What’s Been Invested
Since 2008, approximately $5 million has been contributed to PTEN related research in the laboratories of Drs. He and Steward through the National Institute of Health, private foundation grants and philanthropic donations which has verified the proof of concept for this therapy.
We Are Seeking
We are now seeking to raise $3 million in investment to evolve the PTEN inhibition and viral vector delivery system for therapy. A private placement of 400,000 shares of series seed preferred stock will be given to accredited investors.
Timeline and Milestones
To realize our goal, Axonis will aggressively pursue the following milestones. These milestones represent the best-case scenario. Biological experiments are very complicated and results cannot be guaranteed.
Plasmid Development
Contracting, Plasmid Design (3-4 variants), Plasmid Synthesis, Vector Process Evaluation, Final Plasmid synthesis(Research Grade)
July 2019 - January 2020
Research Grade Vector Batch
Contracting, Upstream Vector Production, Downstream Purification, Analytical, Formulation & Packaging, Release
December 2019 - March 2020
Analytical Methods Validation
Identity, Sterility, Adventitious Agents, Mycoplasma, Endotoxin, Purity
February 2020 - August 2020
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
Stability GMP Batch
August 2021 - January 2022
GMP Manufacturing
Plasmid synthesis (GMP Batch); GMP Clinical Batch: Upstream Vector Production, Downstream Purification, Analytical, Formulation & Packaging, Release
August 2021 - January 2022
Stability GMP Clinical Batch
January 2022 - January 2024
Potency Assay
Development, Qualification, Validation
March 2020 - March 2021
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
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
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
3 month rat GLP toxcicity and biodistribution study
August 2021 - February 2022
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
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
INTERACT Meeting Prep
INTERACT Briefing Package, Meeting request, INTERACT Meeting
March 2019 - July 2019
Pre-IND meeting
Meeting request, Briefing Package, PIND Meeting
March 2021 - May 2021
IND
Drafting IND sections, drafting Investigator Brochure, publishing and eCTD compilation, e-submission, 30-day review period, Active IND
February 2022 - June 2022