Enterprise Value

434.5M

Cash

63.37M

Avg Qtr Burn

-26.77M

Short % of Float

5.72%

Insider Ownership

12.01%

Institutional Own.

62.27%

Qtr Updated

09/30/23


Drug Pipeline

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Drug & IndicationStage & EventCatalyst Date
Bota-vec (Botaretigene sparoparvovec) (AAV-RPGR) Details
X-linked retinitis pigmentosa, Genetic disorder, Eye disease

BLA

Submission

AAV-RPE65 test Details
Eye disease , Retinal dystrophy

Phase 3

Initiation

AAV-CNGB3 Details
Achromatopsia, Genetic disorder, Autosomal recessive congenital ichthyosis , Skin disease/disorder

Phase 2b

Update

AAV-CNGA3 Details
Achromatopsia

Phase 2b

Update

AAV-hAQP1 Details
Salivary gland

Phase 2

Data readout

AAV-GAD Details
Parkinson's disease

Phase 1

Data readout