR101, A CHIMERIC ANTIGEN RECEPTOR T REGULATORY CELL THERAPY FOR LIVING DONOR RENAL TRANSPLANT RECIPIENTS (ESOT 2021) - P1/2 | "CAR-Tregs represent a new frontier in cell therapy. The ST EADFAST study, the first-ever CAR-Treg clinical study, is currently recruiting patients at several European sites."

Clinical • Late-breaking abstract • Graft versus Host Disease • Immunology • Nephrology • Transplant Rejection • Transplantation • HLA-A

Safety & Tolerability Study of Chimeric Antigen Receptor T-Reg Cell Therapy in Living Donor Renal Transplant Recipients (clinicaltrials.gov) - P1/2; N=42; Recruiting; Sponsor: Sangamo Therapeutics; N=21 ➔ 42

Clinical • Enrollment change • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplantation • CD4

Safety & Tolerability Study of Chimeric Antigen Receptor T-Reg Cell Therapy in Living Donor Renal Transplant Recipients (clinicaltrials.gov) - P1/2; N=21; Recruiting; Sponsor: Sangamo Therapeutics

Clinical • New P1/2 trial • Chronic Kidney Disease • Immunology • Nephrology • Renal Disease • Transplantation • CD4