Category: Research

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 […]

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 […]

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,” […]

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) […]

PTC today announced promising 12 month results from their Phase 2 PIVOT-HD study of oral 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 […]

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 […]

FDA grants uniQure Regenerative Medicine Advance Therapy (RMAT) Designation

Today we are very happy in the Huntington community.  The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 […]

SOM Biotech completes the recruitment process in the Phase IIb study of the treatment of chorea in Huntington’s disease

SOM Biotech, a clinical-stage drug discovery and development company based on a unique proprietary Artificial Intelligence platform (SOMAIPRO®), is pleased to announce that the recruitment of the Phase IIb clinical […]

Prilenia Plans to Submit Marketing Authorization Application in the EU for Pridopidine in Huntington’s Disease

Prilenia announced today that they plan to submit a Marketing Authorization Application in the EU for the use of Pridopidine in the treatment of Huntington’s Disease. According to Dr. Michael […]

Breakthrough represents a new hope for HD patients

Scientists from the Weizmann Institute in Israel have published the results of an animal study which holds tremendous promise for the treatment of Huntington’s Disease.  They have discovered that two […]