Gene Therapy

We specialize in preclinical safety studies that help advance novel gene therapies to testing in human clinical trials. Our team consults on experimental design and content of pre-IND packages, determines the safety and efficacy of the gene therapy products and provides final reports for submission to US and EU regulatory agencies. Lovelace Biomedical has been conducting studies in gene therapy for more than a decade, making us one of the country’s longest-running gene therapy programs in the preclinical space.

Gene Therapy Toxicology: Download

Gene Therapy Toxicology: Download

Capabilities include:

  • Development of novel models to evaluate efficacy of gene therapy approach
  • Monitoring of of unwanted lymphocyte or antibody responses to vector or expressed protein
  • Measurement of distribution of the vector in the body and transgene expression in the target location
  • Evaluation of safety and efficacy of the gene therapy product

Gene Therapy Toxicology

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

100+ Years

running complex IND-enabling programs to support gene therapy development

fifty

50+

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

threelakhs

300,000

square feet lab space, including GLP-compliant operations

million

$204 million

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

10-years

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 for over 12 years. 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.

Gene Therapy Toxicology Expertise

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