pg电子app

Translating groundbreaking science into tomorrow’s cures

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Pioneering the future of genomic medicine

pg电子app uses its deep scientific expertise and proprietary zinc finger technology to create genomic cures for patients suffering from severe diseases for which today鈥檚 medicine can only offer symptom management at best.

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Explore our latest corporate presentation by clicking below to stay informed and gain valuable insights into our company’s vision, achievements, and roadmap.

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Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study

Company pipeline and business development opportunities

Clinical Trials

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.
Prion Disease				None	CTA submission anticipated in Q1 2026
Undisclosed neurology target(s)				None	None

NEUROLOGY PIPELINE - PARTNERED
Partnered Indication	Preclinical	Phase 1/2	Pivotal	Partner	Commentary
Tauopathies				Genentech	August 2024: Announced epigenetic regulation and capsid delivery license agreement
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
ALS/FTD				Alexion	None
Huntington's Disease				Takeda	None

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