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Bayer gets go-ahead for Huntington’s Disease gene therapy trial  

Drug Discovery World

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . In 2019, BV-101 was granted orphan drug designation in the European Union. . There are currently no approved disease modifying therapies for Huntington’s Disease.

Therapies 246
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Gene therapy restores vision in first-of-its-kind trial

Drug Discovery World

A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The gene therapy itself caused mild inflammation that was treated with steroids. Side effects were largely limited to minor surgical complications.

Therapies 173
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Dr Gonzalez-Aseguinolaza recognised for gene therapy research

Drug Discovery World

Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. She then moved into rare diseases and has been working on rare hepatic diseases for the last two decades, with a focus on the development of advanced therapies.

Therapies 130
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How to advance AAV-based gene therapies

Drug Discovery World

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

Therapies 162
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Concussion drug therapies on the horizon

Drug Discovery World

Over the past 20 years research has increasingly linked concussions to neurogenerative conditions, including dementia, Alzheimer’s disease and CTE, a degenerative brain disease. More than 413,000 service members were diagnosed with a TBI between 2000 and Q3 of 2019. Over 80% of these were concussions.

Therapies 147
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Diabetic eye disease: The urgent need for early, non-invasive treatments

Drug Discovery World

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Specific numerical values (steps) denote the severity of NPDR, with 47-53 describing the moderately severe disease.

Disease 162
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Could NfL accelerate drug development for neurodegenerative diseases?

Drug Discovery World

Professor Andy Whiting and Dr Tony Lockett discuss how specific levels of a protein could be used to advance drug development in neurodegenerative diseases. These parameters aim to measure the progression of disease over time by looking at the functional or observable change in a patient.