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Also known as VY-AADC02, AAV2-hAADC
VY-AADC01 · 3 trials · 9 indications
Grading will be assessed using NCI CTCAE, version 4.03.
Safety of VY-AADC01 will be assessed by any clinically significant abnormalities on MRI scans as compared to Baseline.
Safety of VY-AADC01 will be assessed by routine physical examinations.
Safety of VY-AADC01 will be assessed by routine clinical laboratory analysis.
C-SSRS is a standardized suicidal rating system.
Safety and Tolerability of AADC Gene Transfer assessed by Adverse Events and Serious Adverse Events.
| Arm | Type | Description |
|---|---|---|
| VY-AADC02 (NBIb-1817) | EXPERIMENTAL | Single administration of up to 3.6 x 10\^12 vector genomes (vg) of VY-AADC02 |
| Sham (Placebo) Surgery | PLACEBO_COMPARATOR | Sham surgical procedure |
| VY-AADC01 Single Dose | EXPERIMENTAL | 9.4 x 10\^12 vector genomes of VY-AADC01 |
| VY-AADC01 Dose 1 | EXPERIMENTAL | 7.5 x 10\^11 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| VY-AADC01 Dose 2 | EXPERIMENTAL | 1.5 x 10\^12 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| VY-AADC01 Dose 3 | EXPERIMENTAL | 4.7x 10\^12 vector genomes of VY-AADC01; Single dose, neurosurgically infused, bilaterally into the striatum. |
| Name | Type | Description |
|---|---|---|
| VY-AADC02 | BIOLOGICAL | Adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2- hAADC) infusion into the brain |
| Sham (Placebo) Surgery | OTHER | Bilateral partial burr/twist holes without dura penetration |
| VY-AADC01 | DRUG | Single dose, neurosurgically infused, bilaterally into the striatum. |
Key Inclusion Criteria: 1. Males and females, 40 to 75 years of age (inclusive) 2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria 3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale...
Top 15 of 23 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Supernus Pharmaceuticals, Inc. | SUPN | 2 | PHASE3 | Onapgo (SPN-830) |
| Prothena Corp. Plc | PRTA | 3 | PHASE3 | Prasinezumab |
| Denali Therapeutics Inc. | DNLI | 1 | PHASE2 | BIIB122 |
| Bayer | BAYRY | 4 | PHASE3 | bemdaneprocel, AB-1005 |
| Biohaven Ltd. | BHVN | 1 | PHASE2 | BHV-8000 |
| AC Immune SA | ACIU | 2 | PHASE2 | ACI-7104.056 |
| Eli Lilly and Company | LLY | 4 | PHASE1 | LY3884961, LY4006896, LY3962681 |
| Gain Therapeutics, Inc. | GANX | 2 | PHASE2 | GT-02287 |
| Annovis Bio Inc | ANVS | 1 | PHASE2 | Buntanetap |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | ALN-SNCA |
| MeiraGTx Holdings Plc | MGTX | 1 | PHASE1 | AAV-GAD |
| Serina Therapeutics, Inc. | SER | 1 | PHASE1 | SER-252 |
| Ardelyx, Inc. | ARDX | 1 | PHASE2 | Tenapanor |
| Hoffmann-La Roche | RHHBY | 1 | PHASE1 | RO7568282 |
| Merck & Co., Inc. | MRK | 1 | EARLY_PHASE1 | MK-0947 |
VY-AADC01 is an investigational gene therapy being developed by Neurocrine Biosciences, Inc. (ticker: NBIX) for Parkinson's disease. It is designed to deliver the AADC gene directly into the brain to restore dopamine production in patients with advanced Parkinson's disease. It is currently in Phase 1 clinical development.
VY-AADC01 is being studied for the treatment of idiopathic Parkinson's disease and Parkinson's disease with motor fluctuations. It is intended for patients with advanced Parkinson's disease who experience motor complications. The therapy is investigational and has not been approved for any indication.
VY-AADC01 is being developed by Neurocrine Biosciences, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol NBIX. Neurocrine acquired the program and is responsible for its clinical development in Parkinson's disease.
VY-AADC01 is in Phase 1 clinical development. Two Phase 1 trials have been completed, and no active trials are currently listed. The therapy remains investigational and has not received FDA approval.
VY-AADC01 has been evaluated in two completed Phase 1 trials: NCT03065192, a safety and efficacy study in advanced Parkinson's disease, and NCT01973543, a safety study of AADC gene therapy. A related trial, NCT03562494 (RESTORE-1), studied VY-AADC02 in Parkinson's disease with motor fluctuations.
VY-AADC01 and VY-AADC02 are related gene therapy candidates. VY-AADC01 was studied in earlier Phase 1 trials, while VY-AADC02 was evaluated in the Phase 1 RESTORE-1 trial (NCT03562494) for Parkinson's disease with motor fluctuations. Both are being developed by Neurocrine Biosciences.