Category: Research

uniQure Provides Regulatory Update

Dear Huntington’s Disease Community, We are writing to share an update following our recent Type A meeting with the U.S. Food and Drug Administration (FDA.) On March 2, 2026, uniQure […]

Harness Therapeutics Nominates HRN001 Drug Candidate for HD and Launches Clinical Advisory Board

Harness Therapeutics Nominates HRN001, a First-in-Class Drug Candidate for Huntington’s Disease and Establishes Clinical Advisory Board Cambridge, UK, 16 February 2026: Harness Therapeutics (‘Harness’), a biotechnology company unlocking previously undruggable […]

Novartis Announces INVEST-HD

Novartis development of Votoplam in HD: INVEST-HD Phase 3 trial information now available February 3rd 2026 Dear Huntington’s disease Community, We are delighted to share an update on the development […]

Sarepta Therapeutics Announces Approval of Clinical Trial Application for SRP-1005, Its Investigational Treatment for Huntington’s Disease

The first-in-human clinical study of SRP-1005, known as INSIGHTT, is expected to begin in the second quarter of 2026 CAMBRIDGE, Mass.–(BUSINESS WIRE)–Feb. 4, 2026– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader […]

An Update on SKY-0515

Dear friends in the Huntington’s disease community, We’d like to share an update on ongoing research into SKY-0515, an investigational medicine being studied for Huntington’s disease (HD). A scientific press […]

uniQure Announces Meeting with FDA

January 9, 2026 7:05 AM EST uniQure Announces Type A Meeting Scheduled with FDA LEXINGTON, Mass. and AMSTERDAM, Jan. 09, 2026 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading […]

An Update from LoQus23

LoQus23 Therapeutics is a biotechnology company investigating small molecule drugs that could stop the pathogenic triplet expansion that is the cause and driver of Huntington’s Disease (HD), myotonic dystrophy type […]

New Phase 1 POINT-HD Study

New Phase 1 POINT-HD study starts for selective huntingtin-lowering approach Dear Huntington’s community leaders, Following your request to receive updates about our research efforts, we are pleased to share that […]

Summary of “EHDN Platform Meeting on uniQure topline results: Unpacking the outcomes so far”

Warsaw, November 19th, 2025. Dear members, As you might know, yesterday the European Huntington’s Disease Network (EHDN) developed a seminar about the topline results of uniQure, a pharmaceutical company that […]

uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease

November 3, 2025 Dear Huntington’s Disease Community, We issued a press release today announcing that uniQure received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics […]