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.

Bloomsburys 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 Companys 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 companys academic founders, a deep understanding of disease biology and world-class translational science resulting from the close collaboration between Bloomsburys academic founders. Additionally, the viral vectors in the Companys 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 industrys 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 Companys academic founders:

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 companys fundraising efforts (Series B, Series C, NASDAQ IPO and follow-on financing) which raised over $750M and built the companys pipeline as well as setting-up Orchards discovery research efforts, alongside managing the program leadership & management function.

Alongside him on the senior leadership team are:

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:

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