OUR ETHICS STATEMENT

1. Prevention is the best and most affordable treatment

2. Ethics should be driven by reducing human suffering

3. Our responsibility is to deliver options to patients

4. Data should drive regulatory approval

Manhattan Project was founded with the mission to end genetic disease and alleviate pain and suffering. Inherited genetic diseases caused by mutations in the genetic code can be fatal during development, or have massive implications in health, well-being and life-span post-birth. They are passed down to future generations, thus propagating the cycle of suffering with limited safe, cheap and readily available treatments in sight. It doesn’t have to be this way.

Powerful gene correction technologies exist that can fix the mutations in the gametes, or as the embryo first develops. There is a magical moment in time when the parents’ germ cells combine to give rise to the fused egg-sperm cell called a zygote, and which offers an incredible window of opportunity to fix severe disease-causing mutations safely, faster, and cheaper.

Billions of dollars flow into companies promising gene therapy and gene correction treatment for genetic disease, but significant challenges with delivery of these therapies into billions of cells simultaneously have hampered efforts to deploy and democratize cheap treatment after decades of promises. These avenues are important, and should be explored in their entirety, but a different approach exists today to eliminate the waiting and the pain in the next generations.

The UK has led the way for the past decade to successfully regulate and explore some advanced assisted reproductive technologies. One incredible example is the technology behind mitochondria replacement therapy (MRT) that eliminates mitochondrial genetic diseases during in vitro fertilization (IVF). By using healthy donor mitochondria at the embryo formation stage, born children do not have to carry maternal mutations, and parents do not have to pass down debilitating diseases. There is an ethical path forward to adopting novel technologies.

The public deserves to know what is possible openly and with evidence-backed arguments, and the parents should have options to give their children a future without avoidable disease.

Yet calls for forever moratoria on human germline gene editing, and fears of misuse have stopped exploration of this work which harbors great benefits if regulated appropriately. The call for continuing to halt this work is rooted in legitimate concerns like the safety of gene correction tools. Fortunately, these powerful technologies have become more precise and efficient over the past decade, and use in cellular and animal models has been achieved successfully. In fact, most of these technologies are currently in clinical trials for individuals suffering from genetic disease. A very successful 2025 case is that of baby KJ who received CRISPR therapy to treat severe carbamoyl phosphate synthetase 1 deficiency.

Moratoria to postpone the germline correction of disease-causing mutations create a paradox. They prevent the very research in embryo models needed to develop the safety frameworks they call for without providing a guideline or framework on how to make sure the technologies are safe for embryos. By prohibiting research in appropriate embryo models, these moratoria prevent the essential work required to test the efficacy and safety of these tools. We firmly believe safety is a concern to be comprehensively assessed and addressed, and our approach is to provide scientific evidence to be shared publicly of what these technologies are capable of before deployment, and what defined data-driven risk factors are observed.

Another important concern is the use of these technologies in broader cases that do not involve treatment of disease and are more aesthetic in nature, sparking fears of eugenics. We are fully aligned in this vision to narrow the use of these technologies to genetic correction to prevent disease and provide crucial health and lifespan benefits to the future generations. We believe a transparent regulatory framework through state and federal legislation that defines the stricter use and subjects it to FDA regulations and approval, is an appropriate avenue to ensure the proper and equitable use of these treatments.

We are encouraged by well-written reports by a few prominent bioethicists as well as fundamental, important steps to be addressed, outlined in the study “Heritable Human Gene Editing” in 2020, commissioned by the National Academy of Medicine, National Academy of Sciences, and the Royal Society. We agree to bring in all the stakeholders to have thoughtful, open and urgent conversations on clinical use of germline gene correction technologies. Our aim is to bring together a committee of bioethicists to frame the scientific work and the approach to regulatory bodies.

We must openly approach the ethical and philosophical arguments of allowing genetic correction as a somatic cell treatment, despite non-negligent harm inflicted by inflammatory reactions of current inefficient delivery mechanisms, but stop it from a safer deployment before the disease has taken root.

We believe Manhattan Project is a catalyst for this change in perspectives. We advocate for a thoughtful approach to the revision of Dickey-Wicker and Aderholt provisions amendments that limit funding and reviewing of work pertaining to human heritable gene editing. The public deserves a transparent and active effort to interrogate and leverage these technologies at all stages of human development and lifespan – for healthier humans, for more affordable treatments that do not feed burdensome insurance costs, and for the benefit of public good.