CHDI 2025: Route to a safe delivery strategy of AAV-based gene therapy to the caudate and putamen of nonhuman primates
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CHDI 2025: SPK-10001 induces a durable and safe reduction of HTT protein supporting further development in Huntington’s disease
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CHDI 2025: HTT exon 1 splice variants are non-allele specific and expressed at low levels in human brain
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Phase 1/2 dirloctocogene samoparvovec PD/PK analysis
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HSG 2024: SPK-10001 GLP-tox Poster
SPK-10001 induces a durable and safe reduction of HTT protein supporting further development in Huntington’s disease Download FIle
HSG 2024: SPK-10001 ROA Poster
Route to a safe delivery strategy of AAV-based gene therapy to the caudate and putamen of nonhuman primates Download FIle
HSG 2024: SPK-10001 Pharmacology poster
SPK-10001 AAV-based microRNA mediates non-allele specific reduction of HTT mRNA through RNA interfernce, demonstrating its potential for further preclinical development. Download FIle
NBDF 2024: Long-term Follow-up of Participants in the Phase I/II Trial of Dirloctocogene Samoparvovec (SPK-8011): Durable FVIII Expression and Clinically Meaningful Reduction of Bleeding
NBDF 2024 – Long-Term Follow-up of Participants in the Phase I/II Trial of Dirloctocogene Samoparvovec (SPK-8011): Durable FVIII Expression and Clinically Meaningful Reduction of Bleeding Powered By EmbedPress
WFH 2024 – Phase 1/2 QOL
WFH – SPK-8011-101 Phase 1/2 QOL Download FIle
WFH 2024 – Phase 1/2 Joint Health
WFH – SPK-8011-101 Phase 1/2 Joint Health Download FIle