SINGAPORE - A local gene therapy start-up has clinched a deal of more than $1.2 billion with a global pharmaceutical giant to develop and eventually commercialise its treatments for rare genetic diseases.
Carmine Therapeutics, formed last year and believed to be the first gene therapy company in South-east Asia, and Takeda Pharmaceutical Company, an R&D-driven, Japanese multinational pharmaceutical company, inked the deal on June 19.
Gene therapy is a treatment where genes are injected into patients to fight or prevent diseases, instead of using chemical drugs or surgery.
Carmine developed a novel technology that uses tiny particles released by red blood cells, called extracellular vesicles, to deliver genes to parts of the body that need to be treated.
Carmine is unable to reveal the rare genetic diseases they are working on at this point.
Once the particles reach the diseased cells or tissues, the genes help them build therapeutic proteins to restore the tissues' normal functions .
Carmine is believed to be the first company in the world to harness red blood cell extracellular vesicles as vehicles for gene therapy.
Most gene therapy treatments use viruses to deliver genes, but that is undesirable because some patients may develop adverse immune responses to the viruses or reject them, said Carmine's founding vice-president, Dr Ronne Yeo.
In Carmine's therapy, only red blood cells from O-negative blood in blood banks are used because it has the lowest risk of causing adverse reactions to patients, he added.
Additionally, the vesicles from red blood cells are also able to pack larger genes compared with viruses, are cheaper to manufacture, and can access more organs than other vehicles.
The vesicles' ability to hold and deliver genes and their advantages over viral vehicles were discovered by two Singaporean researchers, Dr Minh Le and Dr Shi Jiahai, both scientific co-founders of Carmine.
Their findings were published in scientific journal Nature Communications in 2018.
The start-up is also believed to be the first gene therapy company in Asia to use non-viral vehicles.
Carmine's technology is called Red Cell EV Gene Therapy (Regent), where EV stands for extracellular vesicles.
"Developing alternative gene therapy delivery vehicles like the Regent platform that could address the challenges of (virus-based) gene therapy is critical to one day delivering next-generation cures for rare diseases," said Takeda Rare Diseases Drug Discovery Unit head Madhu Natarajan.
Carmine is based in Singapore and Boston, Massachusetts, which is a global hub of biotech.
Since it was formed last year, Carmine has been further developing the vesicles, engineering them to tackle different rare genetic diseases, and conducting pre-clinical studies. The company is also aiming to treat cancers with gene therapy in the future.
Mr Lin Xiangqian, Carmine's founding chief executive, said Takeda's funding and collaboration will help the start-up further optimise its technology and accelerate it towards clinical trials and commercialisation of the treatments.
"We are working very hard for patients with rare genetic diseases, and we are working to put locally developed biotech inventions on the world map," he added.