Newsletter | June 28, 2022

06.28.22 -- The Steps To Executing Gene Therapy Trials

 
Streamlining Regulatory Submissions For A Novel Advanced Therapy Medicinal Product Trial
 

The European regulatory landscape for advanced therapy medicinal products (ATMPs) is complex, and the approval pathway depends on product classification. This case study describes how product classification may not always be clear-cut, given the proliferation of novel approaches to ATMP development.

Pursuing The Promise Of Gene Therapy: Pathways To Successful Clinical Trials
 

The promise of gene therapy is to provide transformative treatments that meaningfully improve quality of life for patients, many of whom are currently living with debilitating diseases. Explore the emerging landscape of gene therapy and discuss high-level considerations for studies of these advanced therapeutics in this blog.

Designing Robust Gene Therapy Studies
 

To develop a successful gene therapy, sponsors must conduct research studies that balance the need for safety oversight and robust clinical evidence with the challenges of finding the right patients, pairing them with qualified sites, providing informed consent, and confirming they are capable of — and committed to — fulfilling all protocol requirements. Explore the key considerations for designing successful gene therapy trials in the available blog.

5 Tips For Operationalizing Gene Therapy Studies
 

Gene therapy clinical trials are logistically complex studies that require highly qualified sites and engaged patients for success. Explore tips on operationalizing gene therapy studies to optimize site selection and patient recruitment and engagement in this blog.

Site Selection, Manufacturing, And Long-Term Follow-Up In A Transition Phase 1/2 Gene Therapy Trial
 

Designing and conducting a gene therapy trial is a complex undertaking. Learn how understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help bring safe, breakthrough treatments to patients with unmet medical needs.