Gene Therapy and Gene Editing Programs

NCATS is involved with multiple programs for accelerating gene-targeted therapies, including gene therapy, antisense oligonucleotide therapy and gene editing. The approaches taken in these programs help speed the development of treatments for multiple rare diseases at a time.

Gene-Targeted Therapy Programs

3D rendering of genetic medicine with DNA isolated.

3D rendering of genetic medicine with DNA isolated. (xsense/Shutterstock)

Bespoke Gene Therapy Consortium

The Bespoke Gene Therapy Consortium (BGTC) is a public–private partnership that aims to develop platforms and standards that will speed the development and delivery of customized, or “bespoke,” gene therapies that could treat the millions of people affected by rare diseases.

Platform Vector Gene Therapy

The Platform Vector Gene Therapy (PaVe-GT) pilot project seeks to increase the efficiency of clinical trial startup by using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies.

Somatic Cell Genome Editing Program

The Somatic Cell Genome Editing (SCGE) program is an NIH Common Fund program co-led by NCATS with the objective of accelerating the development of safer and more effective genome-editing therapeutics.

Conferences and Workshops

NCATS hosts a variety of gene therapy, oligonucleotide and gene editing conferences and workshops. View past events led by NCATS and collaborators.


The TARGETED (Targeted Genome Editor Delivery) Challenge aims to advance genome editing delivery technology. The Challenge is sourcing creative ways to deliver gene editors to somatic cells.


Program Director
Philip John (P.J.) Brooks, Ph.D.
(301) 443-0513

Program Administrator
Deanna Portero
(301) 451-9968