£1 million award to develop gene therapy for rare blood disorder FHL-2
12 Jun 2018, 9:31 a.m.
Professor Bobby Gaspar is to receive investment of up to £1million from the UCL Technology Fund (UCLTF) to support development of a gene therapy for familial haemophagocytic lymphohistiocytosis (FHL type 2 or ‘FHL-2’) through to a clinical trial.
£1 million award to push forward regenerative medicine research
13 Dec 2018, 10:19 a.m.
Researchers at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH) are to lead a new collaboration exploring how stem cells can be engineered to evade immune responses.
$225million investment for gene therapy spinout Orchard Therapeutics
19 Nov 2018, 11:17 a.m.
Orchard Therapeutics, has launched is initial public offering (IPO) on the Nasdaq stock exchange. The company, which specialises in developing gene therapies for complex rare diseases, sold 14.3 million shares to raise $225 million of investment.
£75,000 boost for Duchenne Muscular Dystrophy gene therapy research
8 Aug 2017, 4:03 p.m.
GOSH Biomedical Research Centre researcher Dr John Counsell has been awarded up to £75,000 of funding from the UCL Therapeutic Acceleration Support (TAS) Fund for a study exploring the use of novel vectors in gene therapy for Duchenne Muscular Dystrophy (
Accelerated approval request for a new drug for a rare childhood muscle disorder
9 Sep 2015, 4:41 p.m.
A drug for Duchenne Muscular Dystrophy (DMD), originally developed by BRC Theme Lead Professor Francesco Muntoni’s Consortium in the UK, has been filed by Sarepta Therapeutics for accelerated approval by the United States Food and Drug Adminstration (FDA)
AI bodysuit trial launched to help treat Duchenne muscular dystrophy
1 Feb 2018, 11:57 a.m.
A new artificially intelligent bodysuit to help understand how mobility is affected in boys with Duchenne will be trialled in a collaboration between researchers at Great Ormond Street Hospital (GOSH) and Imperial College London.
Autolus acquires rights to commercialise novel T-cell therapy developed at GOSH and UCLH
24 Apr 2018, 2:56 p.m.
The biopharmaceutical company Autolus has acquired global rights to develop and commercialize a novel T-cell therapy for the treatment of B-cell lymphomas and leukaemias.
Autolus report initial results for cutting-edge cell therapy for cancer
7 Dec 2018, 10:11 a.m.
Clinical trials of a pioneering new cancer therapy known as AUTO3, developed by UCL spinout company Autolus Therapeutics, have shown positive initial results.
BRC part of new centre to create next generation of AI leaders
22 Feb 2019, 4:15 p.m.
The GOSH Biomedical Research Centre (BRC) will form part of a new centre to apply artificial intelligence (AI) to healthcare and support the development of future leaders in AI.
BRC researchers develop a new rapid test for Alzheimer’s and Lewy Body Dementia
18 Dec 2015, 1:29 p.m.
National Institute for Health Research (NIHR) Great Ormond Street Hospital (GOSH) BRC (Biomedical Research Centre) researchers, working in collaboration with the BRC at NIHR Queen Square Dementia and the Wolfson Biomarker Dementia consortium at Institute
BRC researchers part of collaboration to improve efficiency of gene therapy products
26 Jul 2018, 3:56 p.m.
A new collaboration aiming to develop advanced technologies for manufacturing ex vivo gene therapy products has been established between researchers at UCL Great Ormond Street Institute of Child Health (ICH) and Plasticell, a developer of stem cell techno
Diagnosis with a rare disease - one that affects less than 1 in 2,000 people - can leave a family feeling isolated and alone. But we can support those families and make real progress by bringing the rare disease community together. In December 2019, the N
Cannabidiol trial shows reduction in seizures for children with Dravet syndrome
30 May 2017, 12:58 p.m.
A trial conducted in Europe and the USA has shown that cannabidiol – a drug derived from cannabis but with the psycho-active elements removed – reduces seizures in children with a form of drug resistant epilepsy, known as Dravet syndrome.
Catalyst Fellowships launched to support talented child health researchers
14 Dec 2017, 4:32 p.m.
The Catalyst Fellowships are an exciting new initiative supported by the National Institute for Health Research (NIHR) Great Ormond Street Hospital (GOSH) Biomedical Research Centre aiming to build the capacity of child health researchers and enabling the
Chancellor of the Exchequer George Osborne visits Great Ormond Street Hospital and meets young patients
21 Dec 2015, 12:23 p.m.
This morning, the Chancellor of the Exchequer George Osborne visited Great Ormond Street Hospital (GOSH) to announce a new £800 million boost to biomedical research through the National Institute for Health Research (NIHR).
Clinical trial to investigate treatment of acute lymphoblastic leukaemia to open at GOSH
28 Jun 2016, 2:12 p.m.
A Phase I clinical trial (CARPALL) has opened at Great Ormond Street Hospital (GOSH) to investigate the use of immunotherapy with genetically modified T cells for acute lymphoblastic leukemia (ALL). The trial is led by Professor Persis Amrolia, who is sup
Clinical trial for a new stem-cell based therapy for a rare skin condition proves promising
9 Sep 2015, 4:52 p.m.
Promising findings from a trial for a new stem-cell based therapy for a rare skin condition have been published in the Journal of Investigative Dermatology. The study, which involved intravenous injections of stem-cells, has led to an improvement in the q
Cutting edge new method reveals novel cause of Juvenile dermatomyositis
25 Jun 2018, 10:18 a.m.
A new in-depth method has helped shed light on the key role of B cells, a type of immune cell, in the rare childhood condition Juvenile dermatomyositis (JDM).
Developing clinical academic careers for nurses and allied health professionals
22 Feb 2019, 5:47 p.m.
Over 30 nurses and allied health professionals (AHPs) attended a training weekend hosted by GOSH’s Centre for Outcomes and Experience Research in Children’s Health Illness and Disability (ORCHID) earlier this month.
