Kategorie: Forschung

UniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application […]

Update on the Phase II GENERATION HD2 study

Roche has announced the continuation of the GENERATION HD2 Phase II clinical trial evaluating tominersen in individuals with early signs of Huntington’s disease. Following an interim analysis by an independent committee, no […]

SOM Biotech gibt Ergebnisse der Phase-2b-Studie bekannt

SOM Biotech presents the Phase 2b study results with SOM3355 demonstrating a unique profile with robust improvements of chorea in Huntington’s Disease Patients and a safe profile with no somnolence, […]

PTC Therapeutics schließt mit Novartis eine globale Lizenz- und Kooperationsvereinbarung für das PTC518-Programm zur Behandlung der Huntington-Krankheit ab

December 2, 2024 – PTC to receive $1.0B in cash at closing –– PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones –– PTC to share profits in the U.S. and […]

Sage Therapeutics stellt Entwicklung von Dalzanemdor bei HD ein

The Phase 2 DIMENSION Study did not meet its primary endpoint Dalzanemdor was generally well-tolerated; no new safety signals were observed Based on these data, the Company does not plan […]

LoQus23 Therapeutics gibt eine Finanzierung in Höhe von 35 Millionen Pfund bekannt, um ein neues Medikament zur Hemmung der somatischen Expansion bei der Huntington-Krankheit voranzutreiben

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 […]

Pridopidin von Prilenia zur Behandlung der Huntington-Krankheit zur Überprüfung der europäischen Marktzulassung angenommen

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 […]

uniQure gibt positive Zwischenergebnisse bekannt, die eine Verlangsamung des Krankheitsverlaufs in den Phase I/II-Studien mit AMT-130 zur Behandlung von Huntington zeigen

“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,” […]

Wave Life Sciences gibt positive Ergebnisse der Phase 1b/2a SELECT-HD-Studie mit dem ersten klinischen Nachweis einer allelselektiven Senkung der Huntingtin-Mutation bei der Huntington-Krankheit bekannt

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) […]

PTC gab heute vielversprechende 12-Monats-Ergebnisse der Phase-2-Studie PIVOT-HD mit dem oral verabreichten PTC518  

At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where […]