Bloomsbury Genetic Therapies first year: key strategic and operational achievements

14 November 2023

UCLB spinout Bloomsbury Genetic Therapies Limited has marked its one-year anniversary by outlining some key achievements.

Since its official launch last October with £5m in seed funding, the clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, has made important progress across all pipeline programs, demonstrating high performance execution and achieving significant value creation.

Key achievements at a glance:

  • One programme in the clinic: HORACE (Halting Ornithine transcarbamylase deficiency with Recombinant AAV in ChildrEn, NCT05092685), a phase 1/2 clinical trial evaluating BGT-OTCD, Bloomsbury’s investigational AAV-LK03 gene therapy, was successfully initiated this month by its collaborators, University College London (UCL).
  • One new pipeline programme in a large indication: Bloomsbury launched a new pipeline program, BGT-PD, an investigational gene therapy candidate for Parkinson’s Disease (PD), following compelling findings from a preclinical proof-of-concept study. BGT-PD relies on the same gene therapy vector encoding for the dopamine transporter (DAT) protein used in the Company’s BGT-DTDS program, which is being developed concurrently for the treatment of Dopamine Transporter Deficiency Syndrome (DTDS).
  • Five successful regulatory interactions: Bloomsbury successfully held three Scientific Advice meetings with the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its preclinical programmes BGT-DTDS, BGT-NPC, an investigational AAV9 gene therapy for Niemann-Pick Disease type C (NPC) and BGT-INAD, an investigational AAV9 gene therapy for Infantile Neuroaxonal Dystrophy (INAD). Bloomsbury also received meaningful feedback from Type B pre-IND interactions with the US Food and Drug Administration (FDA) for BGT-NPC and BGT-INAD.
  • Two completed preclinical efficacy studies: BGT-NPC and BGT-INAD completed short and long-term preclinical efficacy studies, demonstrating compelling results. Select program data were presented at 29th and 30th ESGCT Congresses, and the 26th ASGCT Annual Meeting.
  • Seven Orphan Drug Designations (ODDs) and four Rare Pediatric Disease Designations (RPDDs): Bloomsbury received EU and US ODDs and US RPDDs for each of its four rare disease gene therapy programmes.
  • Two pilot vector batches manufactured at partner CDMO: Bloomsbury successfully completed pilot vector manufacturing for its BGT-DTDS and BGT-INAD programs, marking the first step towards full-scale GMP manufacturing and preparation for single Phase 1/2/3 clinical trials.
  • Two grants obtained: Bloomsbury has built strong relationships with patient organisations in its focussed disease areas and received grant funding from Cure INAD UK and The Hide and Seek Foundation to support key research activities being undertaken in its BGT-INAD and BGT-NPC programs.

“I am proud of how much the Bloomsbury team of six employees has accomplished over the past year. The success is a testament to the expertise and experience of a dedicated team, the potential of Bloomsbury’s best-in-class programs, and is a validation of the strategic approach Bloomsbury is taking to develop gene therapies for rare diseases,” said Frank Armstrong, Chairman of Bloomsbury. “I am also excited for 2024, which will build on these achievements and is when we expect to achieve clinical proof of concept for BGT-OTCD, as well as further progress towards clinical readiness of our preclinical programmes.”

Sara Garcia Gomez, Senior Business Manager, UCLB, added: “It is amazing to see how much has ben achieved by the first anniversary of Bloomsbury GTx, spectacular progress in a record time. We’re now looking forward to what 2024 will bring.”

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