Autolus announces presentation of preliminary data showing early signs of clinical activity for a GD2-targeting CAR T cell therapy in a solid tumour setting at the AACR Annual Meeting

Phase 1 trial sponsored and managed by Cancer Research UK in paediatric patients with neuroblastoma


Autolus Limited (Autolus), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, today announced that Dr. Karin Straathof, Wellcome Trust Clinician Scientist at UCL Great Ormond Street Institute of Child Health, will present preliminary data at a Clinical Trials Plenary Session at the American Association for Cancer Research (AACR) Annual Meeting on April 17, 2018 in Chicago of an exploratory Phase 1 trial, sponsored and managed by Cancer Research UK, of a glycosphingolipid GD2-targeting programmed T cell product candidate in paediatric patients with neuroblastoma, including a discussion of early signs of clinical activity.

Details for the presentations are as follows:

CTPL04 - Novel Immuno-oncology Strategies Oral Abstract Presentation:

Title: "A Cancer Research UK phase I trial of anti-GD2 chimeric antigen receptor (CAR) transduced T-cells (1RG-CART) in patients with relapsed or refractory neuroblastoma”
Abstract Number: CT145
Date / Time: April 17, 2018, 11:05 AM - 11:25 AM
Presenter: Dr. Karin Straathof, Wellcome Trust Clinician Scientist at UCL Great Ormond Street Institute of Child Health

Autolus has worldwide commercial rights to the Phase 1 clinical data of the GD2-targeting programmed T cell product candidate as well as patents from UCL Business plc covering this program. This program has been designated as AUTO6 by Autolus. Cancer Research UK will continue to enrol patients to compete the current dose cohort of the Phase 1 clinical trial.

In parallel, Autolus is developing a next-generation T cell product candidate, which builds on the product candidate being explored in this Phase 1 study, by incorporating programming modules intended to enhance efficacy by extending persistence, as well as to address the layers of defence cancers deploy to evade T cell killing.

Dr Catherine Bollard, Director of the Center for Cancer and Immunology Research at the Children’s Research Institute, USA said:

“It is very encouraging to see anti-tumour activity in neuroblastoma, an indication where there are limited therapeutic options for patients with relapsed or refractory disease. This is of particular importance as this activity was observed in the absence of neurotoxicity which occurs with antibody-based approaches that target GD2."

Dr Christian Itin, CEO of Autolus, commented:

“First demonstration of clinical activity in a solid tumour indication is an important milestone. We intend to continue this program by initiating a Phase 1/2 clinical trial with the GD2 binder in a next-generation programmed T cell product candidate which is being designed with additional programming modules to enhance the efficacy, safety and persistence. We also plan to explore the utility of our next-generation product candidate in other adult and paediatric solid tumour indications.”

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About Autolus

Autolus is a private, clinical-stage, biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognise cancer cells, break down their defence mechanisms and attack and kill these cells. Autolus has a pipeline of products in development for the treatment of both haematological malignancies and solid tumours. For further information, please visit the company’s website at:

About UCL Business PLC

UCL Business PLC (UCLB) is a leading technology transfer company that supports and commercialises research and innovations arising from UCL, one of the UK’s top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL’s associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market. For further information, please visit:

About Cancer Research UK

• Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research.

• Cancer Research UK’s pioneering work into the prevention, diagnosis and treatment of cancer has helped save millions of lives.

• Cancer Research UK receives no funding from the UK government for its life-saving research. Every step it makes towards beating cancer relies on vital donations from the public.

• Cancer Research UK has been at the heart of the progress that has already seen survival in the UK double in the last 40 years.

• Today, 2 in 4 people survive their cancer for at least 10 years. Cancer Research UK’s ambition is to accelerate progress so that by 2034, 3 in 4 people will survive their cancer for at least 10 years.

• Cancer Research UK supports research into all aspects of cancer through the work of over 4,000 scientists, doctors and nurses.

• Together with its partners and supporters, Cancer Research UK's vision is to bring forward the day when all cancers are cured.

For further information about Cancer Research UK's work or to find out how to support the charity, please call 0300 123 1022 or visit


The study was supported by the teams at the UCL Great Ormond Street Institute of Child Health, the UCL Cancer Institute, Great Ormond Street Hospital NHS Foundation Trust, and the CRUK Centre for Drug Discovery with funding from CRUK, Neuroblastoma UK, the Great Ormond Street Children’s Charity, the National Institute for Health Research (NIHR) and the GOSH/UCL NIHR Biomedical Research Centre.

About Neuroblastoma

Neuroblastoma is a cancer that develops from immature nerve cells found in several areas of the body, and most commonly arises in and around the adrenal glands, which have similar origins to nerve cells and sit atop the kidneys. However, neuroblastoma can also develop in other areas of the abdomen and in the chest, neck and near the spine, where groups of nerve cells exist. Neuroblastoma most commonly affects children age five or younger, though it may rarely occur in older children. According to the American Cancer Society, there are approximately 700 new cases of neuroblastoma each year in the United States.

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