Bloomsbury Genetic Therapies Announces Orphan Drug Designations of BGT-DTDS for the Treatment of Dopamine Transporter Deficiency Syndrome
– ODD granted in the United States and the European Union –
London, UK, 26 January 2023 – Bloomsbury Genetic Therapies Limited (Bloomsbury), a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, today announced that both the United States Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug Designation (ODD) for BGT-DTDS, the company’s neuron-targeted AAV2 investigational gene therapy for the treatment of Dopamine Transporter Deficiency Syndrome (DTDS). BGT-DTDS has completed preclinical efficacy studies and preparations for a first-in-human clinical trial are ongoing.
In the US, ODD is granted by the FDA’s Office of Orphan Products Development to promote the development of products that may offer therapeutic benefits for diseases with a prevalence of fewer than 200,000 individuals per year. Orphan drug designation provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States, if granted FDA approval.
Similiarly, in the EU, ODD is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). It is intended to encourage the development of drugs that may provide significant benefits to patients suffering from rare, life-threatening diseases. If approved for marketing, this designation will provide ten years of marketing exclusivity and also provide special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.
“These Orphan Drug Designations recognise the significant unmet need in patients in the United States and the European Union living with DTDS; a devastating disease with poor prognosis,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We look forward to continuing to investigate BGT-DTDS and to leveraging the benefits that ODD brings, including significant developmental benefits and the provision of post-approval market exclusivity.”
“DTDS is a rare, often poorly-diagnosed, devastating neurological condition caused by a faulty gene that affects brain cells. Infants with DTDS are rarely able to learn to walk or speak and there are currently no effective treatments, with many children sadly dying before reaching adulthood,” said Professor Manju Kurian, Professor of Neurogenetics at University College London and National Institute of Health and Care Research (NIHR) Research Professor. “The promise of gene therapy for DTDS is one we are very excited about, particularly given the excellent preclinical data we have amassed. We are hopeful that it could offer patients the real potential of being a transformative, or even curative, therapy.”
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About Bloomsbury Genetic Therapies (Bloomsbury):
Bloomsbury is a clinical-stage biotechnology company, developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies. The Company was spun out of University College London and launched in October 2022 with £5M in Seed funding from UCL Technology Fund and is underpinned by world-leading gene therapy and rare disease expertise from the Company’s academic founders, Professor Paul Gissen, Professor Manju Kurian, Professor Ahad Rahim and Professor Simon Waddington. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com
BGT-DTDS is a neuron-targeted AAV2 investigational gene therapy designed to provide a potentially curative solution to DTDS patients following a one-time intra-brain injection to the substantia nigra and ventral tegmental area. BGT-DTDS has completed preclinical efficacy studies and preparations for a first-in-human clinical trial are ongoing.
About Dopamine Transporter Deficiency Syndrome (DTDS)
DTDS is a rare, autosomal recessive childhood neurological disorder with significant disease burden, characterised by a severe parkinsonian movement disorder. DTDS results from biallelic mutations in the SLC6A3 gene which encodes the dopamine transporter (“DAT”), a key protein regulating dopaminergic signalling. Dopamine re-uptake by DAT is the principal mechanism by which dopamine is removed and recycled from synapses. Mutations to SLC6A3 result in DAT dysfunction and major dysregulation of dopamine neurotransmission, leading to hyperkinetic movement disorders in the early stages of the disease, and then evolution of Parkinson’s disease-like features (slowing of voluntary movements, loss of facial expression, tremor, stiffness) and eye movement disorders such as oculogyric crises. The disease is often fatal in childhood to teenage years due to unexplained death in sleep or respiratory failure. There are currently no disease-modifying treatments nor approved therapies for DTDS, and patients derive little clinical benefit from available symptom control medications. The disease is currently poorly diagnosed, due to symptoms similarity with other movement disorders including cerebral palsy.