The NIH Common Fund’s Somatic Cell Genome Editing (SCGE) program is bringing genome editing technology to the clinic through the collaborative power of its consortium model. Genome editing uses precise tools to modify a specific part of a person's DNA or RNA. These changes can help treat or prevent diseases, including rare genetic conditions with few or no effective treatments. Through the SCGE Consortium, researchers share ideas, data, and lessons learned to overcome common challenges and bring promising and safe treatments to patients sooner. This consortium also provides a critical space for researchers to share their experiences navigating the complex U.S. Food and Drug Administration (FDA) regulatory system for new treatment approval.
In keeping with SCGE’s commitment to sharing knowledge, these lessons learned are made publicly available to patients, caregivers, communities, healthcare providers, and researchers through the SCGE Platform.
Investigational New Drug (IND) Application Resource for Gene Therapy
When new therapies are brought to the FDA for approval, they often go through the Investigational New Drug (IND) approval process. An IND application is submitted to the FDA if a drug (or biological product) not previously authorized in the U.S. is intended to be used for clinical investigation or treatment. Because gene editing therapeutics are often new and target rare diseases, many approvals go through the IND pathway. Once a therapy clears the IND stage, it can move to clinical trials for further FDA approvals.
Companies developing new treatments may be reluctant to share data because of commercial concerns. As a result, industry or research groups may repeat work or ask the FDA questions that other groups already resolved. The SCGE platform brings together researchers’ experiences into a roadmap to demystify regulatory steps for new gene therapy approvals. It explains guidance broken down by specific regulations, requirements for each type of gene therapy submission, and timing of when to submit packages. The toolkit also provides standardized templates and examples from approved gene editing therapies. This helps first-time IND submitters get started and prepare an effective submission.
SCGE Shared Regulatory Documents
As groups pursue IND submissions, they seek FDA input on how best to generate evidence to support regulatory approval. While private industry often keeps this input confidential, the SCGE platform makes questions and FDA responses available from which anyone can read and learn. Combined with the IND toolkit, these shared regulatory documents create windows into FDA thinking that can facilitate a broader understanding or regulatory decision-making, particularly among those pursuing INDs.
Gene Therapy Clinical Trial Browser and SI Tools
The SCGE platform compiles all ongoing and historical gene therapy clinical trials across nearly 50 countries, including trials that are not SCGE-supported. As of October 2026, the browser lists more than 400 clinical trials, allowing researchers and patients to better understand the gene therapy landscape through filters such as disease indication, recruitment status, FDA trial type designation, clinical trial phase, and many others.
The SCGE platform also includes a custom super intelligence (SI) chatbot to help users navigate its extensive resources. While it cannot be relied on for regulatory advice, the SI assistant can help users with various levels of expertise search the platform quickly, and efficiently.
The FDA provides an important regulatory function to ensure therapeutics in the U.S. are safe and effective. However, the necessary steps to get drug approvals can be complex and challenging to understand, particularly for groups that have never worked with the FDA before. As the SCGE Consortium pushes the frontiers of gene-editing science, these unique, invaluable tools ensure that knowledge is not only used by its funded investigators but also returned to the public for broader impact.
