Gene Therapy

Lovelace Biomedical has one of the longest-running preclinical gene therapy toxicology programs of any contract research organization, with more than a decade of experience conducting preclinical studies to evaluate the safety, efficacy and biodistribution of gene therapy products.

Lovelace gene therapy toxicology program consults on experimental design and content of pivotal, pre-IND packages and provides final reports for submission to U.S. and European regulatory agencies. Preclinical studies in gene therapy are run in compliance with GLP standards in species ranging from rodents to nonhuman primates.

What We Do

  • Develop novel study designs to concurrently measure safety and efficacy of gene therapies
  • Monitor immune responses to vector or expressed protein
  • Measure biodistribution of vector and transgene expression
  • Evaluate traditional and non-traditional dosing routes

Endpoints That Matter

  • Efficacy
  • General toxicity
  • Vector biodistribution (qPCR)
  • Gene expression
  • Immunogenicity

10+ Years running complex IND-enabling programs to support gene therapy development

50+ lovelace Biomedical scientific staff experienced with pre-IND gene therapy programs

300,000 square feet lab space, including GLP-compliant operations

$204 million estimated sales of gene therapy products in the U.S. by 2020

Gene Therapy Toxicology Expertise

Lovelace Biomedical is the leading CRO for performing Gene Therapy nonclinical development. We have been working with the FDA and scientists with leading pioneers in the industry to advance gene therapy products to the clinic. In 2007 our Gene Therapy pharmacology/toxicology Center was established by the NIH along with pioneers at the University of Pennsylvania. We have maintained this core for over 12 years, and have built on its foundation a successful resource for biotechnology and pharmaceutical companies aiming to advance their candidates from discovery to clinical stage by GLP enabling studies as rapidly as possible.

*Lovelace Biomedical, through relationships with the NIH, working directly with the Gene Therapy Resource Program (GTRP), and also working closely with the FDA, allows us to have direct insight to “pre IND enabling,” new gene therapy development. Although we are not responsible for directly setting forth regulatory guidelines, we can offer direction and design for your program with our vast experience in gene therapy, to get your product to the next stage of IND development.

Gene Therapy Toxicology

Highlights

End Points

  • PCR Development & Validation
  • Biodistribution & Gene Expression
  • Clinical Pathology/Histopathology

Regulatory Support

  • Pre IND
  • Pre IND Package Development

Specialized Dose Delivery

  • Inhalation
  • Intra-Articular
  • Cerebral
  • Epicardial
  • Ocular
  • Intravenous Infusion
  • Spinal

Disease Models

  • Animal Model Propagation
  • Animal Model Development

Animal Models

  • NHP
  • Canine
  • Swine
  • Rabbit
  • Ferret
  • Guinea Pig
  • Rat
  • Mouse