Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
John Bernat
Modality
gene_therapy
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06488261CT.gov Long Term Follow-Up for Safety of AVR-RD-02 | — | Active Not Recruiting | — | — | — |
NCT04145037CT.gov Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease | — | Terminated | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Gaucher Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Avr-Rd-02 (this asset) | John Bernat | P1/2 | CCC · 48 |
| Taliglucerase Alfa | Protalix | P4 | A |
| Velaglucerase Alfa | Takeda | P4 | A |
| .Alpha.-D-Glucosyl-N-Acylsphingosine Glucohydrolase | Sanofi | P4 | A |
| Eliglustat | Peking Union Medical College Hospital | P3 | BBB |
| Cerezyme® / Imiglucerase | University Hospital, Clermont-Ferrand | P4 | BBB |
| Isofagomine | Amicus Therapeutics | P2 | BB |
| Sofosbuvir | Mansoura University Children Hospital | P4 | BB |
| Ledipasvir | Mansoura University Children Hospital | P4 | BB |
+20 more in the Gaucher Disease cohort
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Avr-Rd-02 in Gaucher Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/avr-rd-02-gaucher-disease
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