INAD gene therapy is one step closer

from | April 17, 2023

Abridged translation of an English article by Patricia Wood

Work on a gene therapy for infantile neuroaxonal dystrophy, called INAD, got a big boost in October when a London-based biotech firm announced its intention to help bring the therapy to market.

London-based Bloomsbury Genetic Therapies Limited, known as Bloomsbury, is looking to advance its efforts with the help of £5 million in seed funding from the UCL Technology Fund. Bloomsbury is working on a gene therapy based on adeno-associated viruses (AAV) called BGT-INAD to treat INAD.

INAD is a form of PLA2G6-associated neurodegeneration, or PLAN, that usually occurs between 6 months and 3 years of age and progresses rapidly. Many affected children do not survive their first decade of life. (...)

Bloomsbury benefits from the expertise of its academic scientists in gene therapy and rare diseases, including Professors Manju Kurian and Ahad Rahim of University College London. Kurian and Rahim have been working on a gene therapy treatment for INAD for eight years. The NBIA Disorders Association awarded researchers a $150,000 grant in 2014 to begin work. This was followed by £655,000 from the UK's Medical Research Council.

Image: Researchers from University College London at the 8th International NBIA Symposium in October 2022, where they presented their work.
L-R: Professor Ahad Rahim, Dr. Apostolos Papandreou, Dr. Audrey so, Professor Manju Kurian, Dr. Robert Spaull.

At the 8th NBIA International Symposium in Lausanne in October 2022, Rahim presented the promising data for BGT-INAD. Preliminary results show a marked improvement in survival and behavioral parameters in mice treated with BGT-INAD.

Dr Audrey so, who is part of Professor Kurian's research group at UCL, also gave an update on the preparations for a clinical gene therapy trial with BGT-INAD at the symposium. She said the work was based on an extensive retrospective study of the natural course of the disease with more than 300 INAD patients worldwide. (...)

UCL research has improved the level of knowledge about INAD, including its main features and symptoms. Most importantly, the researchers have developed a meaningful disease-specific rating scale for INAD and discovered potential biomarkers for blood and cerebrospinal fluid. Once fully confirmed, the biomarkers can be used as a measure of outcome in clinical trials, accelerating the development and approval of potential treatments for INAD patients. so said she will continue the development and validation of INAD biomarkers throughout 2023.

Bloomsbury is working with researchers on an accelerated timeline to bring its gene therapy research programs into clinical trials as quickly as possible. Bloomsbury plans to complete the comprehensive efficacy evaluation for BGT-INAD in the INAD mouse model in the first quarter of 2023. Subsequently, the company will focus on the necessary safety tests on animals. It hopes that the accelerated clinical trial design will shorten the time to approval by regulatory agencies such as the European Medicines Agency or the U.S. Food and Drug Administration, allowing it to make the therapy commercially available to treat patients.
Bloomsbury keeps its website up-to-date, so that patients and relatives can inform you about the latest developments. (...)

Full original version of the article and image source in December 2022 newsletter of the NBIA Disorders Association, p. 6/7):
Translated with the support of (free version)

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