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Small interfering RNA – a change in the landscape of biotechnology

Summary of the impact

Work by Professor (now Sir) David Baulcombe and colleagues in The Sainsbury Laboratory at UEA was pivotal in discovering small interfering RNAs (siRNAs) and understanding the phenomenon of RNA interference (RNAi). These contributions not only revolutionised understanding of fundamental processes, from cancer to viral infections, but have already underpinned significant economic impacts, including:

  • Synthesis, design and sales of high-grade research chemicals, including oligonucleotides and kits, by several multinational Biotech companies.
  • Establishment of several companies whose product pipelines are driven by RNAi, and some of which have been acquired for sums > $1 billion.

Submitting Institution

University of East Anglia

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics, Microbiology

Using Biotechnology to Protect Plants against Invertebrate Pests

Summary of the impact

Durham has a long-standing record of research into improving the resistance of crop plants towards pests, which includes pioneering work on genetic engineering of plants for insect resistance. The CpTI gene developed in Durham for enhancing insect resistance in transgenic crops has had a major impact on Chinese agriculture, due to the widespread deployment of GM cotton containing genes encoding Bacillus thuringiensis (Bt) toxin and CpTI. The SGK 321 transgenic cotton line was approved for commercial growing in China in 1999, and by the current REF period Bt/CpTI cotton was grown on approximately 0.5 million hectares of land, representing approximately 15% of the total transgenic cotton grown (which in turn represented 67% of total cotton production). The economic value of Bt/CpTI cotton is estimated as approx. £600 million per year.

Submitting Institution

University of Durham

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Zoology
Agricultural and Veterinary Sciences: Crop and Pasture Production, Horticultural Production

CS2 Technologies to control plant parasitic nematodes

Summary of the impact

Plant resistance provides sustainable control of the $125bn annual world crop losses to nematodes to replace environmentally hazardous pesticides. Urwin and Atkinson have developed three biosafe resistance technologies that 1) suppress feeding success, 2) reduce root invasion and 3) suppress nematode development by RNA interference. We have developed GM agriculture with leading industry (Sinochem, Monsanto) and in emerging economies through free access to technology, capacity building initiatives, review of collaborative R&D plans (India) and regulatory approval of field trials (Uganda). The work has also influenced policy-makers in the UK and in Switzerland, leading to new security measures for GM field trials in these countries..

Submitting Institution

University of Leeds

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Genetics, Plant Biology
Agricultural and Veterinary Sciences: Crop and Pasture Production

Health, agriculture and industry benefit from Bristol’s groundbreaking molecular toolkit

Summary of the impact

The Basidio Molecular Toolkit developed at the University of Bristol has enabled the pharmaceutical industry to achieve the efficient genetic manipulation of a group of basidiomycete fungi (mushrooms and toadstools) and thereby produce medically important antibiotics and proteins cost-effectively. For example, GlaxoSmithKline's collaboration with the Bristol team saved 70,000 hours of research and development in getting a natural antibiotic called pleuromutilin to market. In China, the system is used to produce medicinal anti-cancer proteins from fungi in commercially viable quantities. In addition, government agricultural research programmes in the US and Ireland have adopted the toolkit to increase the efficiency of their search for disease-resistant crops in the interests of farmers, consumers and economies.

Submitting Institution

University of Bristol

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Genetics, Microbiology

Oxford Expression Technologies: making baculovirus expression accessible for protein production and vaccine development

Summary of the impact

Oxford Expression Technologies (OET) is a spin out company launched jointly by Oxford Brookes University (Brookes) and the Natural Environment Research Council (NERC) to exploit Intellectual Property (IP) in the field of protein expression using novel insect virus vectors. OET generates revenue through sale of kits, services & licences to a range of global customers including academia, research institutes, pharmaceutical and biotechnology companies. OET provides employment, invests in in-house Research and Development including funding collaborative PhD students, and generates royalty income streams for Brookes and NERC. Customers are able to produce multiple recombinant proteins to higher yields and quality than was otherwise possible and a number of companies are using the developments for the commercial production of vaccines and other uses.

Submitting Institution

Oxford Brookes University

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics
Medical and Health Sciences: Medical Microbiology

BioVex: UCL spin-out company develops viral cancer therapy to phase III trial and is sold for $1bn

Summary of the impact

UCL spin-out company BioVex was launched in 1999 to exploit research undertaken by David Latchman at the UCL Medical Molecular Biology Unit, Department of Biochemistry. (This department is now part of the Department of Structural and Molecular Biology, UCL/Birkbeck and Latchman is now Master of Birkbeck.) Biovex worked to develop inactivated herpes simplex viruses as therapies, and a promising dual-action oncolytic vaccine for solid tumours, OncoVEXGM-CSF, was taken into successful Phase II trials. In 2011 the company was bought out by Amgen for $1 billion — still the largest ever cash sale of a UK biotech — and Amgen has now taken this virus into a Phase III trial with promising initial results.

Submitting Institutions

University College London,Birkbeck College

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Medical and Health Sciences: Immunology, Medical Microbiology

02_Understanding of DNA methylation leads to (a) new reagents and (b) clinical trials for cancer treatments.

Summary of the impact

Impact on commerce: A patented technique for separating methylated and non-methylated DNA has been licensed and a kit brought to market, along with other commercial reagent licenses.

Impact on health and welfare: The demonstration that two mechanisms of epigenetic gene regulation, DNA methylation and histone acetylation, are linked, has led to trials of separate drugs known to affect each mechanism as a combined treatment for high-risk patients with myelodysplastic syndromes (MDS).

Beneficiaries: Companies have gained commercial benefit from licensing UoE IP to market products. High-risk MDS patients will benefit from improved treatment.

Significance and Reach: Commercial earnings across 4 companies from international sales in the period estimated at over [text removed for publication], mainly since 2010. Commercial significance includes the first commercially-available technique for separating methylated and non-methylated DNA.

The incidence of MDS is estimated at 3-4 cases diagnosed annually per 100,000 of the population in Europe (an estimated 26,000 individuals) and up to 20,000 new diagnoses per year in the USA. Incidence increases with age — up to 15 new cases annually per 100,000 in individuals aged over 70 years. MDS occurrence is increasing as the age of the population increases, so the significance of new therapies is high.

Attribution: All research was led by Adrian Bird at UoE. Reik (Babraham Institute) contributed to development of one of the licensed antibodies.

Submitting Institution

University of Edinburgh

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics

Fungus research: impacts on pest control, heritage conservation and public awareness of science

Summary of the impact

Research over the last 20 years by Jane Nicklin (née Faull) and her research group has established expertise in fungi, which has led to impacts in three areas: impacts on the licensing of commercial products for the control of insect pests which affect food crops, which have led to a new product being licensed in the US to the benefit of vine growers; impacts on heritage conservation, where the work has benefitted English Heritage, the National Trust and many other conservation groups; and impacts on public awareness and media engagement with science, in particular through her work with Channel 4's How Clean is your House? in 2009.

Submitting Institutions

University College London,Birkbeck College

Unit of Assessment

Biological Sciences

Summary Impact Type

Environmental

Research Subject Area(s)

Biological Sciences: Genetics, Microbiology, Zoology

12_Prospect of a cure for Rett syndrome has driven the formation of a charity and underpins clinical trials.

Summary of the impact

Impact on society (non-profit organisations) and public attitudes: The discovery of the reversibility of Rett syndrome in a mouse model for the disease has changed attitudes and awareness amongst families of sufferers and has led directly to the formation of two new charities: the Rett Syndrome Research Trust (US) and ReverseRett (UK).

Impact on health and welfare: Two new clinical interventions are being trialled with Rett syndrome patients.

Beneficiaries: Families living with Rett syndrome worldwide.

Significance and Reach: The research has given hope to thousands of families world-wide and has prompted an active philanthropic drive to fund research into a cure based on the UoE findings. The RSRT has raised $15 million since 2008. The incidence of Rett syndrome is 1 in 10,000 females. Some 16,000 individuals have Rett syndrome in the USA, and an estimated 2,400 in the UK.

Attribution: The research was carried out at UoE led by Adrian Bird. The critical underpinning paper was the UoE demonstration of reversibility (2007).

Submitting Institution

University of Edinburgh

Unit of Assessment

Biological Sciences

Summary Impact Type

Health

Research Subject Area(s)

Biological Sciences: Biochemistry and Cell Biology, Genetics

Development of genetic modification tools to enable bioengineering of clostridial species for improved healthcare, chemical commodity and biofuel manufacture.

Summary of the impact

Bacteria of the Clostridium genus are of pathogenic, medical and industrial importance. Development by University of Nottingham School of Life Science researchers of three patented methods for genetic manipulation of clostridial species has led to licensing agreements for commercial exploitation of the methodology to enhance strains for chemical commodity and biofuel production and for targeted cancer therapy. These methods are providing significant world-wide impact by facilitating commercial R&D investment and technology developments in fields ranging from healthcare, through chemicals manufacture, to the environment.

Submitting Institution

University of Nottingham

Unit of Assessment

Biological Sciences

Summary Impact Type

Technological

Research Subject Area(s)

Biological Sciences: Genetics
Technology: Medical Biotechnology
Medical and Health Sciences: Medical Microbiology

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