FREQUENTLY ASKED QUESTIONS

FOR THE GENERAL PUBLIC

  • Our sole mission is to prevent and ultimately eradicate devastating inherited diseases. Our aim is to develop and deploy precise gene correction strategies to ensure that no child is born with a genetic condition like Huntington's disease, cystic fibrosis, or inherited forms of cancer. Our focus is exclusively on health and medical applications.

  • Our work is strictly focused on correcting gene mutations that cause severe diseases with significant burden and cost to the healthcare system, but more importantly, with unimaginable pain and suffering to the patients and their families. Our ethical framework is built around the principle of healing and reducing suffering from inherited diseases. Gene therapy companies have overpromised and underdelivered for these patients. With advancements in gene editing technologies, it should be a public health prerogative to put genetic disease in the past.

  • Our research is centered on understanding and safely correcting genetic defects in early human embryos. To do this responsibly, we follow a careful, step-by-step process that puts safety first. We refine our gene correction protocols using proxy animal and advanced human 3D cellular models to ensure they are safe and effective. While many of these gene correction protocols are no longer novel and unexplored, and are currently in use in pre-clinical and clinical trials, this methodical approach allows us to independently build a strong foundation of data before applying these proven techniques to early human embryos (which we will acquire with regulatory approval and full consent from donors). For those interested in a more detailed, technical overview of our methodologies and long-term research plan, we invite you to read the "For the Scientific Community" section of our FAQ.

  • Safety and ethics are the cornerstones of our work. All our research is conducted in the United States, adhering to all applicable US federal and state regulations, including the highest standards for animal welfare. We are building on decades of research and will only advance our work when we have robust data demonstrating its safety and efficacy. We believe the most ethical path forward is to conduct this research in a transparent and regulated environment where the public has the right to learn and try, and the voice to determine the future.

  • No, an early human embryo is not a fetus or a developing baby in a womb. This research happens at a microscopic level, just days after fertilization and long before a pregnancy could ever be established. We work exclusively with embryos that have been donated with full, informed consent by individuals who have chosen not to use them for their IVF journey. These embryos would otherwise be unusable or discarded (e.g., tripronuclear zygotes). Furthermore, to advance this science while reducing the reliance on donated embryos, we are actively exploring innovative approaches, such as creating embryo models from stem cells in the lab.

  • Our mission is to eradicate genetic disease and democratize the treatment to anyone in need and open to this approach. By pioneering this research now, we aim to create a future where preventing genetic disease is a standard part of healthcare and available to anyone consenting.

  • The technology that finally balances extraordinary precision with a high degree of safety is here, and we feel a profound responsibility to act now. Recent breakthroughs in gene corrections have given us tools to locate and accurately correct the specific genetic errors that cause devastating conditions like cystic fibrosis or Huntington's disease. A future free from these inherited burdens is now an achievable goal, and we are dedicated to using these advanced and responsible tools to make that future a reality for families everywhere.

  • We believe a moratorium would be counterproductive and ultimately harmful. Scientific progress is inevitable and highly beneficial. A ban in the US would not stop the research; it would simply push it to countries with less regulatory oversight, increasing risks. We advocate for a transparent, regulated, and open approach in the US, where we can ensure the highest ethical and safety standards are met, fostering a productive public conversation informed by rigorous data.

FOR THE SCIENTIFIC COMMUNITY

  • Our therapeutic strategy is not limited to a single technology; instead, we employ a versatile portfolio of advanced gene-editing tools to ensure we use the optimal custom strategy for each specific disease target. Our capabilities are focused on addressing a broad spectrum of genetic abnormalities, including the precise correction of single nucleotide variants (SNVs), which are the cause of many devastating monogenic disorders. Furthermore, we are equipped to perform targeted insertions and deletions of larger genetic fragments for more complex variations, and we are developing novel approaches to correct large-scale, chromosome-level abnormalities such as duplications and translocations.

  • Establishing safety and efficacy is our most critical commitment, which we address through a multi-layered process of independent oversight and rigorous research. Every stage of our research plan is reviewed and must be approved by our global bioethics board, composed of independent, world-renowned bioethicists who ensure our work is safe, effective, and ethically sound. Concurrently, our advisory board reviews key milestones to guarantee our work remains strictly aligned with our vision for the medical-only application of this technology. Our research pathway begins in proxy animal and advanced human 3D cellular models, and moves to human embryos upon approval from all relevant regulatory bodies.

    Scientifically, safety is assessed through rigorous whole-genome sequencing and advanced computational analysis to detect any off-target edits. We also conduct extensive research in established proxy species and complex human cellular models to evaluate the long-term health and multi-generational impact of any genetic correction. Efficacy is determined by the precise and consistent correction of the target gene mutation, leading to the intended therapeutic outcome. We commit to publicly share our findings on safety and efficacy.

  • Our research is centered on human-relevant models. Our primary models include advanced human 3D cellular systems. Ultimately, the most relevant model for this work is the early human embryo itself, which we study under strict ethical and regulatory guidelines. Where necessary for foundational safety studies or to meet specific regulatory requirements, we also utilize established proxy animal systems, always in full compliance with NIH and IACUC guidelines.

  • We plan to publish our findings in leading pre-print servers and peer-reviewed journals. We are also dedicated to sharing our data with the scientific community in a manner that is consistent with intellectual property considerations and our collaborative agreements.

  • We are committed to accelerating progress in genomic medicine by directly funding academic labs with synergistic research. Our primary collaboration model is the sponsored research agreement (SRA), through which we provide the necessary financial resources for our partners to conduct cutting-edge research in their areas of expertise. We believe in true partnership and structure our collaborations to be mutually beneficial, which includes the potential for sharing intellectual property. If you are a principal investigator working on relevant research, please reach out to us through our contact page to discuss a funded partnership.