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Birmingham Post
Birmingham Post
Business
Abigail Turner

Bristol University spin-out launches to advance development of rare disease platform

Bristol University spin-out Scarlet Therapeutics has raised seed funding from Science Creates Ventures and Meltwind to advance development.

Founded by blood scientists Professor Ash Toye and Professor Jan Frayne, Scarlet will use the funding to build a pipeline of novel therapies to treat rare diseases.

Therapeutic red blood cells (tRBCs) are very similar to standard red blood cells but carry additional proteins within them to provide a therapeutic benefit. Red blood cells have pervasive reach throughout the body and a long life of up to 120 days, and expressing therapeutic proteins inside the tRBCs keeps them hidden from the immune system.

Professor Toye and Professor Frayne have worked on the RESTORE study, led by NHS Blood and Transplant, with the University of Bristol and Cambridge and other partners, which is investigating transfusion of lab-grown blood into patients.

Read more: Bristol named a 'hot spot' for university spinouts in new report

Scarlet is initially targeting two rare metabolic diseases hyperammonemia and hyperoxaluria. Hyperammonemia is where patients can’t remove toxic ammonia from their system, leading to a range of neurological symptoms and, in severe cases, life-threatening complications. Hyperoxaluria is a condition where there is too much oxalate in the urine and is either caused by a rare inherited disorder of the liver (primary hyperoxaluria) or where excess oxalate is absorbed into the gastrointestinal tract and then excreted in the urine (secondary hyperoxaluria).

The platform also has the potential to treat other metabolic diseases requiring enzyme replacement therapy, as well as cancer and autoimmune diseases.

Alistair Irvine, chief executive of Scarlet Therapeutics, said: “Our game-changing therapeutic red blood cell-based technology is a new modality to treat targets of high value and unmet need. tRBCs have unique qualities; not only are they able to reach all parts of the body, delivering therapeutic benefit to where it is needed, but they are enduring – as their predicted 120 day long life will allow dosing every 2-3 months.

"Because the proteins are hidden inside the therapeutic red blood cell, they are also shielded from the immune system. Our approach allows the cells to be maximally loaded with therapeutic proteins without damaging the properties of the cells and so should be more effective. This funding enables us to further develop our technology to offer patients with debilitating health conditions more effective, longer-lasting treatments.”

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