11/9/25
The world of medicine seeks to cure and enhance the well-being of individuals. Medicine today is a team effort, with an ecosystem of professionals driving toward improving, repairing, and protecting human health. It is known that the best way to achieve these breakthroughs is by pushing boundaries and sometimes finding solutions that exist outside conventional healing practices.
Gene and molecular-level interventions are just one example of these unconventional methods that are set to reshape the future of medicine and possibly the genetic makeup of humans forever. Genome editing, or gene editing, refers to a set of technologies that enable scientists to modify an organism’s DNA. CRISPR-Cas9 is the most common gene-editing technology used today. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a natural DNA pattern found in bacteria. It is part of a bacterial immune system that allows bacteria to recognize and cut viral DNA from past infections. Scientists adapted this natural system into a gene-editing technology that uses the Cas9 enzyme attached to a guiding RNA, which locates the target DNA by matching to it and enables slicing at the exact location. Somatic and germline editing are two main types of CRISPR technology.
Somatic editing targets non-reproductive cells of the body to treat an individual. The changes made to these cells are non-hereditary. Many countries are currently researching and applying somatic editing at a clinical level, as it can be used to treat cancer and other genetic diseases. While the number of gene-editing therapies approved by the Food and Drug Administration (FDA) is limited, there exist a few that have been approved, such as Casgevy, for the treatment of patients ages 12 years and up for Sickle Cell Disease (SCD) and beta Thalassemia (TDT). CRISPR technology has also been used to mitigate other health conditions, such as high cholesterol.
The Cleveland Clinic conducted a Phase 1, first-in-human clinical trial overseas to test the safety and effectiveness of a new CRISPR-Cas9 gene-editing therapy called CTX310. The study was conducted between June 2024 and August 2025 on 15 people, aged between 31 and 68 years old, in six sites across Australia, New Zealand, and the United Kingdom. The goal of the study was to determine whether a one-time intravenous infusion of CTX310 could safely lower LDL (“bad”) cholesterol and triglyceride levels in people with severe lipid disorders that did not respond to standard treatments. The Phase 1 trial focused on safety, dosage levels, and short-term biological effects. The results were released in November 2025, showing that the therapy reduced LDL cholesterol by about 50% and triglycerides by about 55% within two weeks and maintained those reductions for at least 60 days, without treatment-related side effects. Phase 2 trials are set to begin in 2026 to test larger groups and long-term outcomes. Participants of the Phase 1 trial will be followed for 15 years per FDA gene-therapy safety guidelines.
Germline editing is the second type of CRISPR technology that focuses on reproductive cells or early-stage embryos before or right after fertilization. These traits are passed down to all future generations and aim to prevent genetic diseases before they can occur or enhance specific traits and phenotypes. As a result, testing is carefully monitored. The embryos are never to be implanted into a womb, and testing must be conducted under strict ethical oversight.
Recently, a new Silicon Valley biotech startup called Preventive has drawn global attention as it pursues embryo-editing technologies aimed at preventing hereditary disease, and preparing for the possibility of implanting a genetically edited embryo to create a baby. The company is backed by major tech investors such as Sam Altman (OpenAI) and Brian Armstrong (Coinbase). As of now, the FDA cannot review trials involving embryo editing, and much of the world maintains extremely strict limits on embryo editing, especially with the intention of live birth. The startup is seeking locations abroad, including the United Arab Emirates (UAE), where rules may be more flexible.
Preventive’s founder, Lucas Harrington, a former student of CRISPR pioneer Jennifer Doudna, says that the company’s mission is to achieve the “responsible advancement” of pre-birth genome editing to prevent severe diseases. He insists that the company is still in the preclinical research phase.
To the public’s knowledge, there has only been one known case where gene-edited embryos were implanted and led to the birth of babies. Chinese scientist He Jiankui announced the birth of the world’s first CRISPR-edited babies in 2018 and was found guilty of illegal medical practices. He was sentenced to three years in prison and fined 3 million Chinese Yuan. Jiankui was joined by two collaborators; together, the trio forged ethics documents that misled doctors and illegally implanted embryos into two women. One woman gave birth to twin girls in 2018, and the other gave birth to a singleton sometime after. The health and genetic status of the children remain unknown and private. Scientists worldwide condemned the experiment, which resulted in the Chinese government tightening its regulations.
CRISPR technology is constantly developing and lies at the intersection of various fields, such as biology, chemistry, and engineering. Both somatic and germline editing come with significant risks, as a single change in the genetic makeup could result in changes that affect a person or even the future of the human race. Unpredictable long-term effects, regulatory uncertainty, and procedural errors are just some of the concerns surrounding this new technology. It is difficult to say whether any ethical framework could adequately navigate this issue, beyond continuing research under extremely strict supervision and maintaining a firm ban on implanting gene-edited embryos for live birth.