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ALN-HTT02 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| ALN-HTT02 | EXPERIMENTAL | Participants will be administered a single dose of ALN-HTT02 during the Double-blind Part of the study. Participants who received ALN-HTT02 in the Double-blind part of the study will not receive an additional dose during the Open-label Part of the study. Participants have the option to receive repeat doses of ALN-HTT02 in the Open-label Extension (OLE) Part of the study. |
| Placebo + ALN-HTT02 | PLACEBO_COMPARATOR | Participants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-label Part of the study. Participants have the option to receive repeat doses of ALN-HTT02 in the OLE Part of the study. |
| Name | Type | Description |
|---|---|---|
| ALN-HTT02 | DRUG | ALN-HTT02 will be administered intrathecally |
| Placebo | DRUG | Placebo will be administered intrathecally |
Inclusion Criteria * Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS) Exclusion Criteria * Has significant structural or degenerative neurologic disease other than Huntington's Disease (HD) at screening * Has primary or second...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
ALN-HTT02 is an investigational small molecule being developed for the treatment of Huntington's disease, a progressive neurological disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
ALN-HTT02 targets the huntingtin gene, which is the genetic cause of Huntington's disease. By targeting this gene, the drug aims to address the underlying cause of the condition.
ALN-HTT02 is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company. Alnylam is listed on the stock exchange under the ticker symbol ALNY.
ALN-HTT02 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved for commercial use.
ALN-HTT02 is being studied in a Phase 1 clinical trial with the identifier NCT06585449. This trial is recruiting adult patients with Huntington's disease and is being conducted in Canada, Germany, and the United Kingdom.
ALN-HTT02 is a distinct investigational drug candidate. It is not known to have any alternative names or to be the same as any other approved or investigational medication.