Gene and RNA Therapies Rebound with a Surge in Startups and New Targets

Summary

The field of genetic medicine is experiencing a significant resurgence. According to a recent industry report, worldwide approvals have now reached 42 gene therapies, 38 RNA therapies, and 76 cell therapies that are not genetically modified. This clinical success is mirrored by a rebounding business landscape. In the first quarter of the year, 26 new startup companies were formed in this sector, a number that sits well above the historical average of five to twenty.

Alongside this growth, researchers are shifting their focus toward a wider variety of diseases. While these advanced treatments have historically been associated with fighting cancer, over 60 percent of current gene therapy clinical trials are now targeting non-cancer conditions. In the realm of RNA medicine, a fascinating split is occurring. Therapies based on RNA interference (a technique known as RNAi that helps silence faulty genes) are seeing strong pipeline growth. On the other hand, therapies using messenger RNA (mRNA, which gives cells temporary instructions to make proteins) are actually experiencing a decline quarter over quarter.

Despite these promising signals, it is important to note that most of this research is still in its infancy. While the overall pipeline of potential new medicines is massive, the vast majority of these assets remain in the earliest stages of human testing. Currently, there are 669 gene therapy trials in Phase I or Phase II (the initial stages to check safety and basic effectiveness) compared to only 41 trials that have made it to the final Phase III stage.

Why it matters.

This ongoing evolution in gene and RNA therapies signals a major turning point for patients living with untreated genetic conditions. Because the majority of new gene therapy trials are moving beyond oncology to target other diseases, entirely new patient populations may soon have access to cutting-edge treatments. The sharp increase in new startup formations also suggests that investors are renewing their confidence in the field. This financial backing is essential for taking complex laboratory discoveries and turning them into real-world medicines.

However, the path from early clinical trials to pharmacy shelves is notoriously long. Because only a small fraction of these experimental therapies have reached late-stage Phase III trials, most of these potential treatments are still years away from reaching patients. Even so, the sheer volume of early-stage research provides a robust foundation for the future of medicine, promising a steady stream of new therapeutic options over the coming years.

From the source

The story in numbers.

Therapies currently in development: >4,200
Increase in start-up funding vs Q1 2025: 30%
Gene therapy Phase III trials: 41

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This AI-assisted summary is a starting point. Read the original reporting for its methods, scope, and limitations.

First inside-the-body CRISPR gene therapy heads to the FDA

Intellia Therapeutics is making medical history as it seeks approval from the US Food and Drug Administration for a groundbreaking genetic treatment. The drug, called lonvoguran ziclumeran or lonvo-z, is the first CRISPR-based gene therapy designed to work "in vivo," meaning the genetic editing happens directly inside the human body. CRISPR is a biological tool that allows scientists to make precise changes to DNA. Intellia is using this technology to treat hereditary angioedema, a genetic condition that causes severe and unpredictable swelling attacks. The company recently completed a Phase 3 clinical trial, which is the final testing step before seeking regulatory approval. In the study, a single dose of lonvo-z reduced swelling attacks by 87 percent compared to a placebo over a six-month period. Even more strikingly, 62 percent of the patients who received the treatment had absolutely no attacks during those six months and were able to completely stop taking their usual preventative medications. While these numbers are highly encouraging, the research does come with a few early caveats. The trial was relatively small, involving a total of 80 patients, with 52 receiving the actual therapy and 28 given a placebo. Additionally, the currently reported results only track the patients for six months. Researchers will need to monitor these individuals for a longer period to confirm that the single dose continues to work safely and effectively over time.

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Promising Duchenne Gene Therapy Sets Sights on FDA Approval

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For decades, doctors have used harmless viruses known as adeno-associated viruses, or AAVs, as biological delivery trucks to treat genetic diseases. These viruses are engineered to carry healthy genes directly into a patient's cells. But a newly published study reveals an unexpected complication. Researchers recently linked a specific brain tumor in a five-year-old boy to an AAV9 gene therapy he received as an infant to treat an inherited disorder called MPS1 deficiency. When the boy was five, doctors discovered a rare neuroepithelial tumor in his brain. By sequencing the genetic material of the mass, scientists found that the gene therapy payload had accidentally woven itself into the boy's own genome. Specifically, the viral delivery cassette fused with a human gene called PLAG1, which is known to drive tumor growth. Curiously, the tumor also contained a stray fragment of a different human chromosome, which researchers suspect was a manufacturing contaminant from when the viral vector was created. Thankfully, the story has a positive outcome so far. Surgeons successfully removed the tumor. Eight months after the operation, the patient shows no signs of the tumor returning and is actually demonstrating advanced cognitive development.

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Follow your curiosity.

Gene and RNA Therapies Rebound with a Surge in Startups and New Targets

Summary

Why it matters.

The story in numbers.

Follow the evidence.

Your next discovery.

First inside-the-body CRISPR gene therapy heads to the FDA

Promising Duchenne Gene Therapy Sets Sights on FDA Approval

Gene Therapy Linked to Rare Brain Tumor in a Single Patient

Summary

Why it matters.

The story in numbers.

Follow the evidence.

Your next discovery.

First inside-the-body CRISPR gene therapy heads to the FDA

Promising Duchenne Gene Therapy Sets Sights on FDA Approval

Gene Therapy Linked to Rare Brain Tumor in a Single Patient

A little science goes a long way.