INAD gene therapy moves one step closer

INAD gene therapy moves one step closer

By Patricia Wood

 December 2022

Work on a gene therapy for Infantile Neuroaxonal Dystrophy, called INAD, got a big boost in October when a London biotech company announced its intentions to help bring the treatment to market.


London-based Bloomsbury Genetic Therapies Limited, known as Bloomsbury, said it will advance its efforts by using 5 million pounds in seed financing from UCL Technology Fund. Bloomsbury is working on an adeno-associated virus-based (AAV) gene therapy named BGT-INAD for treating INAD.

INAD is a form of PLA2G6-Associated Neurodegeneration or PLAN, and it usually has an onset between the ages of 6 months and 3 years that progresses rapidly. Many affected children do not survive beyond their first decade.

Bloomsbury has three other AAV gene therapy programs in its pipeline and is raising money from investors and other sources to support the timely development of all four programs.

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

 London researchers
 University College London researchers at the 8th International Symposium on NBIA in October where they presented their work. L-R: Prof. Ahad Rahim, Dr. Apostolos Papandreou, Dr. Audrey Soo, Prof. Manju Kurjan, Dr. Robert Spaull.

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

Dr Audrey Soo, part of Professor Kurian's research group at UCL, also presented an update at the symposium to outline preparations for a gene therapy clinical trial testing BGT-INAD. She said the work is informed by an extensive retrospective natural history study with more than 300 INAD patients worldwide.

Also in October, Dr. Amy Geard presented the research findings at the 29th European Society of Cell and Gene Therapy Conference in Edinburgh. She won the 2022 Fairbairn Award for best presentation at the conference by an early-career UK researcher.

The UCL research has advanced understanding of INAD, including its key features and symptoms. Most importantly, the researchers have developed a meaningful disease-specific rating scale for INAD, along with discovering potential blood and cerebrospinal fluid biomarkers. Once the biomarkers are fully validated, they can be used as outcome measures in clinical trials, thus accelerating the development and approval of potential treatments for INAD patients.

Soo said she will continue developing and validating INAD biomarkers over the course of 2023.

Bloomsbury is working with the researchers on an accelerated timeline to bring its gene therapy research programs to clinical trials as soon as possible. It plans to complete comprehensive efficacy assessment for BGT-INAD in the INAD mouse model in the first quarter of 2023. The company will then focus on the required safety tests in animals. It hopes the accelerated clinical trial design will shorten the timeline to gain regulatory approval from the regulatory authorities such as the European Medicines Agency or the U.S. Food and Drug Administration so it can make the therapy commercially available to treat patients.

Bloomsbury keeps its website updated, so patients and families can see the latest developments at

UCL holds shares in Bloomsbury as part of the intellectual property agreement rights transfer. The UCL Technology Fund is an independent venture capital fund managed by Albion VC. The fund invests in intellectual property commercialization opportunities at UCL.


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