Company pipeline and business development opportunities
| CORE NEUROLOGY PIPELINE | |||||
| Indication | Preclinical | Phase 1/2 | Pivotal | Partner | Commentary |
| Idiopathic Small Fiber Neuropathy (ST-503) | Data presented at ASGCT 23 | None |
December 2024: IND cleared for iSFN, a type of chronic neuropathic pain. Ph1 study to begin mid-2025 |
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| Prion Disease | Data presented at Prion 24 | None |
Prion CTA anticipated Q4 2025 |
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| Tauopathies | Data presented at ASGCT 24 |
Genentech
|
August 2024: Announced epigenetic regulation and capsid delivery license agreement with Genentech | ||
| Undisclosed |
Genentech
|
August 2024: Announced epigenetic regulation and capsid delivery license agreement with Genentech | |||
| Undisclosed |
Astellas
|
December 2024: Announced capsid license agreement with Astellas for up to five neurological diseases | |||
| ALS/FTD | Data presented at ASGCT 24 |
Alexion
|
None |
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| Huntington's Disease |
Takeda
|
None |
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| OTHER PROGRAMS | |||||
| Indication | Preclinical | Phase 1/2 | Pivotal | Partner | Commentary |
| Hemophilia A (Giroctogene fitelparvovec) | Phase 3 AFFINE data presented at ASH 2024 |
Pfizer
*until April 21, 2025 |
July 2024: Positive topline readout in Phase 3 AFFINE trial. December 2024: Sangamo to regain rights to Hem A following Pfizer’s decision to cease development. |
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| Fabry Disease (Isaralgagene civaparvovec) | Data presented at WORLDSymposium 2025 | None |
October 2024: Agreed Accelerated Approval pathway with FDA using Phase 1/2 data. Potential BLA submission expected 2H 2025. | ||