Bloomsbury Genetic Therapies launches with Seed financing of £5 million to develop potentially curative gene therapy treatments for rare neurological and metabolic diseases

Bloomsbury Genetic Therapies launches with Seed financing of £5 million to develop potentially curative gene therapy treatments for rare neurological and metabolic diseases

London, UK, 7 October 2022 – Bloomsbury Genetic Therapies Limited (“Bloomsbury”), a  biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, based on clinically proven gene therapy technologies, today announced its launch with £5 million Seed financing led by UCL Technology Fund.

Bloomsbury’s innovative approach is designed to optimise therapeutic efficacy and safety, enable high manufacturability, accelerate development timelines and maximise regulatory success to create a pipeline of highly differentiated first- or best-in-class programs. The Company’s lead programs are liver and CNS targeted gene therapies:

Liver targeted therapies:       BGT-OTCD: Ornithine Transcarbamylase Deficiency (OTCD)

CNS targeted therapies:        BGT-DTDS: Dopamine Transporter Deficiency Syndrome (DTDS)

BGT-NPC: Niemann-Pick Disease Type C (NPC)

BGT-INAD: Infantile Neuroaxonal Dystrophy (INAD)

These programs are underpinned by the unique medical insights derived from the internationally recognised clinics run by the company’s academic founders,  a deep understanding of disease biology and world-class translational science resulting from the close collaboration between Bloomsbury’s academic founders. Additionally, the viral vectors in the Company’s programs have been designed with well-characterised adeno-associated virus (AAV) capsids that have demonstrated success in clinical and/or commercial settings. They have also been combined with innovative cassettes and routes of administration designed for optimal therapeutic effect.

“Bloomsbury is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “Our approach has been designed with a single goal in mind - to deliver the promise of gene therapy to rare disease patients by leveraging our industry’s existing state-of-the art technology and we have already attracted a highly talented team of founders and experienced scientific and business professionals to the Company to that effect as we progress our first liver targeted gene therapy program, BGT-OTCD for the treatment of Ornithine Transcarbamylase Deficiency, into the clinic.”

“Our commitment to Bloomsbury is a great example of our strategy to seek to build truly innovative companies anchored by exceptional science and experienced teams,” added  Leigh Brody, Investment Manager of UCL Technology Fund. “We are excited by the potential to develop best-in-class products in the gene therapy space.”

Bloomsbury was founded as a spinout from UCL, and was supported by UCL Business, who helped foster the progression of these translational projects, supporting the team whilst managing the intellectual property, and making this deal possible. Bloomsbury is underpinned by world-leading gene therapy and rare disease expertise from the Company’s academic founders:

• Professor Paul Gissen - Professor in Paediatric Metabolic Diseases at UCL. He is also an Honorary Consultant and research lead in paediatric metabolic diseases at Great Ormond Street Hospital (GOSH), lead for specialist clinics in Niemann Pick type C and Batten diseases, an NIHR Senior Investigator and a UK Chief Investigator for a number of industry clinical trials
• Professor Manju Kurian - Professor in Neurogenetics at UCL and NIHR Research Professor. She is also the Head of Molecular Neurosciences at UCL Great Ormond Street Institute of Child Health (GOS ICH), an Honorary Consultant in Paediatric Neurology at GOSH, Lead of Accelerating Novel Therapies theme at GOSH Biomedical Research Council and head of an internationally renowned specialist service in the UK for genetic movement disorders in children
• Professor Ahad Rahim - Professor of Translational Neuroscience at UCL, Head of Research Department of Pharmacology and Wellcome Chair of Pharmacology at UCL and co-founder of UCL’s GeneTxNeuro Viral Vector Core Facility
• Professor Simon Waddington - Professor of Gene Therapy at UCL and Chair of UCL Cell & Gene Therapy Therapeutic Innovation Network

“After years of working relentlessly to identify and develop potential treatments for OTCD, NPC, DTDS and INAD, we are excited to have partnered with the team at Bloomsbury and are confident that these programs are in safe hands to be progressed through the development stage and towards regulatory approvals, with an ultimate goal of providing curative therapies to patients,” added Professors Gissen, Kurian, Rahim and Waddington.

Senior leadership

Bloomsbury is led by Co-Founder & Chief Executive Officer, Adrien Lemoine, who brings 20 years of experience in pharma and biotech, including senior commercial, strategy and operation roles at AstraZeneca and GSK and joined the Company from Orchard Therapeutics where he served as Chief Business Officer. In this role he played a leading part in the company’s fundraising efforts (Series B, Series C, NASDAQ IPO and follow-on financing) which raised over $750M and built the company’s pipeline as well as setting-up Orchard’s discovery research efforts, alongside managing the program leadership & management function.

Alongside him on the senior leadership team are:

• John Ilett, who serves as Bloomsbury’s Chief Operating Officer and Chief Legal Officer. John, who was most recently Chief Legal Officer and Chief of Staff at Orchard Therapeutics, has over 30 years of experience in pharma and biotech both in private practice and working in-house, with biopharma companies including Oxford GlycoSciences, Cephalon, BioMarin and Consort Medical. His responsibilities have included the building and leading of legal, intellectual property and corporate compliance functions as well as the management of human resources, quality, security and facilities functions.  John’s experience includes private and public fundraisings, IPOs and secondary listings, international mergers and acquisitions, complex commercial and patent litigation, rare disease product launches and the in-licensing and out-licensing of a number of gene therapy products.
• Leigh Shaw, who serves as Bloomsbury’s Senior Vice President, Head of Regulatory Affairs and Non-Clinical GLP Safety/Toxicology. Leigh has 30 years of industrial experience in the development of gene therapies and therapeutic vaccines for a range of neurological and orphan indications.  Most recently he has held positions at Nightstar Therapeutics, United Neuroscience and GenSight Biologics where he played a leading role in the successful transition of products from research into the clinic, end of Phase 2 negotiations with US FDA and EMA, and Marketing Authorisation Application in the EU.

Board of Directors

Bloomsbury has also assembled an impressive trio of industry leaders as Independent Directors to assist management in driving the business forward; namely:

• Frank Armstrong, who serves as Chairman of the Board.  Frank has held Chief Executive roles with five biotechnology companies, both public and private, including Fulcrum Pharma, CuraGen, Bioaccelerate, Provensis and Phoqus. He also held senior executive positions at Merck Serono, Bayer and Zeneca. Frank is currently the Chairman of BioCaptiva and Faron Pharmaceuticals, is a Non-Executive Director of Newcells Biotech and ECO Animal Health Group plc, as well as a member of the Senior Advisory Board at Healthcare Royalty Partners.
• Michael Diem, who serves as independent Non-Executive Director. Michael is Chief Business Officer and Principal Finance Officer at Century Therapeutics. Before joining Century, he held senior positions at Amicus Therapeutics, Aevi Genomic Medicine, AstraZeneca and GSK. He currently serves as a Board Director at Venatorx Pharmaceuticals.
• Andrea Spezzi, who serves as independent Non-Executive Director. Andrea is the Co-Founder, President and CEO of the discovery stage start-up, Rejuvitas Inc. She co-founded and was Chief Medical Officer of Orchard Therapeutics, where she was responsible for leading the successful development of Libmeldy®, and has also held senior positions at Takeda and GSK.