Huntington's Disease: the receipts

uniQure will seek approval for Huntington's disease gene therapy after switchup...

drugdiscoverynews.com · Jun 19, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ⚡ Fda Approval

uniQure Plans BLA Submission for AMT-130 Gene Therapy in Huntington’s Disease

clinicaltrialvanguard.com · Jun 18, 2026 Standard

• Amt 130 Gene Therapy ✚ Fda Approval
• Amt 130 Gene Therapy ↓ Mutant Huntingtin Protein

FDA opens path for uniQure’s Huntington’s gene therapy filing - European Biotechnology Magazine

european-biotechnology.com · Jun 18, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ✚ Htt Lowering Therapy

UniQure to seek approval of Huntington’s gene therapy after FDA U-turn - Pharmaceutical Technology

pharmaceutical-technology.com · Jun 18, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ⚡ Fda Approval

UniQure Shares Soar After FDA Reversal on Its Huntington's Therapy

Bloomberg · Jun 17, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ✚ Uniqure

UniQure to seek approval for Huntington's therapy after FDA U-turn, shares skyro...

Reuters · Jun 17, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ✚ Uniqure

UniQure plans Q3 submission for Huntington’s gene therapy after FDA reverses course—again

biospace.com · Jun 17, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ✚ Uniqure

UniQure to seek FDA approval for Huntington's disease gene therapy after previous clash with agency

cnbc.com · Jun 17, 2026 Standard

• Gene Therapy ✚ Fda Approval
• Uniqure ⚡ Fda Approval

The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130 – HDBuzz

en.hdbuzz.net · Jun 17, 2026 Standard

• Amt 130 Gene Therapy ✚ Fda Approval

UniQure stock surges after FDA U-turn clears path to gene therapy filing

fiercebiotech.com · Jun 17, 2026 Standard

• Fda Approval ✚ Uniqure
• Gene Therapy ✚ Fda Approval
• Gene Therapy ✚ Uniqure

Regulatory U-turn opens door for uniQure to refile BLA for snubbed gene therapy

firstwordpharma.com · Jun 17, 2026 Standard

• Fda Regulatory U Turn ✚ Uniqure
• Fda Regulatory U Turn ✚ Gene Therapy

uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease

globenewswire.com · Jun 17, 2026 Standard

• Uniqure ✚ Fda Approval

Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval

statnews.com · Jun 17, 2026 Standard

• Fda Dispute Resolution ✚ Fda Approval
• Fda Dispute Resolution ✚ Uniqure

Toxic Clumps in Huntington's Disease May Protect the Brain Too

singularityhub.com · Jun 5, 2026 Standard

• Mutant Huntingtin Protein ⚡ Neurodegeneration
• Mutant Huntingtin Protein Aggregates ✕ Neurodegeneration

Skyhawk reports encouraging 12-month results for oral Huntington’s disease drug SKY-0515 – HDBuzz

en.hdbuzz.net · Jun 1, 2026 Standard

• Sky 0515 ↓ Mutant Huntingtin Protein

Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam – HDBuzz

en.hdbuzz.net · May 4, 2026 Standard

• Votoplam ↓ Mutant Huntingtin Protein

Proteins in tiny cell tunnels may be Huntington’s treatment target

huntingtonsdiseasenews.com · Mar 24, 2026 Standard

• Proteins In Tunneling Nanotubes ✚ Neurodegeneration

FAU Discovery of Tiny Cell ‘Tunnels

fau.edu · Mar 20, 2026 Standard

• Tiny Cell Tunnels ✚ Mutant Huntingtin Protein Spread
• Tiny Cell Tunnels ✚ Neurodegeneration

Unraveling the Connections between TDP-43 and Huntingtin

jneurosci.org · Mar 11, 2026 Standard

• Mutant Huntingtin Protein ⚡ Tdp 43 Pathology

Putting it in print: oral drug branaplam lowered huntingtin, but safety concerns halted development – HDBuzz

en.hdbuzz.net · Feb 12, 2026 Standard

• Branaplam ↓ Mutant Huntingtin Protein
• Branaplam Safety Concerns ✕ Branaplam

A hitch in the stitch reveals why DNA sequence patterns matter in Huntington’s disease   – HDBuzz

en.hdbuzz.net · Feb 9, 2026 Standard

• Htt Gene Mutation ⚡ Neurodegeneration

Scientists Are Finally Closing In on a Treatment For Huntington's Disease

sciencealert.com · Jan 15, 2026 Standard

• Htt Gene Mutation ✚ Mutant Huntingtin Protein

Sequence Variants in Small CAG Repeat Expansions of the HTT Gene and Disease Onset and Progression in Huntington Disease | Neurology

neurology.org · Dec 19, 2025 Standard

• Htt Gene Mutation ⚡ Neurodegeneration

ASOs Restore Cognitive Deficits in Huntington Mice | Neurology Today

neurologytoday.aan.com · Oct 25, 2025 Standard

• Antisense Oligonucleotide ↓ Mutant Huntingtin Protein
• Antisense Oligonucleotide ↓ Neurodegeneration

Development of cognitive, motor, metabolic, and mutant huntingtin aggregation in the zQ175 mouse model of Huntington’s disease - Scientific Reports

Nature · Oct 3, 2025 Standard

• Mutant Huntingtin Protein ⚡ Neurodegeneration
• Htt Gene Mutation ✚ Mutant Huntingtin Protein

Huntingtin protein arranges cytoskeletal filaments, offering insight into neurodegeneration

medicalxpress.com · Oct 1, 2025 Standard

• Htt Gene Mutation ⚡ Neurodegeneration

Huntington’s disease treated for first time using gene therapy

Nature · Sep 25, 2025 Standard

• Gene Therapy ↓ Mutant Huntingtin Protein

The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial – HDBuzz

en.hdbuzz.net · Sep 24, 2025 Standard

• Gene Therapy ↓ Neurodegeneration
• Gene Therapy ✚ Uniqure

In a first, a gene therapy seems to slow Huntington disease

science.org · Sep 24, 2025 Standard

• Gene Therapy ↓ Neurodegeneration

SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update – HDBuzz

en.hdbuzz.net · Sep 18, 2025 Standard

• Antisense Oligonucleotide ↓ Mutant Huntingtin Protein

SKY-0515 Demonstrates Dose-Dependent Reductions in Mutant Huntingtin Protein in Phase 1 Update | NeurologyLive - Clinical Neurology News and Neurology Expert Insights

neurologylive.com · Sep 18, 2025 Standard

• Sky 0515 ↓ Mutant Huntingtin Protein
• Sky 0515 Dose Level ↓ Mutant Huntingtin Protein

Huntington’s: Huntingtin protein function loss plays key role

huntingtonsdiseasenews.com · Jul 22, 2025 Standard

• Htt Gene Mutation ✚ Mutant Huntingtin Protein

Update on the Phase II GENERATION HD2 study – International Huntington Association

huntington-disease.org · Apr 17, 2025 Standard

• Clinical Trial Readout ✚ Antisense Oligonucleotide

Long somatic DNA-repeat expansion drives neurodegeneration in Huntington’s disease

cell.com · Dec 1, 2024 High trust

• Somatic Cag Expansion ↑ Neurodegeneration

MSH3, part of the MutSβ heterodimer that is thought to recognize the potential s...

Oxford Academic · Jan 1, 2024 High trust

• Msh3 ↑ Somatic Cag Expansion
• Fan1 ✕ Somatic Cag Expansion

Elevated plasma and CSF neurofilament light chain concentrations are stabilized in response to mutant huntingtin lowering in the brains of Huntington’s disease mice - PMC

ncbi.nlm.nih.gov · Jan 1, 2024 High trust

• Htt Lowering Therapy ↓ Neurofilament Light

Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease - PMC

ncbi.nlm.nih.gov · Nov 1, 2023 High trust

• Msh3 Lowering Therapy ↓ Somatic Cag Expansion

Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases

Brain Sciences · Feb 22, 2020 High trust

• Gene Therapy ⚡ Neuroinflammation
• Neuroinflammation ↓ Transgene Expression
• Cns Targeted Aav Delivery ⚡ Neuroinflammation
• Cns Targeted Aav Delivery ↑ Transgene Expression
• Systemic Aav Dose ↑ Neuroinflammation

Targeting Huntingtin Expression in Patients with Huntington’s Disease

New England Journal of Medicine · May 7, 2019 High trust

• Htt Gene Mutation ✚ Mutant Huntingtin Protein
• Antisense Oligonucleotide ↓ Mutant Huntingtin Protein

Targeting Huntingtin Expression in Patients with Huntington's Disease

openalex.org · Jan 1, 2019 High trust

• Htt Gene Mutation ⚡ Mutant Huntingtin Protein
• Antisense Oligonucleotide ↓ Mutant Huntingtin Protein

Huntington's disease: a clinical review

European Journal of Neurology · Aug 17, 2017 High trust

• Htt Gene Mutation ✚ Mutant Huntingtin Protein
• Mutant Huntingtin Protein ⚡ Neurodegeneration
• Htt Lowering Therapy ↓ Mutant Huntingtin Protein
• Antisense Oligonucleotide ↓ Mutant Huntingtin Protein

Neurofilament light protein in CSF and blood is associated with neurodegeneration and disease severity in Huntington’s disease R6/2 mice - PMC

ncbi.nlm.nih.gov · Jan 1, 2017 High trust

• Neurodegeneration ↑ Neurofilament Light

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9

Human Molecular Genetics · Sep 15, 2016 High trust

• Htt Gene Mutation ✚ Mutant Huntingtin Protein
• Mutant Huntingtin Protein ⚡ Neurodegeneration
• Allele Specific Crispr Cas9 ✕ Mutant Huntingtin Protein
• Allele Specific Crispr Cas9 ✚ Htt Lowering Therapy

Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression - Scientific Reports

Scientific Reports · Jul 15, 2015 High trust

• Htt Gene Mutation ↑ Mutant Huntingtin Protein
• Neurodegeneration ↔ Mutant Huntingtin Protein
• Htt Lowering Therapy ↓ Mutant Huntingtin Protein

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

Advanced Drug Delivery Reviews · Feb 7, 2015 High trust

• Phosphorothioate Backbone Modification ↑ Plasma Protein Binding
• Plasma Protein Binding ↑ Tissue Bioavailability
• Sugar 2 Prime Position Modification ↑ Antisense Oligonucleotide Potency
• Blood Brain Barrier ✕ Antisense Oligonucleotide Cns Distribution
• Intrathecal Administration ✚ Antisense Oligonucleotide Cns Distribution

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

Molecular Therapy · Oct 4, 2011 High trust

• Htt Gene Mutation ⚡ Mutant Huntingtin Protein
• Mutant Huntingtin Protein ⚡ Neurodegeneration
• Antisense Oligonucleotide ↓ Mutant Huntingtin Protein
• Wild Type Huntingtin Protein ✚ Neurodegeneration
• Cet Modification ↑ Antisense Oligonucleotide

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

Molecular Therapy · Feb 24, 2009 High trust

• Htt Gene Mutation ⚡ Neurodegeneration
• Htt Lowering Therapy ↓ Neurodegeneration
• Htt Lowering Therapy ↑ Motor Coordination
• Htt Lowering Therapy ↑ Survival

Caspase Cleavage of Mutant Huntingtin Precedes Neurodegeneration in Huntington's Disease

Journal of Neuroscience · Sep 15, 2002 High trust

• Htt Gene Mutation ✚ Mutant Huntingtin Protein
• Caspase Cleavage Of Htt ↑ Mutant Huntingtin Protein Aggregates
• Caspase Cleavage Of Htt ⚡ Neurodegeneration
• Mutant Huntingtin Protein Aggregates ⚡ Corticostriatal Dysfunction

Formation of Neuronal Intranuclear Inclusions Underlies the Neurological Dysfunction in Mice Transgenic for the HD Mutation

Cell · Aug 1, 1997 High trust

• Htt Gene Mutation ⚡ Neuronal Intranuclear Inclusions
• Neuronal Intranuclear Inclusions ⚡ Neurodegeneration
• Mutant Huntingtin Protein Aggregates ✚ Neuronal Intranuclear Inclusions