The SIG Beat

NEWS FROM AND ABOUT THE SCIENTIFIC INTEREST GROUPS

NEW SIG: Trans-NIH Gene Therapy Scientific Interest Group

The Trans-NIH Gene Therapy Scientific Interest Group (GRx SIG)was developed to strengthen cross-disciplinary gene-therapy research at NIH. The diversity of gene therapies dictated the need to establish the Gene Therapy SIG to promote synergy across the intramural research program (IRP). For example, the broad clinical areas of interest are rare diseases, pediatric diseases, neurological diseases, infectious diseases, metabolic diseases, and cancer. Approaches and technologies include antisense oligonucleotides; adeno-associated vectors and lentiviral vectors; small-interfering RNAs; and gene-, transcript-, and base-editing technologies. The translational and regulatory aspects of gene therapy span the following areas of interest: target identification, preclinical research and investigational-new-drugs-directed studies (including pharmacokinetics, pharmacodynamics, toxicology, and safety pharmacology), formulation and manufacturing, clinical trials, bioethics, and regulatory affairs.

The goals of the GRx SIG are to 1) provide leadership, vision, and support to promote gene-therapy research and development within the IRP; 2) share knowledge about gene-therapy research across institutions, disciplines, and NIH institutes and centers (ICs); 3) collect, evaluate, and disseminate resources and opportunities for gene-therapy research and development at NIH; 4) promote interactions, collaborations, and mentorship among intramural scientists and clinicians, and with extramural academic and industry researchers; and 5) establish and implement a cohesive roadmap for gene-therapy research at NIH.

The GRx SIG members include program officers, program directors, and intramural investigators from different ICs. SIG activities will be coordinated by a steering committee that oversees the organization of meetings and educational offerings on developing and applying gene therapy within NIH. Extramural academic and industry researchers may be invited to attend as guests. A combination of face-to-face meetings and remote webinars will be held, featuring guest speakers to provide perspective, comments, and expert feedback. The steering committee will communicate the Gene Therapy SIG recommendations and suggestions to the Gene Therapy Taskforce and vice versa. Meetings will be held monthly (days and times to be determined).

To receive e-mail notices of meetings and how to attend, please subscribe to the Gene Therapy LISTSERV at https://list.nih.gov/cgi-bin/wa.exe?A0=gene-therapy-sig and click on the “Subscribe or Unsubscribe” link in the right sidebar. For more information and a schedule of meetings, visit the Gene Therapy SIG web page (https://oir.nih.gov/sigs/gene-therapy-scientific-interest-group) or contact the chair of the Gene Therapy SIG, Bryan Traynor (bryan.traynor@nih.gov), or advisors Avi Nath (NINDS), Donald Lo (NCATS), and Carsten Bönnemann (NINDS).

For a full lists of scientific interest groups, go to https://oir.nih.gov/sigs/view-name