Sangamo Therapeutics has received orphan medicinal product designation (OMPD) from the European Commission (EC) for its Investigational autologous Chimeric Antigen Receptor Regulatory T Cell (CAR-Treg) cell therapy, TX200, for solid organ transplantation.
The latest move follows the receipt of positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA).
TX200 is a collection of autologous regulatory T cells (Tregs) that are engineered to express an HLA-A2 Chimeric Antigen Receptor (CAR) to prevent immune-mediated rejection in HLA-A2 mismatched kidney transplants from a living donor.
It has been developed for reducing the transplant rejection risk by suppressing inflammation in the local area and promoting immunological tolerance.
The cell therapy is being evaluated in patients with HLA-A2 negative receiving a mismatched kidney from a living donor with HLA-A2 positive kidney.
Sangamo Development head Rob Schott said: “Patients who have received a solid organ transplant require lifelong surveillance and chronic immunosuppressive medications to manage the risk of transplant rejection.
“Our goal with TX200 is to create a transformative therapy that reduces the risk of organ rejection while reducing the patient burden from chronic immunosuppressive therapy.
“Achieving this important regulatory milestone takes us one step closer to realising that goal for patients.”
In March, the company dosed the first participant in the Phase I/II STEADFAST clinical study of TX200.
The open-label, multicentre, dose-ranging, single ascending dose study has been designed to evaluate the safety and tolerability of TX200.
In 2020, Sangamo and Novartis signed an exclusive, global licensing collaboration agreement to develop and commercialise genomic therapies for the treatment of neurodevelopmental disorders, including autism.