CRO Expertise

Gene Therapy Overview

• Housing and breeding programs for transgenic lines
• Experience with administration of various client’s test articles for multiple
  gene therapy methods e.g. CRISPR, AAV, Oligo, mRNA, RNAi, siRNA,
  ceDNA, antisense
• Proficient in multiple routes of administration of test materials for gene therapy
• Multiple tissue collections according to client requests
• Established and developed in-vitro assays to assess the efficiency of the
  gene therapy treatments e.g. ammonia, hEPO, FIX, FVIII, PHE, luciferase
• Evaluate test compound toxicity by clinical observations and laboratory
  test e.g. liver enzymes

IVIS Imaging

Routes of Administration

• Intravenous
• Hydrodynamic intravenous
• Subcutaneous
• Oral gavage
• Intramuscular
• Nebulization
• Intravitreal
• Intrathecal
• Intratracheal
• Intraarticular
• Intranasal
• Intracerebral ventricular

Summary

Experience with multiple transgenic strains

Proficient with multiple dose administration methods

Established various in-vitro assays

IVIS imaging capabilities

White Papers

Webinars

Guangping Gao, University of Massachusetts Medical School – Gene Therapy for Rare Diseases – A New Paradigm in Medicine

Casey Maguire, Harvard Medical School – Strategies for in vivo Barriers to Gene Therapy Vectors

Jeffrey Vick, Chameleon Biosciences Inc. – Chameleon’s EVADER Technology: A more potent AAV vector with less immune response

Miguel Muñoz-Alía, Mayo Clinic – Retargeting and Shielding Measles Virus Vectors for Oncolytic Virotherapy

Molly Shoichet, University of Toronto – Regenerative Strategies in the Central Nervous System, Part One

Molly Shoichet, University of Toronto – Regenerative Strategies in the Central Nervous System, Part Two

Technical Sheet

Gene Therapy Overview

Articles

AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear

Therapeutic efficacy in a hemophilia-B model