About the Clinical Vector Core

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The Clinical Vector Core (CVC), situated within a world-renowned research campus that includes Children’s Hospital of Philadelphia and the University of Pennsylvania, partners with in-house and external investigators to advance their early-stage therapeutics to clinical trials, bringing life-changing precision medicine treatments to patients with with rare and ultra-rare diseases.

Embedded in the fabric of the gene and cell community at CHOP, the CVC team has a special connection to those young patients visiting CHOP’s medical campus located in Philadelphia, a hub of scientific and medical innovation known as “Cellicon Valley.”

The CVC manufactures adeno-associated virus (AAV) and lentiviral (LV) vectors in a new state-of-the-art current Good Manufacturing Practices (cGMP) facility that became operational in 2021 in the Colket Translational Research Building. Their local, national, and international success results from an impressive proven track record over more than 17 years based on:

  • World-class expertise
  • Consistency
  • Quality Control and Quality Assurance
  • Affordability

CVC Clinical Applications

Clinical Indications

Clinical Vector Core Milestones

  • Milestone 1
    Raymond G. Perelman Center of Cellular and Molecular Therapeutics

    The CVC is part of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT). Founded in 2005, Beverly Davidson, PhD, Chief Scientific Strategy Officer of CHOP Research Institute, currently directs the CCMT, which has the capability to move basic pre-clinical discoveries rapidly into clinical application.

  • Milestone 2
    American Recovery and Reinvestment Act Program (ARRA)

    The ARRA award granted by the National Institutes of Health in 2010 supported the CVC’s ability to optimize workflows and provide a higher level of efficiency at a time when the new field of gene therapy was often considered a high-risk investment.

  • Milestone 3
    Spark Therapeutics Launched

    Spark Therapeutics spun off from CVC in 2013 to advance the execution of safe and effective gene therapy clinical trials. Hoffmann-La Roche acquired Spark Therapeutics in 2019 for $4.3 billion.

  • Milestone 4
    First-ever Gene Therapies Approved by U.S. Food and Drug Administration

    Collaborating with scientists at the University of Pennsylvania, CVC experts produced the first early-phase vectors for CAR T-cell therapy to treat advanced acute lymphoblastic leukemia, as well as vectors that supported the development and approval of a product used to treat a rare form of inherited blindness. Both were approved in 2017 by the U.S. FDA.

  • Milestone 5
    Grand Opening of CVC’s Clinical Manufacturing Facility

    CHOP’s investment in building a new Clinical Manufacturing Facility in 2018 demonstrated CVC’s ongoing commitment as a global leader in the production of clinical-grade biotechnology tools and enhanced its capacity to bring more cell and gene therapies to patients. The facility’s novel design facilitates standardized manufacturing and quality processes and procedures compliant with cGMP and, at the same time, is flexible enough to accommodate the custom process that is typical for early stage of pre-clinical and clinical development investigational drugs. The facility received an International Society of Pharmaceutical Engineering 2021 Facility of the Year award.

  • Milestone 6
    CVC’s Innovation Today

    To date,* CVC has helped to produce over 177 unique products supporting clinical trials and treatment of more than 814 patients. CVC-manufactured products have been used in support of 39 clinical trials at 99 study sites in 10 countries around the world.

    *As of April 2024