Company pipeline and business development opportunities
| NEUROLOGY PIPELINE - WHOLLY OWNED | |||||
| Indication | Preclinical | Phase 1/2 | Pivotal | Partner | Commentary |
| Idiopathic Small Fiber Neuropathy (ST-503) | None |
IND cleared, patient enrollment and dosing planned mid-2025. |
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| Prion Disease | None |
CTA submission anticipated in Q1 2026 |
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| Undisclosed neurology target(s) | None |
None |
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| NEUROLOGY PIPELINE - PARTNERED | |||||
| Partnered Indication | Preclinical | Phase 1/2 | Pivotal | Partner | Commentary |
|
Genentech
|
August 2024: Announced epigenetic regulation and capsid delivery license agreement | ||||
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Undisclosed neurology target
|
Genentech
|
August 2024: Announced epigenetic regulation and capsid delivery license agreement | |||
|
Undisclosed neurology target
|
Astellas
|
December 2024: Announced capsid license agreement for up to five neurological diseases | |||
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Alexion
|
None |
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Takeda
|
None |
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| OTHER PROGRAMS | |||||
| Indication | Preclinical | Phase 1/2 | Pivotal | Partner | Commentary |
| Hemophilia A (Giroctogene fitelparvovec) |
Pfizer
*until April 21, 2025 |
July 2024: Positive readout in Phase 3 AFFINE trial. | |||
| Fabry Disease (Isaralgagene civaparvovec) | None |
October 2024: Agreed Accelerated Approval pathway with FDA. BLA submission expected 2H 2025. | |||
Cargo Capsid