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LoQus23 Therapeutics announces £35 million financing to advance a new drug aimed at inhibiting somatic expansion in Huntington’s Disease

Cambridge, UK, 2 October 2024 – LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s Disease, myotonic dystrophy type 1 and similar triplet repeat expansion diseases, today announces the successful close of its £35 million (c.$43 million) Series Read more…

By Daniela Carvalho, ago

FDA Grants Fast Track Designation to PTC518 Huntington’s Disease Program

PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression.   Programs with Fast Track designation can benefit from early interactions with the FDA, and may be eligible for priority review and accelerated approval. This follows the release of 12 month interim data from PTC’s Phase 2 study, which Read more…

By Daniela Carvalho, ago

Prilenia’s Pridopidine for Huntington’s Disease Accepted for European Marketing Authorisation Review

Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for pridopidine as a treatment for Huntington’s disease (HD). An MAA Read more…

By Daniela Carvalho, ago

IROS, Prilenia and International Huntington’s Disease Association collaborate on first-ever Huntington’s disease (HD) trial in the MENA region

Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial for Huntington’s disease (HD) in the UAE and MENA region, Read more…

By Daniela Carvalho, ago

uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for HD

“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe this is the first clinical trial of any investigational medicine Read more…

By Daniela Carvalho, ago

Wave Life Sciences Announces Positive Results from Phase 1b/2a SELECT-HD Trial with First Clinical Demonstration of Allele-Selective Mutant Huntingtin Lowering in Huntington’s Disease

Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT) protein and preserves healthy, wild-type huntingtin (wtHTT) protein for individuals with Huntington’s disease. Trial ResultsIn the analysis, it was compared Read more…

By Daniela Carvalho, ago

Sage Therapeutics Phase 2 Study Reinforces Cognitive Impact of Huntington’s Disease

Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease.  Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference between 29 healthy volunteers and 40 participants with HD.   As a secondary outcome the study measured the safety and tolerability Read more…

By Daniela Carvalho, ago
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