• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • JobWatch
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Peter Marks Envisions New Development Model for Gene Therapies

Peter Marks Envisions New Development Model for Gene Therapies

April 11, 2022

To expand the field of gene therapy, Peter Marks, director of the FDA’s Center for Biologics Evaluation (CBER), believes that the current clinical development framework is in need of rethinking in terms of trial design, endpoints, communication and other areas.

As it stands now, gene therapy developers commonly refer to the clinical development framework for small-molecule drugs, Marks wrote in an editorial published in Expert Opinion on Biological Therapy. While many components of this framework apply to gene therapies — good manufacturing practices (GMP), understanding nonclinical aspects of the investigational drug and demonstrating safety and effectiveness, for instance — other aspects of gene therapies might be better served by other models, the CBER chief believes.

Marks posed that greater use of novel trial designs and endpoints, global regulatory harmonization, standardized manufacturing processes and improved communication with regulators would have a big impact on getting more gene therapies to market.

Marks believes that sponsors should consider the use of Bayesian clinical trial designs that include reassessments of therapeutic benefit probability as each patient goes through the trial process, as these measures can help to lower the number of participants needed in gene therapy trials and the small pools of rare disease patients that can make enrollment a struggle.

Additionally, because current gene therapies use a device-like “vector backbone,” most often an adeno-associated virus or lentivirus, to deliver an artificial gene, Marks thinks that the reuse of backbone-related information should be considered, as appropriate, for other gene therapies in development. Successfully doing this could accelerate development for multiple products.

Access Marks’ article here: https://bit.ly/3NPD1Vv.

 

To view more CenterWatch Weekly stories, click here.

Upcoming Events

  • 28Jun

    Effective Root Cause Analysis and CAPA Investigations for the Life Sciences

  • 16Oct

    WCG MAGI's Clinical Research Hybrid Conference - 2022 West

Featured Products

  • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

  • Surviving an FDA GCP Inspection

    Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

Featured Stories

  • Protocol-360x240.png

    Avoid Deviations by Making Protocol Review a Team Effort

  • SelectionProcess-360x240.png

    Give Us a Voice: Sites Clamor for a Say on Vendor Selection

  • Convince-360x240.png

    Use Data and Details to Convince Site Leadership to Add Staff

  • AsktheExpertsBadge-360x240.png

    Ask the Experts: Listing Trial Staff and Others on the Statement of Investigator

Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

The information you need to adapt your monitoring plan to changing times.

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy
  • Do Not Sell My Personal Information

Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2022. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing