香港六合彩中特网

Dr Pamela Tranter, 香港六合彩中特网 Translational Research Office

The first speaker was Dr Pamela Tranter, Director of the Translational Research Group (TRG), part of the TRO (Translational Research Office, Maple House). Dr Tranter has over 20 years of experience in various sectors of drug development in big pharma and over 10 years of experience as a project manager at 香港六合彩中特网 in several therapeutic modalities. She is now director of the TRG, a professional service available for everyone at 香港六合彩中特网 that helps scientists organising a bench-to-bed scientific strategy and supporting grant applications. Dr Tranter mentioned that the grant writing success rate with the TRO involvement is close to 40% vs a national average that goes as low as 6% up to 25%, depending on the scheme. Additionally, to help researchers with any matter related to industrial collaborations, there are dedicated and experienced business and innovation managers part of the Business and Innovation Group (BIG) within the TRO.

Dr Barney Bryson, Department of Neuromuscular Diseases,聽香港六合彩中特网 Queen Square Institute of Neurology

We then listened to Dr Barney Bryson, from the Department of Neuromuscular Diseases (Queen Square House), talking about the journey to set up 鈥楢 Novel Optogenetic Cell Therapy for Amyotrophic Lateral Sclerosis鈥�. Dr Bryson is an expert in the field of neuro-regeneration, focusing on the development of for traumatic injuries and neurodegenerative conditions for motor neurons. In this talk, he highlighted his work on combining allogeneic human stem cell derived motoneurons with optogenetics in a mouse model for ALS. Dr Bryson explained that one of the advantages of using optogenetic training compared to standard electrical stimulation is the possibility of聽stimulating聽the muscles in a predetermined order and in a physiological manner, overcoming the problem of rapid muscle fatigue. This neuroreplacement therapy could potentially pave the way for a customised brain-computer interface technology for voluntary control of muscle function. Part of Dr Bryson's funding for the proof of concept work was achieved by an MND/LifeArc grant awarded with the support of the TRO.

Dr Paolo Spingardi, Business Manager at the 香港六合彩中特网 Tech Transfer Office

The fourth speaker was Dr Paolo Spingardi, Business Manager at the 香港六合彩中特网 Tech Transfer Office. 香港六合彩中特网B is 香港六合彩中特网鈥檚 commercial arm, and 香港六合彩中特网B's main aim is to evaluate researchers鈥� ideas and work for maximising their real-world impact. Importantly, an idea alone is not enough to create positive change for patients: you need to put in the work. But what is 鈥渢he work鈥�? (i.e. what is intellectual property?). IP can be many things, from a patent to data coming from a database or a clinical trial, from a copyright for protecting a software to know-how. Dr Spingardi explained that to be able to progress a technology/therapy, this needs to be novel, commercially exploitable, and more advantageous compared to what already exists on the market. The discovery needs to be legally protected and requires an initial proof of concept to confirm its validity. 香港六合彩中特网B has been so far very successful both in patent filing, licensing and spin out creation, with 5 spin outs created in the last 4 years and listed on Nasdaq.

Professor Sarah Tabrizi, Director of 香港六合彩中特网 Huntington Disease Centre and Joint Head of the Department of Neurodegenerative Disease,聽香港六合彩中特网 Queen Square Institute of Neurology

The next speaker was Professor Sarah Tabrizi, Director of 香港六合彩中特网 Huntington Disease Centre and Joint Head of the Department of Neurodegenerative Disease聽at IoN. Prof Tabrizi gave a remarkable overview on the challenges of a recent genetic therapy phase I/II and III developed for Huntington Disease (HD) in combination with different pharmaceutical companies (Ionis and then Roche). The first study regarded an ASO (anti-sense oligonucleotide) targeting mutant HTT (huntingtin protein) in the brain of adult patients injected via an intrathecal bolus. The was a success, collecting safety data and demonstrating a dose-dependent reduction in the concentration of mutant huntingtin in the CSF. Contrastingly, a tough lesson arrived when the trial went straight into phase III, lacking a traditional phase II for dose finding, using the maximum tolerated dose for maximising striatal penetration. After a few weeks of treatment, the pharma sponsoring the study decided to stop the recruitment due to clear signs of toxicity and lack of efficacy of the treatment. Despite this result, there were many opportunities to advance the knowledge of ASOs therapeutics in patients and on HD biomarkers. For example, CSF-nfl (neurofilamin) protein emerged clearly as a reliable indicator of safety, now used worldwide. Prof Tabrizi finished her talk examining the opportunities arising from continuous ground-breaking work on DNA repair strategies for HD and new horizons for .

Professor Rob Brownstone, Brain Research UK Chair of Neurosurgery

Professor of Neurosurgery Rob Brownstone then talked about the steps to set up his own company, Sania Therapeutics, to tackle neuromodulation for spasticity using gene therapy to improve patients鈥� lives. Neuromodulation does not mean treating the disease, Prof Brownstone explained, but it means treating symptoms by altering nerve (neuronal) circuit function and activity through targeted delivery of a stimulus. This stimulus can be of electrical or chemical nature, to specific sites in the body/brain. Prof Brownstone鈥檚 approach to the problem was different such that his collaborators and him spun-out a company very early on. They thought that maintaining the work nicely split out between academic lab vs independent spin-out labs would be the best way to organise and speed up things. Were they right? Is this out-of-the-shelf solution going to revolutionise the translational path as we know it?聽

Dr聽Cath Mummery, Consultant Neurologist at the National Hospital for Neurology and Neurosurgery (香港六合彩中特网H) and Honorary Associate Professor at 香港六合彩中特网 Queen Square Institute of Neurology

The eighth speaker of the afternoon was Dr聽Cath Mummery, Consultant Neurologist at the National Hospital and Honorary Associate Professor at 香港六合彩中特网 Queen Square Institute of Neurology. In her presentation, she talked about the history聽of dementia trials and how these changed through the years. Prof Mummery explained how genetic therapies for dementia started in the mid-80s with a letter from to Prof John Hardy and Prof Martin Rossor (香港六合彩中特网 Queen Square Institute of Neurology), asking to understand her family鈥檚 uncommon form of dementia. After testing their blood, the APP gene was first found. This discovery, together with others, paved the way for the amyloid cascade hypothesis which set off the early-on treatment trajectories for Alzheimer鈥檚 disease (AD). Historically, were the first therapy to be applied; for a long time, no positive results were shown, until June 2021 with the accelerated approval of Aducanumab that demonstrated a reduction of amyloids in the patients鈥� brain. The greatest result, linking the reduction in amyloid with effective cognitive protection, came just a few months ago in September 2022, with the success of an Eisai AD drug, Lecanemab, showing protection in loss of cognition compared to placebo. This protection was not linear with the amount of amyloid disruption though, highlighting that there is still a lot to do to fully understand this disease. 聽More recent alternative approaches are silencing RNAs, activation of TREM2 gene, peripheral checkpoint inhibitors, Tau clearing by using antibodies, antisense oligonucleotides (ASOs) and more. Dr聽Mummery remarked how philanthropic funding and industry collaborations such as Eisai and IONIS made it possible to run this trial in the UK and specifically in 香港六合彩中特网.聽