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Plasmid Development

Contracting, Plasmid Design (3-4 variants), Plasmid Synthesis, Vector Process Evaluation, Final Plasmid synthesis(Research Grade)

July 2019 - January 2020
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Research Grade Vector Batch

Contracting, Upstream Vector Production, Downstream Purification, Analytical, Formulation & Packaging, Release

December 2019 - March 2020
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Analytical Methods Validation

Identity, Sterility, Adventitious Agents, Mycoplasma, Endotoxin, Purity

February 2020 - August 2020
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GLP Manufacturing

Plasmid synthesis (GLP Batch): Order, Synthesis, QC; GLP Demo Batch: Upstream Vector Production, Downstream Putification, Analytical, Formulation & Packaging, Release

February 2021 - August 2021
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Stability GMP Batch

August 2021 - January 2022
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GMP Manufacturing

Plasmid synthesis (GMP Batch); GMP Clinical Batch: Upstream Vector Production, Downstream Purification, Analytical, Formulation & Packaging, Release

August 2021 - January 2022
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Stability GMP Clinical Batch

January 2022 - January 2024
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Potency Assay

Development, Qualification, Validation

March 2020 - March 2021
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Rat 1-month POC transduction expression, biodistribution, knockdown

Protocol, animal arrival & acclimation, AAV dose stereotactic procedure, in-life 1 wk cohort, in-life 1 mth cohort, histopath, biodistribution, protein knockdown, draft report

April 2020 - July 2020
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Rat SCI efficacy/tox study

Protocol, animal arrival & acclimation, injury, AAV dose stereotactic procedure, inlife (3 staggered 12 week cohorts 1 wk apart), motor assessment, tracer injection, survival to necropsy (4wks), histology, draft report

July 2020 - February 2021
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NHP 1-month non-GLP exploratory, dose optimization and biodistribution study

Protocol, animal arrival & acclimation, AAV dose stereotactic procedure, inlife, histopath, biodistribution, protein knockdown, draft reforp

December 2020 - April 2021
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3 month rat GLP toxcicity and biodistribution study

August 2021 - February 2022
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3 month NHP GLP toxicity and biodistribution study

Protocol, animal arrival & acclimation, AAV dose stereotactic procedure, inlife, histopath, biodistribution, draft report

August 2021 - March 2022
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Clinical FIH Phase 1 Study

CRO selection and Contracting, Protocol, IRB approval, Screening, Ph 1/2a enrollment period, FIH Dose, Sentinel Cohort, Main Cohort, database lock, CSR

November 2021 - June 2024
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INTERACT Meeting Prep

INTERACT Briefing Package, Meeting request, INTERACT Meeting

March 2019 - July 2019
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Pre-IND meeting

Meeting request, Briefing Package, PIND Meeting

March 2021 - May 2021
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IND

Drafting IND sections, drafting Investigator Brochure, publishing and eCTD compilation, e-submission, 30-day review period, Active IND

February 2022 - June 2022