A new publication authored by the Precision Health Alliance, a consortium sponsored and coordinated by the Duke Clinical Research Institute (DCRI), stresses the need for an unprecedented degree of collaboration across the scientific community. According to the authors, this level of cooperation is essential to ensure that gene editing interventions are patient-centered, safe, and ethical.
The peer-reviewed article, “Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing” was published recently in Human Gene Therapy and showcases the leadership of the Precision Health Alliance and the DCRI in the field of gene editing and precision medicine. The authors outline eight major themes that warrant careful consideration:
- Objectives (treatment, risk reduction, and enhancement) for gene therapy;
- Competing interests of safety, equity, and desire for research efficiencies;
- Unique aspects of gene editing related to rare and ultrarare genetic conditions;
- Considerations in the pediatric population;
- Regulatory requirements and ethics oversight;
- Challenges with long-term follow-up and data sharing; and
- Communication.
As the field of gene therapy rapidly evolves, the authors argue that its success and ethical implementation depend on three linked commitments: protecting patients while responsibly advancing science, embedding equity at every stage from study design to health care delivery, and building harmonized governance systems.
Authored by several DCRI researchers and partners across academic and industry organizations, the article reflects DCRI’s long‑standing commitment to ethically advancing innovative, data‑driven approaches to clinical research.
"The DCRI's ability to bring together multidisciplinary expertise across clinical research, translational science, biostatistics, and regulatory strategy to the global research community serves as a model for achieving this goal of careful collaboration," DCRI Executive Director Adrian Hernandez, MD, MHS, said.
In addition to Hernandez, DCRI authors include Lauren Cohen, MA, Tammy Reece, MS, Hannah Webster, MPH, and Kevin Weinfurt, PhD.
“Gene editing represents one of the most transformative advances in modern medicine, but its success will depend on more than scientific progress alone. Building an aligned ethical and regulatory framework will be critical to realizing its full potential for patients,” said first author Pishoy Gouda, MB, MSC, an associate faculty member at the University of Alberta and recent DCRI fellow.
The article is based on a multistakeholder workshop of Precision Health Alliance partners and subsequent conversations. The Alliance aims to engage a variety of stakeholder groups, including patients, clinicians, health care leaders, health care systems, life science companies, regulatory experts, and bioethicists. The article stressed that this collaborative model is increasingly essential as precision medicine initiatives grow in complexity and scale, and as companies encounter similar challenges regardless of therapeutic area or population.
“As precision medicine continues to evolve, the DCRI remains focused on addressing the scientific, operational, and ethical considerations required to bring the most innovative and effective therapies to patients safely and efficiently,” Hernandez said.
The Alliance is supported by AskBio/Bayer, BEAM Therapeutics, CRISPR Therapeutics, the DCRI, and Intellia Therapeutics.