https://innovateuk.blog.gov.uk/2016/01/27/uk-companies-advance-development-of-regenerative-medicines-and-cell-therapies/

UK companies advance development of regenerative medicines and cell therapies

Innovate UK looks for innovative regenerative medicine, cell and gene therapy projects

Genmanipulation
Genmanipulation

With our 2015 competition ‘Developing Regenerative Medicines and Cell Therapies’, Innovate UK will invest up to £8 million in collaborative R&D projects that address critical challenges faced by UK companies developing regenerative medicines and cell therapies as clinical treatments and commercial products.

The competition aimed to advance the commercial development of new treatments and underpinning technologies by supporting a number of projects that connect the research pathway, from early discovery to clinical application and commercial supply.

Regenerative medicine restores normal organ function

Regenerative medicine is a broad group of innovative medical approaches, including cell and gene therapies and tissue engineering that aim to replace or regenerate human cells, tissues or organs to restore or establish normal function.

Huge promise to treat chronic illnesses

Regenerative medicines and cell therapies have demonstrated unprecedented promise to treat many areas of unmet medical need and chronic burden, estimated to take up nearly 75% of all UK health system costs.

Recent successes against cancer encourages commercial investment

Success in recent small-scale clinical trials of cell-based cancer immunotherapies has encouraged increased commercial investment, whereas the development of new gene-editing technologies shows the increasing capability, growth and opportunity in the field.

Innovate UK support commercialisation of promising discoveries

Cell cultures for gene therapy

Cell cultures for gene therapy

Turning promising discoveries into products, services and sustainable businesses is crucial if the UK is to become a global leader in the field, calling for truly innovative thinking and strong interdisciplinary collaboration.

Five exciting projects win funding

The five projects detailed below have succeeded in the final assessment of the competition, and their grant award confirmation is subject to the offer conditions being met.

  1. Next generation DNA constructs for the rapid and safe manufacture of AAV vectors for Regenerative Gene Therapy

Lead name: Cobra Biologics Ltd

Partner names: Touchlight Genetics Ltd

Proposed project costs: £507,600

Proposed project grant: £318,106

Gene therapy is becoming an increasingly important method of treatment for a variety of major unmet medical needs. Especially in the areas of inherited and rare diseases and diseases of the eye, conditions which are life threatening or significantly diminish quality of life. Adeno-associated virus (AAV) vectors are currently the delivery vehicle of choice for gene therapy treatments but the advancement these treatments into clinical trials is currently hampered by the time and expense required to manufacture these vectors.

The proposed collaboration between Cobra and Touchlight will develop a fast and less expensive route to manufacture of AAV vectors, which will enable the acceleration of more potential products into clinical testing. This in turn will increase the chances of treatment being developed for a whole range of these currently intractable diseases.

  1. Development of an Industrial Manufacturing Platform for Adeno-Associated Virus (AAV) production to support Regenerative Gene Therapy products

Lead name: Cobra Biologics Ltd

Partner names: Centre for Process Innovation Limited

Proposed project costs: £1,796,783
Proposed project grant: £1,403,616

Adeno-associated virus (AAV) vectors are currently the delivery vehicle of choice for gene therapy treatments but the advancement of these treatments into clinical trials is currently hampered by the lack of scalability needed to manufacture these vectors.

The proposed collaboration between Cobra and CPI will develop the scientific understanding to allow scalable flexible process to be developed to manufacture AAV vectors. This will enable the acceleration of more potential products into clinical testing and ultimately new medicines. This in turn will increase the chances of treatment being developed for a whole range of these currently intractable diseases.

  1. Enhanced Vector Bioprocessing Technologies for Cell and Gene Therapies

Lead name: Synpromics Ltd

Partner names: Cell Therapy Catapult

Proposed project costs: £1,963,206
Proposed project grant: £1,577,724

Evolving therapeutic approaches of cell and gene therapy are harnessing the power of viruses in order to modify genomes of cells to produce a therapeutic effect. Such therapies are starting to show efficacy in the clinic, but one of the key challenges to their widespread use is the ability to make large quantities of virus at a low cost.

This project seeks to address this challenge by creating new methods of producing large quantities of virus at a low cost. It brings together Synpromics, a synthetic biology company based in Edinburgh, and the Cell Therapy Catapult, one of the UK’s network of Catapult centres focused on developing and growing a cell and gene therapy industry in the UK.

  1. Stem cell-derived exosomes for regenerative medicine

Lead name: ReNeuron Limited

Partner names: University College London, Cell Therapy Catapult

Proposed project costs: £2,498,924
Proposed project grant: £2,104,240

ReNeuron is a leading UK regenerative medicine company currently conducting clinical trials with a novel human neural stem cell (hNSC) product for the treatment of stroke and limb ischaemia.

These stem cells also produce small parcels of biologic material called exosomes. CTX-derived exosomes have been found to have functional properties in laboratory models of cancer, particularly glioblastomas. It may therefore be feasible to use the exosomes as an alternative class of regenerative medicine that has a long shelf-life and application to a wide range of diseases.

In this project, we will tackle how to purify exosomes in large enough quantities cost- effectively at scale and then complete preclinical studies for a clinical trial application in glioblastoma. If successful, our project will benefit patients with the target disease as well as the UK regenerative medicine industry.

  1. CAR Therapy for T Cell Malignancies

Lead name: Autolus Ltd

Partner names: University College London

Proposed project costs: £2,175,539
Proposed project grant: £1,682,111

T-cells are immune cells in our bodies whose function is to "seek and destroy" cells that are infected by viruses. Since T-cells actively move around our bodies looking for infected cells, medical science has long tried to make these T-cells attack cancer cells. Because cancer cells usually come from normal cells in our bodies without virus infections, T-cells don't normally attack them.

By taking T-cells from a blood sample and "re-programming" them using genetic engineering, medical science has found a way of using T-cells to attack cancer. These engineered T-cells are called "CAR T-cells" and they can be given back to the patient as a drip. Once back in the patient, they "seek and destroy" cancer cells as if they were virus-infected cells.

CAR T-cells seem to be a very effective new form of cancer treatment. Cancers can come from any cells in our bodies. Even T-cells can become cancer cells and some of these cancers are called T-cell lymphomas. This project deals with the particular problem of CAR T-cells attacking T-cell lymphomas. Scientists working on this project have found a way of making CAR T-cells that attack the cancerous T-cells but leave plenty of normal healthy T-cells alone.

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