RARE – Adrien Lemoine 

Adrien is the Co-Founder and CEO at Bloomsbury Genetic Therapies, a spin-out biotech of University College London, focusing on the development of treatments for patients suffering from rare neurological and metabolic diseases, with a pipeline of first- or best-in-class gene therapy programs. 

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Key Takeaways 

Adrien trained as an Engineer in France, before moving quite early on into consulting in the field of biotech/pharma where he has been for the past 20 years. Following his consultancy experience, he moved to big pharma working for GSK for 10 years and it was there that he was part of the set-up of their rare disease unit. This was his first experience in the world of rare diseases and gene therapies. 

Adrien then did a stint with AstraZeneca before being approached about a project that turned out to be Orchard Therapeutics with a few ex-GSK colleagues at the helm. He went for it and was lucky to be one of the first members of the company, which has been a great success with some commercial programs and gave him the opportunity to delve deeper into the field of gene therapy and rare diseases. 

Adrien then left Orchard to found Bloomsbury Genetic Therapies. The project was coined about 3 years ago, in collaboration with their current seed investor UCL Tech Fund (managed by AlbionVC), a UK-based investor dedicated to investing and funding innovation created at UCL. They thought that there must be a more efficient way to develop gene therapies for rare diseases, with Adrien having experienced first-hand all the challenges during his time at Orchard. Regulatory challenges, difficulties with manufacturing and also the time it has taken for the first gene therapy to be approved. In cases of rare disease where there may be no other option, the promise of gene therapy is so important because you go after the root cause, you fix the faulty gene. Ultimately, patients can’t wait too long as sadly a lot of rare diseases are life-threatening in the first couple of decades of life. 

Now that there have been a few approved gene therapy products, Adrien thought there must be a way to leverage some of the learnings from those approvals to move faster to translation to the clinic and eventually to approval. 

Adrien goes on to talk about how the idea for Bloomsbury Genetic Therapies germinated. Speaking with the investors at AlbionVC and with his co-founders who are all academics at UCL, they all had this vision of bringing something quickly to patients by leveraging the building blocks of gene therapy that have already been proven to work and so they created Bloomsbury. Creating something from scratch that is very pragmatic and efficient in terms of time and money in developing gene therapies for rare diseases. 

When asked about taking the step from CBO to Co-Founder of a company, Adrien relates this to his previous experience at Orchard being one of the first employees but highlights the difference between setting something up and the ability to really set the vision of the company yourself. He goes on to talk about the challenges that come with being a first-time CEO and managing a variety of functions where you are not necessarily the expert, to fund-raising in the current biotech environment and shows appreciation for the support of the experienced Board of Directors. 

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Plugging the gap and delivering on the promise of gene therapy in rare diseases which conceptually should be fast. Trying to leverage what has already worked in their field -AAV gene therapy. Adrien then goes on to explore the science behind the decision, including overcoming the safety and CMC challenges through the use of “de-risked” capsids with innovation coming from other AAV design elements. 

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On 6th September 2023, Bloomsbury announced that they were expanding one of their programmes called BGT- DTDS which is primarily developed for a very rare childhood disorder called Dopamine Transporter Deficiency Syndrome (DTDS), into Parkinson’s Disease. DTDS is a very severe childhood disease where children are born without the ability to express the dopamine transporter protein in the brain which causes a variety of complications. The DTDS program uses an AAV2 vector and is injected directly into the brain of patients. That vector carries a functional copy of the dopamine transporter gene to restore the expression of that protein in the brains of patients. 

Adrien goes on to talk about how they realised that there is another disease where patients progressively lose the ability to make that transporter – Parkinson’s Disease. Their approach is to use the same vector as the one developed for DTDS with the view to rebalance the expression of the transporter and re-establish dopamine homeostasis in the brain of patients with Parkinson’s Disease. Bloomsbury has just completed an experiment in a rodent model of Parkinson’s Disease using that vector and they are very excited to report a profound effect on levodopa-induced dyskinesia and they have seen some effects on anxiety behaviours. It is very exciting as they may have found a very novel approach to treat Parkinson’s Disease, tackling both motor function and non-motor function symptoms. 

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More of the same! More very efficient and productive operations. First and foremost – fundraising! Adrien is actively engaged in fundraising activities now in order to deliver an exciting set of key milestones in the next 12 months. For their lead programme for a liver disorder – OTC Deficiency – they expect to be starting a first-in-human phase 1/2 clinical trial in a matter of weeks in the UK. 

For their second programme, DTDS, they got the go-ahead from the MHRA to move straight from mice efficacy studies to clinical trial, and they intend on running a single trial, a phase 1/2/3 trial, in a very compact design, following their vision of bringing treatments to patients quickly. 

Then the sister program for Parkinson’s Disease expects more preclinical data, and they want to test the vector in different rodent models to further elucidate its potential benefits. 

For the last two programmes (for the diseases NPC and INAD), they have just scored great success with the MHRA where they got support for short safety and biodistribution studies in rodents only, and they are going to meet and engage with the FDA in October for both programmes. 

A very rich calendar of events, all driven by their vision to deliver innovative gene therapies to patients, fast. 

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Thank you to Adrien for taking the time to feature and I wish you and all those at Bloomsbury success moving forward. 

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For more information on Bloomsbury Genetic Therapies, please visit https://bloomsburygtx.com/ 

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For any questions or if you are interested in being featured on a future episode, please feel free to reach me at l.hulley@sciproglobal.com