Huntington's Disease pipeline constitutes 45+ key companies continuously working towards developing 50+ Huntington's Disease treatment therapies, analyzes DelveInsight
LAS VEGAS, Oct. 24, 2022 /PRNewswire/ -- DelveInsight's 'Huntington's Disease Pipeline Insight – 2022' report provides comprehensive global coverage of available, marketed, and pipeline Huntington's disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Huntington's disease pipeline domain.
Key Takeaways from the Huntington's Disease Pipeline Report
- DelveInsight's Huntington's disease pipeline report depicts a robust space with 45+ active players working to develop 50 + pipeline therapies for Huntington's disease treatment.
- Key Huntington's disease companies such as Prilenia Therapeutics, Ionis Pharmaceuticals, Annexon, Vaccinex, Neurocrine Biosciences, SAGE Therapeutics, PTC Therapeutics, WaVe life Sciences, Novartis, Neurimmune Therapeutics, SOLA Biosciences, UniQure Biopharma, Roche, Annexon, Inc., Azevan Pharmaceuticals, SOM Innovation Biotech SA, Asklepios BioPharmaceutical, Luye Pharma Group Ltd., BrainStorm Cell Therapeutics, Voyager Therapeutics, Passage Bio, Locana Bio, Ceptur Therapeutics, Enzerna Biosciences, AFFiRiS AG, Arvinas, Atalanta Therapeutics, Anima Biotech, and others are evaluating novel Huntington's disease treatment drugs candidate to improve the treatment landscape.
- Promising Huntington's disease pipeline therapies in various stages of development include Pridopidine, Tominersen, ANX-005, Pepinemab, Valbenazine, SAGE-718, PTC-518, WVE-003, AMT 130, Branaplam, NI-302, SOL-176, RO7234292, ANX005, SRX246, Bevantolol, SOM3355, BV-101 Gene Therapy, LPM3770164, Passage Bio Research Project, Huntington disease Research Project, BrainStorm Research Project, Locana Huntington disease Research Project, Ceptur Huntington Research Project, HTT RNA Research project, mHTT Research Project, AFFiRiS Huntington Research Project, HTT Research Project, Anima Biotech mHTT Research Project, and others.
- In August 2022, Asklepios BioPharmaceutical, a wholly owned and independently operated subsidiary of Bayer AG, received clearance to conduct a Phase I/II trial for its novel Huntington's Disease (HD) gene therapy, BV-101, in France through its subsidiary BrainVectis. This authorization, provided by the National Agency for Safety of Medicines and Health Products (ANSM), the country's governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge, enables the company to begin recruiting participants
- In March 2022, PTC Therapeutics announced the initiation of the PIVOT-HD Phase II clinical trial evaluating PTC518 in people with Huntington's disease (HD). PIVOT-HD is a global trial starting in the United States. PTC518 is an oral, small molecule splicing modifier that was specifically designed to lower huntingtin mRNA and protein selectively. There are no current treatments for the underlying cause of HD
- In January 2022, Ionis Pharmaceuticals announced that its partner Roche is designing a new Phase II trial to evaluate tominersen in Huntington's disease (HD). After halting dosing in the Phase III GENERATION HD1 study, exploratory posthoc analyses suggest tominersen may benefit younger adult patients with lower disease burden. These results require confirmation in a randomized, placebo-controlled study
- In September 2021, Sage Therapeutics announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation to SAGE-718 for development as a potential treatment for Huntington's disease (HD).
- In September 2021, Voyager Therapeutics introduced new programs in Huntington's disease, a monogenic form of ALS (SOD1), spinal muscular atrophy, and diseases linked to GBA1 mutations, all powered by its proprietary AAV capsids that have demonstrated superior transgene expression in the brain compared to AAV9 delivery in non-human primates.
Request a sample and discover the recent advances in Huntington's disease treatment @Huntington's Disease Pipeline Report
The Huntington's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Huntington's disease products, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Huntington's disease pipeline landscape.
Huntington's Disease Overview
Huntington's disease is a rare, inherited disease that causes the progressive breakdown (degeneration) of brain nerve cells. It's an autosomal dominant condition caused by a single faulty gene on chromosome 4. The common symptoms of Huntington's disease include involuntary jerking or writhing movements (chorea), muscle problems such as rigidity or muscle contracture (dystonia), slow or abnormal eye movements, impaired balance, difficulty speaking or swallowing, difficulty organizing, prioritizing, or focusing on tasks, and fatigue and loss of energy. A general physical examination, medical history, and thorough neurological and psychiatric examinations are used for Huntington's disease diagnosis.
Huntington's disease treatment focuses on symptom management, which includes medications and psychotherapy. Tetrabenazine (Xenazine) and deutetrabenazine (Austedo) have been specifically approved by the FDA to suppress Huntington's disease-related involuntary jerking and writhing movements (chorea).
Find out more about Huntington's disease treatment @Drug Treatment for Huntington's Disease
A snapshot of the Huntington's Disease Pipeline Drugs mentioned in the report:
Drugs |
Company |
Phase |
MoA |
RoA |
Pridopidine |
Prilenia Therapeutics |
Phase III |
Dopamine D2 receptor antagonists; Glutamate modulators; Sigma-1 receptor agonists |
Oral |
RO7234292 |
Roche |
Phase III |
RNA interference |
Intrathecal |
Tominersen |
Ionis Pharmaceuticals |
Phase III |
HD protein inhibitors; RNA interference |
Intrathecal |
Valbenazine |
Neurocrine Biosciences |
Phase III |
Vesicular monoamine transporter 2 inhibitors |
Oral |
ANX-005 |
Annexon |
Phase II |
Complement C1 inhibitors |
Intravenous |
Pepinemab |
Vaccinex |
Phase II |
CD100 antigen inhibitors |
Intravenous |
SRX246 |
Azevan Pharmaceuticals |
Phase II |
Vasopressin 1a receptor antagonists |
Oral |
SAGE-718 |
SAGE Therapeutics |
Phase II |
NMDA receptor modulators |
Oral |
WVE-003 |
WaVe life Sciences |
Phase I/II |
RNA interference |
Intrathecal |
AMT 130 |
UniQure Biopharma |
Phase I/II |
Gene silencing |
NA |
LPM3770164 |
Luye Pharma Group |
Phase I |
Vesicular monoamine transporter 2 inhibitors |
Oral |
SOL-175 |
SOLA Biosciences |
Preclinical |
Gene transference |
Parenteral |
Learn more about the emerging Huntington's disease pipeline therapies @Huntington's Disease Clinical Trials
Huntington's Disease Therapeutics Assessment
The Huntington's disease pipeline report proffers an integral view of Huntington's disease emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.
Scope of the Huntington's Disease Pipeline Report
- Coverage: Global
- Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
- Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravitreal, Subretinal, Topical
- Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy
- Therapeutics Assessment By Mechanism of Action: Dopamine D2 receptor antagonists, Glutamate modulators, Sigma-1 receptor agonists, HD protein inhibitors, RNA interference, Complement C1 inhibitors, CD100 antigen inhibitors, Vesicular monoamine transporter 2 inhibitors, P38 mitogen-activated protein kinase inhibitors, NMDA receptor modulators
- Key Huntington's Disease Companies: Prilenia Therapeutics, Ionis Pharmaceuticals, Annexon, Vaccinex, Neurocrine Biosciences, SAGE Therapeutics, PTC Therapeutics, WaVe life Sciences, Novartis, Neurimmune Therapeutics, SOLA Biosciences, UniQure Biopharma, Roche, Annexon, Inc., Azevan Pharmaceuticals, SOM Innovation Biotech SA, Asklepios BioPharmaceutical, Luye Pharma Group Ltd., BrainStorm Cell Therapeutics, Voyager Therapeutics, Passage Bio, Locana Bio, Ceptur Therapeutics, Enzerna Biosciences, AFFiRiS AG, Arvinas, Atalanta Therapeutics, Anima Biotech, and others
- Key Huntington's Disease Pipeline Therapies: Pridopidine, Tominersen, ANX-005, Pepinemab, Valbenazine, SAGE-718, PTC-518, WVE-003, AMT 130, Branaplam, NI-302, SOL-176, RO7234292, ANX005, SRX246, Bevantolol, SOM3355, BV-101 Gene Therapy, LPM3770164, Passage Bio Research Project, Huntington disease Research Project, BrainStorm Research Project, Locana Huntington disease Research Project, Ceptur Huntington Research Project, HTT RNA Research project, mHTT Research Project, AFFiRiS Huntington Research Project, HTT Research Project, Anima Biotech mHTT Research Project, and others.
Dive deep into rich insights for FDA-approved drugs for Huntington's disease, visit @Huntington's Disease Drugs
Table of Contents
1. |
Huntington's Disease Pipeline Report Introduction |
2. |
Huntington's Disease Pipeline Report Executive Summary |
3. |
Huntington's Disease Pipeline: Overview |
4. |
Analytical Perspective In-depth Commercial Assessment |
5. |
Huntington's Disease Pipeline Therapeutics |
6. |
Huntington's Disease Pipeline: Late Stage Products (Phase III) |
6.1 |
RO7234292: Roche |
7. |
Huntington's Disease Pipeline: Late Stage Products (Phase II) |
7.1 |
SAGE-718: Sage Therapeutics |
8. |
Huntington's Disease Pipeline: Mid Stage Products (Phase I/II) |
8.1 |
AMT 130: UniQure Biopharma |
9. |
Huntington's Disease Pipeline: Early Stage Products (Phase I) |
9.1 |
LPM3770164: Luye Pharma Group |
10. |
Huntington's Disease Pipeline Therapeutic Assessment |
11. |
Inactive Products in the Huntington's Disease Pipeline |
12. |
Company-University Collaborations (Licensing/Partnering) Analysis |
13. |
Key Companies |
14. |
Key Products in the Huntington's Disease Pipeline |
15. |
Unmet Needs |
16. |
Market Drivers and Barriers |
17. |
Future Perspectives and Conclusion |
18. |
Analyst Views |
19. |
Appendix |
For further information on the Huntington's disease pipeline therapeutics, reach out @Drugs for Huntington's Disease
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