drug-development
-
2025 HDBuzz Prize: When Good Huntingtin Goes Missing: A Step Toward Designing Safe HD Treatments
Weāre proud to announce Gravity Guignard as a 2025 HDBuzz Prize winner! A new study points to one of the reasons why past HD trials might have failed. The good news? This shows the path to safer drugs, getting us closer to effective therapies.
-
Pridopidine Hits a Roadblock: EMA Says No to Approval for Huntingtonās Disease Treatment
The EMA has rejected pridopidine for HD treatment in Europe. While disappointing, this outcome aligns with the trial data. Prilenia plans further studies, and despite setbacks, HD research in 2025 continues to bring momentum and hope.
-
Full Steam Ahead: uniQureās On Track With Hope for Accelerated Approval of Huntingtonās Disease Drug
uniQure has aligned with the US FDA on next steps to achieve accelerated approval of AMT-130. If data remain positive, they will be on track to market the first HD gene therapy.
-
Stopping C-A-G Repeat Expansion In Its Tracks
A new study shows that lowering MSH3, a key DNA repair protein, with antisense oligonucleotide (ASO) therapy can stop CAG repeat expansions in HD patient-derived brain cells. This could be a promising way to slow symptom onset and progression.
-
Moving into the Fast Lane: uniQure and the FDA Are on the Same Track for Accelerated Approval
In an update shared today, uniQure announced alignment with the US drug regulator on key criteria for accelerated approval of drugs for Huntingtonās disease.
-
Bringing HD Treatments to Market: The Role of Regulatory Oversight
Moving drugs from the lab bench to pharmacy shelves is no small task. In this article we delve into the role regulatory oversight plays in clinical trials and the approval of medicines for Huntingtonās disease.
-
Highlighting a link between brain disorders on Ataxia Awareness Day
September 25th is Ataxia Awareness Day, so HDBuzz is highlighting the connections between HD and Ataxia research. Learn about how these fields have historically grown in parallel and informed one another.
By Dr Leora Fox -
Hope vs. hype: seeking truth in recent Prilenia headlines
Although pridopidine has suffered four negative trials for HD, the message from Prilenia continues to be positive. What is hope and what is hype in this sixteen-year quest for regulatory approval?
-
Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
Thereās more good news in the forecast in the Huntingtonās disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
-
No pivot needed for PTC-518
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.