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Also known as AAV-GAD Low Dose
AAV-GAD · 2 trials · 1 indication
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related treatment-emergent adverse events.
The primary outcome measure is the safety of treatment with AAV-GAD, assessed by the absence of IMP-related serious treatment-emergent adverse events.
| Arm | Type | Description |
|---|---|---|
| Follow-up group | NO_INTERVENTION | Participants who were randomized to immediate treatment in Study MGT-GAD-025 will transition directly to a long-term follow-up schedule to complete an additional 54 months of follow-up. All study participants are to be followed for 60 months after vector administration. |
| Active treatment group | EXPERIMENTAL | Participants who were randomized to sham surgery in Study MGT-GAD-025 will transition to an active treatment schedule, including open-label bilateral treatment, upon confirmation of continued eligibility. Upon completion of the treatment period, participants will enter the long-term follow-up schedule to complete a total of 60 months of follow-up. |
| AAV-GAD Low Dose | EXPERIMENTAL | Eligible participants received bilateral infusion of AAV-GAD low dose into the STN |
| AAV-GAD High Dose | EXPERIMENTAL | Eligible participants received bilateral infusion of AAV-GAD high dose into the STN |
| Sham Surgery | SHAM_COMPARATOR | Eligible participants underwent a sham surgical procedure |
| Name | Type | Description |
|---|---|---|
| AAV-GAD | GENETIC | Bilateral infusion of AAV-GAD |
| AAV-GAD Low Dose | GENETIC | Bilateral infusion of AAV-GAD low dose |
| AAV-GAD High Dose | GENETIC | Bilateral infusion of AAV-GAD high dose |
| Sham Surgery | PROCEDURE | Sham infusion |
Inclusion Criteria: * Previously enrolled in Study MGT-GAD-025. Exclusion Criteria: * None
Top 12 of 20 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Supernus Pharmaceuticals, Inc. | SUPN | 2 | PHASE3 | Onapgo (SPN-830) |
| Prothena Corp. Plc | PRTA | 3 | PHASE3 | Prasinezumab |
| Biohaven Ltd. | BHVN | 1 | PHASE2 | BHV-8000 |
| Denali Therapeutics Inc. | DNLI | 1 | PHASE2 | BIIB122 |
| AC Immune SA | ACIU | 2 | PHASE2 | ACI-7104.056 |
| Gain Therapeutics, Inc. | GANX | 2 | PHASE2 | GT-02287 |
| Eli Lilly and Company | LLY | 3 | PHASE1 | LY3884961, LY4006896, LY3962681 |
| Annovis Bio Inc | ANVS | 1 | PHASE2 | Buntanetap |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | ALN-SNCA |
| MeiraGTx Holdings Plc | MGTX | 1 | PHASE1 | AAV-GAD |
| AgeX Therapeutics, Inc. | SER | 1 | PHASE1 | SER-252 |
| Ardelyx, Inc. | ARDX | 1 | PHASE2 | Tenapanor |
AAV-GAD is an investigational gene therapy being developed for Parkinson's disease. It delivers the gene for glutamic acid decarboxylase into the brain with the goal of restoring GABA production in the affected neural circuits. It is not approved and remains in Phase 1 clinical development.
AAV-GAD targets GAD1 and GAD2, the genes encoding glutamic acid decarboxylase, the enzyme that converts glutamate into GABA. By introducing these genes via an adeno-associated viral vector, the therapy aims to increase GABA synthesis in the subthalamic region and rebalance the excitatory and inhibitory signaling that is disrupted in Parkinson's disease.
AAV-GAD is being developed by MeiraGTx Holdings plc, which trades on the Nasdaq under the ticker MGTX. The company holds an RMAT designation from the FDA for the program, which covers the gene therapy in Parkinson's disease.
AAV-GAD is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by the FDA. Two Phase 1 trials have been conducted in Parkinson's disease, one completed and one active but no longer recruiting, with a combined enrollment of 27 participants.
AAV-GAD has been studied in two Phase 1 trials. NCT05603312 was a double-blind, no-treatment-controlled study of glutamic acid decarboxylase gene transfer in Parkinson's participants, and it is completed. NCT05894343 is a long-term follow-up study of GAD gene transfer in Parkinson's disease and is active but not recruiting.
Yes, AAV-GAD Low Dose is another name used for AAV-GAD. Both refer to the same MeiraGTx gene therapy program for Parkinson's disease, which uses an adeno-associated viral vector to deliver the glutamic acid decarboxylase gene. The low dose label distinguishes the dose level studied in the clinical program.