Therapy Areas: Cardiovascular
European Orphan Medicinal Product Designation Granted to Sangamo Therapeutics Investigational CAR-Treg Cell Therapy TX200 for Solid Organ Transplantation
21 July 2022 - - The European Commission has granted Orphan Medicinal Product Designation to TX200, a wholly-owned autologous Chimeric Antigen Receptor Regulatory T Cell (CAR-Treg) cell therapy product candidate for treatment in solid organ transplantation, US-based genomic medicine company Sangamo Therapeutics, Inc. (NASDAQ: SGMO) said.

Dosing of the first patient in the STEADFAST Phase 1/2 clinical study took place in March 2022, with the second patient dosing planned later this quarter.

The EC granted OMPD to TX200 following a positive opinion from the European Medicines Agency's Committee for Orphan Medicinal Products.

To qualify for orphan designation, a treatment must be intended for a life-threatening or chronically debilitating disease affecting fewer than five in 10,000 people.

Importantly, no satisfactory method of treatment must exist, or if such a method exists, the treatment must be of significant benefit to patients.

The OMPD status offers a range of incentives to encourage the development of orphan medicines, including protocol assistance on study protocols, potential fee reductions, and 10 years of market exclusivity upon regulatory approval.

TX200 is a cell therapy composed of autologous regulatory T cells (Tregs) engineered to express an HLA-A2 Chimeric Antigen Receptor, with the aim of preventing immune-mediated rejection in HLA-A2 mismatched kidney transplants from a living donor.

TX200 is intended to reduce the risk of transplant rejection by suppressing local inflammation and promoting immunological tolerance to the graft.

TX200 is being assessed in HLA-A2 negative patients receiving a mismatched HLA-A2 positive kidney from a living donor.

TX200 CAR-Treg cells are expected to localize to the graft and activate upon binding to the HLA-A2 antigen.

Through their ability to regulate the immune system, TX200 cells may protect the graft from immune-mediated rejection and reduce or eliminate the need for lifelong treatment with immunosuppressants. The first patient in the ongoing STEADFAST Phase 1/2 study was dosed in March 2022.

Kidney transplantation is the treatment of choice for patients with end-stage renal disease who must otherwise remain on long-term dialysis. Approximately 21-26% of transplanted kidneys are estimated to be HLA-A2 mismatched.

To prevent graft rejection, transplanted patients are treated with lifelong immunosuppressive therapy, which impacts the body's immune system and is associated with multiple side effects, including an increased risk of severe life-threatening infections, malignancies, cardiovascular disease, and other drug-related toxicities, such as nephrotoxicity, which can impact the function and survival of the newly transplanted kidney.

Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a genomic medicines pipeline.


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