In 2026, the FDA proposed a novel approach to expedite reviews of gene therapies for patients with rare genetic disorders such as cystic fibrosis, potentially bypassing the need for years-long clinical trials if approved.

A planned clinical trial to treat a rare lysosomal storage disorder in utero has buoyed hopes of providing prenatal gene therapies, but it was still awaiting FDA approval to begin enrolling patients as of May 2026.

In 2023, the FDA approved gene therapy for sickle cell disease, enabling one-time infusion bespoke treatments that use a patient's own stem cells to effectively cure a painful condition that disproportionately affects people of African American descent.

Gene therapy, a hope of the Human Genome Project, has successfully treated a baby born with a rare urea cycle disorder that causes toxic ammonia level buildup.

The US FDA considered mitochondrial replacement therapy during 2014 hearings

An agency committee assessed if this approach could have undesirable ripple effects across multiple generations and whether a clinical trial involving this approach would be appropriate. As of 2026, the US has continued to prohibit this procedure though it is legal in other settings including the UK and Australia.

A single-gene mutation in the FOXC2 gene gave actress Elizabeth Taylor two rows of eyelashes.

How personalized gene-editing saved a baby's life

KJ Muldoon had a 50% chance of surviving a genetic mutation until doctors developed a one-of-a-kind gene-editing therapy. In an interview with one of the researchers behind the treatment, learn what it will take to make personalized genetic medicine possible for everyone.

The US FDA recently tried to regulate the expanded prenatal genetic tests

The agency proposed a new rule in 2023 to make it explicit that these types of tests are devices under existing law and clarify that that the agency could move to regulate them. They finalized a regulatory change in 2024 that added the words "including when the manufacturer of these products is a laboratory" to an existing law, but a federal district court vacated that edit in March 2025—returning the law to as it was before.

Gene therapy treats a disease by editing the disease's underlying DNA, replacing it with a functional variant.

Gene therapy can occur inside the body, but it can also be completed outside the body—with scientists treating cells in the lab, and then those corrected cells are transplanted back into the body.

Genetic drug matching, called pharmacogenomics, can help avoid dangerous drug reactions

Certain gene variants can affect how quickly or slowly medications are broken down in the body, affecting drug effectiveness and the risk of drugs failing. Such testing is often not covered by insurance companies though research indicates if these tests were performed for more than six drugs across the US population, avoided ailments would likely climb into the hundreds of thousands. (Some users may encounter a paywall.)

The first CRISPR-approved treatment was for sickle cell anemia in 2023

Victoria Gray was the first patient to receive a CRISPR-based therapy, which eliminated her symptoms and restored her health. The therapy's initial cost of $2.2M has kept it out of reach of many patients.

See how a novel gene editing technique called base editing can create changes in a single DNA base, using a more precise targeting approach than CRISPR-Cas9.

Scientists using gene therapy recently managed to regenerate retinal cells in the lab—a breakthrough for mammals that could potentially lead to future treatments for human retinal diseases.

Genetic engineering will change everything forever

Designer babies, the end of diseases, genetically modified humans that never age. Outrageous things that used to be science fiction are suddenly becoming reality. As scientists increasingly understand how specific genes cause disease, the possibility of engineering our genes to not only "fix" problems but enhance our natural ability grows.

As of February 2025, there are more than 150 clinical trials using CRISPR technology

The clinical trials are designed to determine whether experimental CRISPR-related therapies are safe and effective. The majority of trials involve treatments for blood disorders, including beta thalassemia.

A new daily MS pill is seeking regulatory approval in 2026

In clinical trials, Roche's fenebrutinib showed promise treating primary progressive MS and doubling relapse-free time in patients with relapsing MS as compared to existing treatment. But some liver damage and two possible drug-related deaths in the trials may make it difficult for the drug to net approval. (Some users may encounter a paywall.)

Gene therapy is restoring hearing among kids with genetic deafness

Gene therapy for profound genetic deafness has been advancing in recent years, with multiple clinical trials enabling hearing at normal levels for the first time without hearing aids or cochlear implants. The therapies target hearing loss caused by mutations in the OTOF gene that codes for a protein called otoferlin. That protein normally plays a key role in relaying sound signals in synaptic transmission between the ear's inner hair cells and the auditory nerve.

CRISPR is a gene-editing tool that lets scientists cut out and replace damaged DNA

One of the most significant advances in modern medical science, CRISPR is a molecular tool for making precise changes to a gene's DNA. The technology has already been approved for treating sickle-cell disease.

Gene therapy for a rare type of deafness is allowing deaf children and adults to hear for the first time, according to research results reported in April 2026.

Restoring vision in impaired patients via CRISPR

Since CRISPR was revealed as a gene editing tool in 2012, it has been on an accelerated track to real-world applications. Among other clinical demonstrations, doctors have now injected a CRISPR treatment directly into eye cells, helping vision-impaired patients see shapes and colors again.

A first-of-its-kind clinical trial taps livers from gene-edited pigs to clean blood

The experimental dialysis-like treatment from United Therapeutics, eGenesis, and OrganOx, is designed to help people with liver failure. The therapy, which involves attaching the modified pig liver externally, filters a patient’s blood so their own livers can temporarily rest and perhaps heal.

Somatic expansion, the lifelong growth of specific repeating DNA sequences, is being studied as a potential treatment target for Huntington's disease and other disorders. (Paywall may apply.)

The promises and perils of using at-home genetic tests for your health

A primer on genetic testing, personalized medicine, and the promise of pharmacogenomics–the use of genetics to predict an individual's response to a drug.

The 'Armamentarium' is a set of new gene therapy tools—including viruses that could shuttle therapies into the body—designed to fight brain diseases.

Peptides like GLP-1 account for 31 of 370 new drugs approved by the US FDA between 2016 and 2023.

In 2025, researchers in the UK began working on developing artificial DNA

The Synthetic Human Genome Project aims to one day construct human DNA, molecule by molecule, in the hopes of creating disease-resistant cells and synthetic chromosomes for study. However, critics warn it may be misused to create enhanced or modified humans.

US regulators have approved an at-home genetic test looking for BRCA gene mutations

The direct-to-consumer genetic test, approved by US regulators in 2018, examines 3 out of the more than 1,000 known breast cancer-linked genetic mutations. It looks for genetic variations that can increase risk for breast, ovarian, and other cancers. These mutations are most commonly found among Jewish people with Eastern European ancestry.

Explaining CRISPR at five levels of technical detail

CRISPR is a new area of biomedical science that enables gene editing and could be the key to eventually curing diseases like autism or cancer. WIRED challenges biologist Neville Sanjana to explain this concept to 5 different people; a 7-year-old, a 14-year-old, a college student, a grad student and a CRISPR expert.

Pharmaceutical companies can use ChatGPT to improve drug development

Moderna has incorporated the tool into its workflows to accelerate analysis and identify optimal drug dosage from clinical data. The company's legal team also uses a custom GPT—Contract Companion—to break down and summarize contracts quickly.

Visualize how CRISPR-Cas9—a type of immune system used by single-celled organisms to attack invasive viruses—targets and makes precise changes to a gene's DNA.

Australia legalized 3-parent IVF in 2022

It was the second country, after the UK, to legalize the procedure, which is still prohibited in the US. There are more than 300 rare mitochondrial diseases that can be passed from a mother to her offspring.

Silicon Valley startups Orchid, Nucleus, and Genomic Prediction are selling so-called polygenic tests for prospective parents

These tests claim to show how at-risk a hypothetical baby would be for genetic syndromes. The newfound industry has prompted ethical and medical debates, with critics questioning their accuracy and highlighting the potential problems behind pursuing the perfect baby.

A man with organ failure used AI to speed discovery of his miracle drug

David Fajgenbaum had a few months to live after a diagnosis with a rare form of Castleman's disease. By studying his blood, medical records, and broader literature, he was able to identify the cause of his condition and a treatment. He's been in total relapse for ten years.

Eight British children have been born via mitochondrial replacement therapy

In July 2025, a decade after British lawmakers signed off on allowing three-parent IVF, scientists announced that eight healthy babies had already been born using this approach. The news was included in a pair of research articles published in a peer-reviewed journal that noted the oldest kids were already more than two years old, and that they were hitting developmental milestones as expected. (Some users may encounter a paywall.)

CRISPR is a gene-editing tool that lets scientists cut out and replace damaged DNA

Originally a bacterial defense system against viruses, CRISPR uses guide RNA and a protein called Cas9 to identify and cut specific DNA sequences. Scientists can repurpose this system to switch off faulty genes, insert new ones to treat genetic diseases, or modify organs grown in other animals so that they appear less foreign to a human immune system.

Precision medicine, offering the right treatment at the right time for individuals, integrates genomics, data analytics, and AI to predict risks, personalize treatments, and improve patient outcomes.

3-parent baby technique fuels worries of future 'designer babies'

Some critics of this approach worry that any genetic manipulation will both culturally and technologically normalize the idea of "designer babies"—embryos that are manipulated or selected based on physiological characteristics including height, eye color, athletic ability, and intelligence.

Carrier screening enables individuals without symptoms to see if they carry a gene for disease

Some conditions are autosomal recessive—meaning a person may carry a gene for that condition, but not have symptoms. Cystic fibrosis, sickle cell disease, and Tay-Sachs disease are examples of autosomal recessive diseases in which a person must inherit two copies of a nonfunctioning gene—one from each parent—to develop disease.