Asset profile

VGN-R08b

Gene therapy / editing

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Earlyevidence

Evidence outlook

Early-stage evidence only

Phase 1 / first-in-human studies establish safety, tolerability, and dosing; they are not designed to demonstrate efficacy, so no evidence signal is assigned yet. The first graded signal arrives with a completed confirmatory readout.

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Competitive landscape

64 competitors in this universe: 4 direct rivals (same mechanism and indication), 0 same-class, 60 same-indication; 62 further along in development.

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DrugStageModalityMechanismEvidence signalReported efficacygraded featured trials
VGN-R08b: this drugPhase 1/2Gene therapy / editingGBA1/glucocerebrosidase
AmbroxolPhase 3Small moleculeGBA1/glucocerebrosidase
GT-02287Phase 2Small moleculeGBA1/glucocerebrosidase
PR001 (LY3884961)Phase 1/2Gene therapy / editingGBA1/glucocerebrosidase
CAP-003Not yet studiedGene therapy / editingGBA1/glucocerebrosidase
Amantadine ER (Gocovri / ADS-5102)ApprovedSmall moleculeNMDA receptor
Apomorphine (Apokyn)ApprovedSmall moleculedopamine
Apomorphine Sublingual Film (Kynmobi)ApprovedSmall moleculedopamine
Crexont (IPX203)ApprovedSmall moleculedopamine precursor
Entacapone (Comtess)ApprovedSmall moleculeCOMT
Foslevodopa / Foscarbidopa (Vyalev)ApprovedSmall moleculedopamine precursor
Inbrija (CVT-301)ApprovedSmall moleculedopamine precursor
Istradefylline (Nourianz)ApprovedSmall moleculeadenosine A2A
Levodopa-Carbidopa Intestinal Gel (Duopa)ApprovedSmall moleculedopamine precursor
Opicapone (Ongentys)ApprovedSmall moleculeCOMT, PDE4
ParcopaApprovedSmall moleculedopamine precursor

…and 49 more. Research shows the complete landscape.

Research

Unlock the full comparison

Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.

Upcoming events

(2 total)

Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.

2027 (1)
September 2027 (1)
Phase 1/2Parkinson's DiseaseNot yet recruiting
Est. primary completion 2027-09-01 · NCT07414290 · CT.gov ↗
What to watch
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2028 (1)
May 2028 (1)
Phase 1/2Gaucher Disease Type 3Not yet recruiting
Est. primary completion 2028-05-29 · NCT07603050 · CT.gov ↗
What to watch
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See the full readout calendar ↗

All registered studies

Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.

Phase 1/2 (2)
Phase 1 (1)