“For most of human history, viruses have been our unseen enemies -- particles of RNA and DNA that hijack our cells. Yet a more astonishing truth lies quietly within us: Nearly 8% of the human genome is viral in origin. Evolution repurposed ancient infections into essential parts of human biology, including the placenta and adaptive immunity. Now scientists are using viruses to treat previously deadly diseases.
Groundbreaking research on adeno-associated viruses, or AAV, in the 1960s led to the discovery that they could deliver therapeutic genes to patients' cells. Because AAV don't reliably integrate into human DNA, their clinical use remains largely confined to diseases affecting tissues where cells have minimal division such as the liver, muscle and eye.
But in the 1990s the virus that causes AIDS turned out to offer a solution. Researchers discovered how to strip away HIV's destructive genes and repurpose it as a "lentiviral vector" -- a tool to deliver therapeutic genes directly to cells and integrate them into a patient's DNA. That expanded gene therapy beyond rare inherited disorders and into complex diseases characterized by rapidly dividing cells. Lentiviral vectors became revolutionary in oncology.
Novartis's Kymriah in 2017 became the first treatment using the technology to gain Food and Drug Administration approval. The therapy reprograms a patient's immune cells to fight leukemia, turning a death sentence into a treatable condition for many. Since then, lentiviral vectors have powered gene therapies that treat diseases from sickle-cell anemia to beta-thalassemia.
These treatments are "ex vivo," meaning they remove the patient's cells and treat them in specialized facilities. The process can take months and cost more than $1 million a dose to make. But a new generation of biotech companies is making gene therapy more accessible through in vivo approaches, in which lentiviral vectors deliver genetic instructions directly inside the body.
In 2025 my company, Umoja Biopharma, treated leukemia and lymphoma patients using an in vivo lentivirus. The therapy is engineered to reprogram a patient's T cells to fight cancer cells -- no cell extraction, no months-long manufacturing, no dangerous chemotherapy preparation, no multimillion-dollar production cost. The innovation could bring the treatment within reach of hospitals and clinics that lack advanced cell-manufacturing infrastructure.
This, along with other lentiviral in vivo treatments from companies like AstraZeneca and AbbVie, points to a future in which reprogramming the immune system could become as routine as receiving a vaccine. The same technology could be adapted to treat autoimmune diseases such as lupus, or even solid tumors resistant to current immunotherapies.
Currently approved AAV in vivo therapies often carry a seven-figure price tag. Zolgensma, an in vivo therapy for spinal muscular atrophy, launched at $2.1 million a dose -- at the time the most expensive drug the FDA ever approved. As the in vivo field expands from AAV therapies to lentiviral-vector-based therapies, those prices could fall sharply. A single manufacturing run for an in vivo lentiviral-vector product could treat tens of thousands of patients, whereas a run for an AAV therapy could treat fewer than 10.
That shift will transform gene therapy from boutique medicine for rare diseases into a mainstream therapy for cancer, cardiovascular disease and beyond. Policymakers and investors need to be ready.
The industry is moving fast. The global cell and gene-therapy market is projected to exceed $100 billion by 2034. Major pharmaceutical companies are racing to secure viral vector manufacturing capacity -- an industry bottleneck that could define competitive advantage for the next decade.
Venture investment in biopharma exceeded $26 billion globally in 2024, with 10% of that total going toward cell and gene therapy. Large pharmaceutical firms -- from AbbVie and Novartis to Johnson & Johnson -- are making partnerships or acquisitions to secure next-generation gene-delivery platforms. Early winners will be companies that can manufacture at scale, lowering price and increasing accessibility.
Yet with innovation comes new problems. The same technology that could cure muscular dystrophy could, in theory, be misused for cosmetic or selective traits. Regulators must navigate these frontiers carefully, balancing innovation with ethics. Transparent oversight, global standards and fair pricing will determine whether this revolution delivers on its promise or fuels public backlash.
If the 20th century belonged to antibiotics and chemotherapy, the 21st may belong to viral vectors -- a development that promises to fight disease by erasing it at its genetic root. It's a reminder that even our smallest adversaries can become our greatest assets.
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Mr. Crisman is a co-founder and chief technology officer of Umoja Biopharma.” [1]
1. How AIDS Helps Cure Cancer. Crisman, Ryan. Wall Street Journal, Eastern edition; New York, N.Y.. 02 Jan 2026: A15.
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