Biotech is one of the most promising parts of the British drug industry, not least according to the investors who continue to pump vast sums into the sector, despite the looming shadow of Brexit.
In the first eight months of this year alone it received nearly ￡1.6 billion (US$2.03 billion), compared with ￡1.2 billion for the whole of last year.
An unassuming building in a science park near the Hertfordshire town of Stevenage is hosting four biotech firms that hope to achieve a major breakthrough for the field — and go some way to justifying that faith from investors.
The premise of biotech is radical: It harnesses living organisms to fight diseases. These firms — Adaptimmune Therapeutics PLC, Cell Medica Ltd, Autolus Therapeutics PLC and Freeline Therapeutics Ltd — are working on so-called living medicines that use human cells and genes to treat conditions ranging from hard-to-treat cancers to hemophilia and eye diseases.
They are based at a British government-funded cell and gene manufacturing center, which opened in May and is the largest such collaborative center in the world. Cell and gene therapies can fix genetic defects and re-engineer patients’ cells to recognize and attack tumors and other diseases.
There are more than 60 biotech firms specializing in cell and gene therapy in the UK, making it the second-biggest cluster in the world after the US.
In Stevenage, Adaptimmune, Cell Medica and Autolus all focus on cancer, while Freeline is working on therapies for bleeding disorders.
Another UK firm, London-based Nightstar Therapeutics PLC, has a gene therapy in late-stage studies to treat a rare retinal disorder that leads to blindness.
Keith Thompson, CEO of Cell and Gene Therapy Catapult (CGT), which set up the Stevenage center, believes that turning cells and genes into living medicines can offer cures rather than just treating symptoms — and could be exported by Britain around the world.
He has described it as “an absolute revolution.”
He is optimistic, despite the looming threat of Brexit.
“We’re trying to take the UK science into a new industry and not let it bleed offshore,” Thompson said.
Simon Pegg, director at Adaptimmune, which is developing T-cell therapies for cancer, said that the re-engineered cells start working straight away and cancer patients can see benefits within a week.
In T-cell therapy, white blood cells called T-cells are taken from a patient. They are refrigerated and shipped to be reprogrammed to combat cancer cells and then frozen under liquid nitrogen before being transported back to the clinical site for infusion into the patient.
However, there are challenges. Living therapies are expensive to make and unlike traditional drugs cannot be stored in blister packs in a pharmacy.
Along with others, CGT is working hard to scale up the new therapies so they become routine treatments in Britain. The Stevenage center would eventually be able to make 6,000 patient doses per year.
Three new government-funded treatment centers, where patients would receive cell and gene therapies, are also in the works — in the north, Manchester, and the Midlands and Wales.
There has been a string of positive news recently.
In October, a stem cell gene therapy for severe inherited blood disorders that is being developed by London-based Orchard Therapeutics PLC, a spin-off from University College London, was given priority status by industry regulator the European Medicines Agency.
In September, NHS England said that it would make Novartis International AG’s gene therapy Kymriah available for children and young people with a form of leukemia.
NHS England Chief Executive Simon Stevens called the CAR-T therapy as a “true game-changer.”
The industry has a long way to go — only a dozen cell and gene therapies are on the market in Europe, but there could be more soon, with 1,003 clinical trials of new therapies under way worldwide, half of them for cancer.
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