Achilles Therapeutics

Achilles Therapeutics launches with funds of £13.2 million to develop immunotherapies for cancer

The UCL Technology Fund (UCLTF) announced its participation in the formation of Achilles Therapeutics Ltd, led by SYNCONA LLP and CANCER RESEARCH TECHNOLOGY (CRT).

The new private company will bring together world-class research from UCL (University College London) and the Francis Crick Institute, funded by Cancer Research UK and the National Institute for Health Research (NIHR).

Achilles Therapeutics will design therapies to target truncal tumour neo-antigens – unique flags to the immune system present on the surface of every cancer cell*, which were first discovered by Cancer Research UK and the NIHR University College London Hospitals (UCLH) Biomedical Research Centre (BRC) funded scientists at the Francis Crick Institute and UCL Cancer Institute.

www.achillestx.com

Freeline Therapeutics

Freeline Therapeutics is a biopharmaceutical company based at the UCLB Royal Free London Hospital focused on the development of liver-directed gene therapies.

Gene therapy has the potential to transform lives for people with severe diseases by providing a long-lasting, safe and reliable source of enzymes to the blood. The Company’s next-generation AAV gene therapy platform has been developed by Professor Amit Nathwani, Professor of Haematology at UCLB, and it builds on the successful haemophilia B phase I/II trial conducted by him with St. Jude Children’s Research Hospital, Memphis. The results of the study, published in the New England Journal of Medicine, demonstrated that all ten treated haemophilia B patients showed safe and sustained expression of blood clotting Factor IX after a single administration. The company will develop and commercialise gene therapies for bleeding and other debilitating disorders. Freeline’s shareholders include Syncona LLP, UCLB and UCL Technology Fund.

www.freelinetx.com/

Orchard Therapeutics

Orchard is a biotechnology company dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases

Orchard Therapeutics is a spin-out from the Institute of Child Health (ICH) at UCL, commercialising a gene therapy platform with the potential to cure a range of rare childhood genetic disorders in a single treatment. The company’s programmes use the potential of ex-vivo autologous haematopoietic stem cell gene therapies to restore normal gene function in severe and life-threatening inherited disorders, including immunodeficiencies and a range of neurological disorders, and have demonstrated excellent safety and efficacy in ongoing clinical trials. Orchard’s shareholders include F-Prime Ventures, UCLB and the UCL Technology Fund.

www.orchard-tx.com

Canbex Therapeutics

Canbex Therapeutics is dedicated to treating spasticity in multiple sclerosis (MS) and other disorders.

Spasticity is a debilitating and painful symptom that consists of involuntary spasms and stiffness of limbs and torso. The goal of the Canbex lead programme is to improve the lives of people with this serious and incurable disorder, and set a new standard in the treatment of spasticity.
The company’s lead compound, VSN16R, is currently in a Phase II study to determine its efficacy in relieving spasticity in people with MS. VSN16R was shown to be safe and well tolerated in a Phase I human safety trial in healthy volunteers that was completed in 2014.
In February 2015, Canbex entered into an agreement with Ipsen SA, a leading global specialty pharmaceutical firm with expertise in spasticity and a commitment to providing better treatments for this important and poorly served medical need.
Canbex is supported by UCL, the Wellcome Trust, MS Ventures (the corporate venture arm of Merck KGaA), Esperante Ventures and the UCL Technology Fund. The company has also received grant funding from InnovateUK, and Fast Forward, the drug development arm of the US National Multiple Sclerosis Society.

www.canbex.co.uk

CGQ Targeting

The team is developing a series of small-molecule compounds which bind to novel genetic targets associated with genomic dysfunctions which underlie cancer

Prof Stephen Neidle of the UCL School of Pharmacy and his team have developed a series of small-molecule compounds which bind to novel genetic targets associated with genomic dysfunctions which underlie cancer. This has the potential to open a new area of cancer therapeutics against well-established, but previously undruggable cancer targets.
The UCL Technology Fund is directly supporting Prof Neidle’s lab with funding of up to £1m to deliver to a pre-clinical stage a first-in-class, optimised drug that will have superior efficacy against human pancreatic cancer, one of the most significant areas of unmet need in medicine. Standard current drug treatment (chemotherapy) has limited effects at best on improving patient survival in this devastating disease; the overall goal of this commercialisation program is to make a significant difference to survival for pancreatic cancer patients. The drug under investigation also has applicability to many other cancer types.

p47-CGD Gene Therapy

The team is developing a novel gene therapy for the p47phox-deficient variant of chronic granulomatous disease (CGD).

Prof Adrian Thrasher and his team at the UCL Great Ormond Street Institute for Child Health have developed a novel gene therapy for the p47phox-deficient variant of chronic granulomatous disease (CGD). A significant unmet need exists for patients who inherit this disease, as there is no existing curative gene therapy, stem-cell transplantation is not indicated for most patients and, in the absence of a cure, expensive lifetime prophylaxis against infection is required – and even then, recurrent infections and extended hospitalisations are common.
With direct funding from the UCL Technology Fund, Prof Thrasher and colleagues will be completing the final preclinical and manufacturing steps required to take this gene therapy into the clinic; as well as supporting a first-in-man clinical trial which itself has the potential to transform the lives of patients.

MediaGamma

MediaGamma is developing an enterprise grade reinforcement machine learning platform

MediaGamma, a specialist in AI and Machine Learning solutions, is developing an enterprise grade reinforcement machine learning platform, delivering machine decisions in under 5 milliseconds for a new class of business problems.

A spin-out from UCL’s Computer Science department founded on research by Dr. Jun Wang, MediaGamma already has partnered with a 1st tier telecoms provider to deliver high value data products and are processing over 700GB of data per day.

By enhancing data and decision making they are building a strong pipeline across telecoms, adtech and finance.

www.mediagamma.com

Intrinsic

Intrinsic is developing a new type of all silicon non-volatile memory

The Resistive Random Access Memory (RRAM) technology has shown 400x lower switching energy and 6 times lower voltage than FLASH memory, whilst being highly compatible with current semiconductor manufacturing processes.
This ground breaking technology is being developed in Anthony Kenyon’s group within Electronic and Electrical Engineering.

The UCL Technology fund have invested to introduce a commercial mentor as the business prepares to spin-out and to fund a number of technological developments.

Infectious Disease Biomarkers

A project to develop a simple test to rule out active TB

On presentation with symptoms of tuberculosis (TB), patients are immediately put onto strong antibiotics and it currently takes up to 6 weeks to determine whether or not this infection is present – and in the vast majority of cases it isn’t.

A simple test to rule out active TB could have the potential to significantly change clinical practice both in the developed and developing world. This PoC project is supporting the development of a panel of RNA (and potentially protein) biomarkers which could not only rule out TB on presentation, but could be used to monitor disease progression once a patient is being treated.

Normalisation of pathological neovasculature in ocular disease and cancer

Abnormal growth of blood vessels is a key cause of blindness in several ocular diseases of the retina and macula. The gold-standard treatment for these diseases is anti-VEGF ocular injections (usually monthly), which reduce blood vessel growth.

A new target protein involved in pathological neovascularisation has been discovered, and its inhibition acts to return blood vessels to their normal state; and work on this target is currently progressing towards a clinical trial in age-related macular degeneration. However, this approach may also be useful to enhance the efficacy of drugs for treatment of solid tumours by normalising the often highly disorganised vasculature therein. This PoC is supporting clinical and commercial workup towards developing the therapeutic as a platform technology.

BloomsburyAI

BloomsburyAI is automating expertise by building systems able to capture knowledge from unstructured and structured data and allow interrogative access in natural language.

Built on the research of founder Guillaume Bouchard and UCL’s Sebastian Riedel the company has a world leading machine reading capability.

The UCL Technology Fund invested in BloomsburyAI’s seed round together with IQ Capital, Fly Ventures and SeedCamp amonst others.

Blood-brain barrier delivery technology for small molecules and biologics

Over 98% of known drug molecules are unable to enter the brain, rendering a broad range of CNS diseases untreatable by traditional pharmacological mechanisms. Neurodegenerative diseases, brain cancer and mechanical- or hypoxia-induced brain trauma remain areas of enormous unmet clinical need; and a mechanism which could successfully carry drugs – which would otherwise be unable to cross the BBB – into the brain at relevant concentrations could provide enormous patient benefit, and have material commercial prospects.

This PoC project aims to experimentally and commercially validate a novel, annexin-based method for carrying therapeutic molecules into the brain.

Cell therapy for peripheral nerve repair

Hundreds of thousands of people in the developed world are affected by severe peripheral nerve damage, resulting in paralysis and loss of sensation, and often accompanied by chronic pain. Current therapies are successful in fewer than half of cases.

A cell therapy is under development at UCL for repair of severe peripheral nerve damage, mimicking nerve structure and better enabling functional recovery.

This PoC project includes a set of product and process optimisation steps, and a critical experiment to underpin the case for further development.

New bromodomain-focused cancer target for acute myeloid leukaemia

Acute myeloid leukemia (AML) is an aggressive cancer of the bone marrow and blood characterised by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells.

AML affects people of all ages, but predominantly occurs in people over 60, in whom survival is poor. A number of therapies are under development for AML, but none have yet replaced the gold-standard chemotherapeutic combination which supports survival rates of just 15% beyond 5 years for over-60s.

This PoC project seeks to establish a scientific and commercial case for the development of a novel small molecule cancer therapeutic with potential to treat this devastating disease.

Next generation linker technology for protein-drug conjugation

There is significant clinical utility in conjugating drugs to proteins, particularly in the antibody-drug conjugate (ADC) space, and in oncology where there is substantial utility in carrying a drug which may otherwise be highly toxic directly into cancer cells. However, a number of challenges remain to be solved, including the optimisation of product homogeneity and serum stability, and the ability to selectively conjugate potentially multiple different types of drugs or effector molecules to a single protein.

This PoC project is supporting the commercial, IP and technical development of a next-generation, site-specific protein-drug conjugation platform which has the potential to provide a competitive and versatile solution for generating a broad range of homogenous and multi-drug-enabled protein therapeutics.