Michael Pirovolakis was diagnosed with spastic paraplegia type 50 (SPG50) when he was 18 months old. SPG50 is an “ultra-rare” progressive neurodegenerative disorder that causes developmental delays, speech impairment, seizures, a progressive paralysis of all four...
We recently came across a comprehensive review in Nature Review Drug Discovery by Ling, Q. et al., which provides in-depth insights into AAV-based gene therapy, particularly for neurological disorders. In this article, we’d like to share a concise summary of the...
Gene therapy has been a focal point in the biopharmaceutical industry over the past five years. Initially considered for the treatment of rare genetic diseases, gene therapy has gradually been applied to neurological diseases such as Alzheimer’s and...
NeuExcell Therapeutics, a leading biotechnology company focused on in vivo neural regenerative therapies, announces the successful dosing of the first patient with the first-in-class NeuroD1 gene therapy product NXL-004. This milestone represents significant progress...
Not all drug targets are created equal. For those that result in a tissue specific gain-of-function, the strategy can be not-so-straightforward. That’s exactly what’s going on in some forms of amyotrophic lateral sclerosis (ALS), such as those with a mutation in the...
