Staff Profile: Philip J. (P.J.) Brooks

Philip J. (P.J.) Brooks
Philip J. (P.J.) Brooks, Ph.D.

Acting Director

Division of Rare Diseases Research Innovation

National Center for Advancing Translational Sciences

National Institutes of Health

Email Philip J. (P.J.) Brooks


Philip J. (P.J.) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. Prior to his tenure in the division, he worked in NCATS’ Division of Clinical Innovation, where he was the lead program director for the Clinical and Translational Science Awards (CTSA) Program Collaborative Innovation Awards, designed to fund projects that will result in novel and creative approaches to overcoming roadblocks in translational science (PAR-18-244 and PAR-18-245). Brooks represents NCATS in the NIH-wide Gene Therapy Working Group and on the Regenerative Medicine Innovation Project. He also is the working group coordinator for the NIH Common Fund program on Somatic Cell Genome Editing and is one of the leaders of the PaVe-GT pilot project. Brooks recently was elected as the chair of the Interdisciplinary Scientific Committee of the International Rare Diseases Research Consortium.

Brooks received his doctorate in neurobiology from The University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at The Rockefeller University, he became an investigator in the intramural program conducted by the National Institute on Alcohol Abuse and Alcoholism. He developed an internationally recognized research program focused on two distinct areas: the molecular basis of alcohol-related cancer; and rare neurologic diseases resulting from defective DNA repair, including xeroderma pigmentosum, Cockayne syndrome and Fanconi anemia.

Research Topics

Brooks’ research on rare genetic diseases gave him the opportunity to meet with patients and their families, which had a powerful impact on his view of the importance of translational science in rare diseases. He believes that nucleic acids have clear therapeutic potential for single-gene disorders, as indicated by recent approvals of oligonucleotide therapeutics and gene therapy. However, the ability to deliver these complex molecules to the affected cell types remains the limiting factor in many diseases. Brooks developed funding opportunities (PAR-20-098 and PAR-20-109) to support innovative approaches to delivering genome editors to target cell types.

Brooks is interested in accelerating clinical trials in rare diseases by moving beyond “one disease at a time” approaches. Examples include the development of therapeutics that target shared molecular mechanisms underlying multiple rare diseases and the implementation of platform vector gene therapy trials via the NCATS Collaborative Rare Disease Platform Vector Gene Therapy Trial cooperative agreement.

Selected Publications

  1. The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup
  2. The NIH Somatic Cell Genome Editing Program
  3. Expanding Rare Disease Drug Trials Based on Shared Molecular Etiology
  4. Blinded by the UV Light: How the Focus on Transcription-Coupled NER Has Distracted from Understanding the Mechanisms of Cockayne Syndrome Neurologic Disease