NIH Announces Consortium to Streamline Gene Therapy Research
The National Institutes of Health (NIH) announced this week that they were joining forces with the Food and Drug Administration (FDA), ten pharmaceutical companies and five non-profit organizations to accelerate the development of gene therapies for rare diseases. The new public-private partnership, called the Bespoke Gene Therapy Consortium, aims to overcome obstacles and streamline the process of developing therapies that could treat diseases that collectively impact millions of people.
The consortium will fund basic and clinical research from a five-year budget of $76 million. This represents an opportunity for investigators. For institutions interested in participating in the expected clinical trials, it may be time to revisit their policies for biosafety and gene transfer. Institutional Biosafety Committees (IBC) are required at institutions that conduct NIH-funded recombinant DNA, or gene therapy, research.
An IBC is concerned for the protection of not only research subjects, but also staff and communities in which the research takes place. The committee has oversight for establishing, monitoring, and enforcing policies and procedures for handling biohazardous materials, such as recombinant DNA.
An IBC works in parallel with an Institutional Review Board (IRB), with special attention to risk assessment for areas including:
- Study agent
- Containment levels and procedures required to safely handle the study agent
- Preparedness of the facility and its personnel
- Potential impact to the environment
Activities of the IBC include:
- Review agent characteristics (e.g. virulence, pathogenicity, environmental stability)
- Determining the appropriate biosafety level for physical and biological containment, as required by the NIH Guidelines
- Inspection of facilities, procedures, and practices
- Evaluating the training and experience of the personnel involved in the research
- Confirming appropriate policies and procedures are in place for handling spills or accidents to minimize exposure to or contamination from any potentially hazardous material
- Reporting significant events or violations to regulatory authorities including NIH and institutional officials
- File an annual report with the NIH
NIH requires that an IBC have at least five members who collectively have broad experience and expertise that allows them to conduct such assessments. This may include members with technical expertise in potentially hazardous biological materials, human research protocols, regulatory requirements, and in the health and protection of the community and environment. Additionally, at least two members must be unaffiliated with the organization conducting the research, and these members should represent the interest of the surrounding community with respect to health and protection of the environment.
The establishment and funding of the Bespoke Gene Therapy Consortium will increase the opportunity for investigators to participate in basic and clinical research to address significant unmet medical needs of patients. The safe conduct of experiments involving recombinant or synthetic nucleic acid molecules depends on the individual conducting such activities, as well as having appropriate safety mechanisms at the institutional level.
BRANY offers IBC services that can help expedite the review of research that involves recombinant DNA (“rDNA”) or synthetic nucleic acid molecules, or DNA or RNA derived from recombinant or synthetic nucleic acid molecules, providing rigorous biosafety oversight so that institutions can focus their efforts on scientific research and advancement. Contact us for more information.