3 May 2016

UCL Business PLC (“UCLB”), the technology transfer company of University College London (“UCL”), and F-Prime Capital Partners have launched Orchard Therapeutics, a biotechnology company that will develop transformative gene therapies for life threatening orphan diseases. Orchard Therapeutics has secured £21-million in Series A financing led by F-Prime.

As part of its launch, Orchard has announced formal partnerships with UCL, Great Ormond Street Hospital for Children NHS Foundation Trust (“GOSH”), University of California Los Angeles (“UCLA”), The University of Manchester and Boston Children’s Hospital for the development of these transformative gene therapies.

Orchard’s development programmes focus on restoring normal gene function in primary immune deficiencies, metabolic diseases and haematological disorders. This pioneering technology uses a sample of the patient’s own stem cells, which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient’s body. The use of the patient’s own (autologous) cells removes the need to search for a matching stem cell donor, which can take months or even years.

Orchard’s lead programme is for the treatment of the rare and fatal disorder called severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID), sometimes referred to as “bubble baby” syndrome in the press. Children with the disorder must be protected from any infections due to their highly fragile immune system. In a first-in-man clinical study, global leaders in the field at UCL/GOSH and UCLA have shown significant immune reconstitution and 100% survival in 32 patients treated, as of March 2016.

Bobby Gaspar, Professor of Paediatrics and Immunology at UCL’s Institute of Child Health/GOSH and Orchard’s Chief Scientific Officer said, “Orchard’s founding scientists, also including Professors Adrian Thrasher and Waseem Qasim from UCL and GOSH, have been pioneering ex-vivo autologous haematopoietic stem cell gene therapy for the last 20 years. We have seen very promising effects in several different diseases and are hopeful that this technology will change the lives of many children with life-threatening conditions in the future.

Orchard’s Series A financing has been led by F-Prime Capital, a leading venture capital investment fund with extensive experience in rare diseases and gene and cell therapies, with support from UCLB and additional participation from the UCL Technology Fund. Alex Pasteur and Ben Auspitz, partners at F-Prime Capital, commented, “We are delighted to have played a central role in the formation of Orchard. We view ex-vivo gene therapy as an exciting field for investment in the creation of transformative medicines”.

Cengiz Tarhan, Managing Director of UCLB, added, “We are very excited to see UCL’s breakthrough treatments move forward in a commercial environment in a way that will benefit patients globally. The formation of Orchard represents the culmination of decades of research at UCL and its partners and we are delighted to be able to work with F-Prime Capital on this important venture.

The work at the UCL Institute of Child Health has been developed through the laboratories of Professors Bobby Gaspar, Adrian Thrasher and Waseem Qasim in collaboration with GOSH, UCLA and King’s College London, with funding and support from the National Institute for Health Research (“NIHR”) Biomedical Research Centre (“BRC”) at Great Ormond Street Hospital for Children NHS Trust and University College London, the Great Ormond Street Hospital Children’s Charity, the Medical Research Council, the Wellcome Trust and the Histiocytosis Research Trust.

Find out more www.orchard-tx.com

About Orchard Therapeutics
Orchard Therapeutics is a biotechnology company focused on the development and commercialization of transformative ex-vivo gene therapy medicines for patients with orphan diseases.

Visit www.orchard-tx.com for more information.

About F-Prime
Prime Capital is a leading international venture capital firm with a focus on healthcare & life sciences that is backed by Fidelity Investments. F-Prime Capital has invested over US$ 1 billion through healthcare dedicated venture funds. F-Prime Capital has extensive experience investing in rare diseases and gene and cell therapies, including prior investments in Ultragenyx, Adaptimmune, Blueprint Medicines, Proteostasis, Dimension Therapeutics, REGENXBio, Precision Biosciences, FoldRx, and Caribou Biosciences.

Visit www.fprimecapital.com for further information.

About UCL
UCL was founded in 1826. We were the first English university established after Oxford and Cambridge, the first to open up university education to those previously excluded from it, and the first to provide systematic teaching of law, architecture and medicine. We are among the world’s top universities, as reflected by performance in a range of international rankings and tables. UCL currently has over 35,000 students from 150 countries and over 11,000 staff. Our annual income is more than £1 billion.

www.ucl.ac.uk – Follow us on Twitter @uclnews – Watch our YouTube channel YouTube.com/UCLTV

About UCL Institute of Child Health
The UCL Institute of Child Health (ICH) which, together with its clinical partner Great Ormond Street Hospital for Children (GOSH), forms the largest concentration of children’s health research in Europe.  The inspirational mission of the UCL Institute of Child Health is to ‘improve the health and well-being of children, and the adults they will become, through world-class research, education and public engagement’.

Please visit www.ucl.ac.uk/ich for more information.

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 commercialization process of technologies from laboratory to market.

For further information, please visit: www.uclb.com – Twitter: @UCL_Business

About Great Ormond Street Hospital for Children NHS Foundation Trust and Great Ormond Street Hospital Children’s Charity
Great Ormond Street Hospital for Children Foundation Trust is the UK’s leading centre for treating sick children, with the widest range of specialists under one roof. With the UCL Institute of Child Health, we are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future. The Great Ormond Street Hospital Children’s Charity needs to raise vital funds to enable the hospital to continue to provide the very best care for its young patients and their families through helping to rebuild and refurbish the hospital, buy vital equipment, fund pioneering research and provide support for staff and families.

About the NIHR Great Ormond Street Biomedical Research Centre
The Biomedical Research Centre (BRC) at Great Ormond Street Hospital (GOSH) and the UCL Institute of Child Health (ICH) enables the translation of basic scientific discoveries in laboratories into ‘first-in-man’ or ‘first-in-child’ clinical studies. Our research aims to accelerate discoveries into the basis of childhood rare diseases and to develop novel diagnostics, imaging techniques and new treatments, including cellular and gene therapies.

The GOSH BRC is funded by the National Institute for Health Research (NIHR) and supports various programmes such as the Gene and Cell Therapy Facility, GOSomics, GOSgene and the Somers Clinical Research Facility, as well as providing financial support to individual researchers and training opportunities. Visit the GOSH BRC website: http://www.gosh.nhs.uk/research-and-innovation/nihr-great-ormond-street-brc