The number of gene therapy candidates in development worldwide exceeded 2,150 at the start of 2026, with the United States and China leading the field. Under government plans as part of the national project “New Technologies for Health Preservation,” Russia aims to develop around 25 gene therapy drugs and personalised cell products by 2030. One such therapy is expected to arrive next year at a facility run by the Pirogov Russian National Research Medical University (RNRMU).
In March 2026, US researchers reported successful testing of a new method for safely inserting large DNA fragments into mouse genomes, opening broad prospects for gene therapy. The method, described in the journal Nature, uses circular single‑stranded DNA rather than the typical double‑stranded form. It is called INSTALL.
With the approach now proven effective in mice, it is being tested as a therapy for liver diseases, blood disorders and metabolic conditions, Fierce Biotech reported, citing one of the scientists involved in developing the genome‑editing technique.
To develop the new method, the researchers drew inspiration from certain bacteria and viruses that naturally insert circular single‑stranded DNA fragments into double‑stranded genomes. The technology delivers long DNA strands into the genome with greater precision than viral vectors. INSTALL uses lipid nanoparticles for delivery instead of the viruses typically employed in gene therapy.
Gene replacement therapy is divided into viral and non‑viral delivery methods. In viral delivery, the shell of a virus is taken, its own DNA removed, and the desired gene inserted inside for administration to the patient. Examples include adenoviruses, lentiviruses and herpesviruses. Non‑viral delivery typically uses lipid microparticles – tiny lipid spheres containing DNA molecules. These spheres are injected into the body, fuse with cell membranes and thus deliver the gene into cells.
Scientists say INSTALL could be used to treat diseases caused by the absence of large gene segments, such as Prader‑Willi syndrome or Lejeune syndrome (also known as cri‑du‑chat syndrome). With INSTALL, there will be no need for personalised gene therapy, they added. “We will have one tool to treat 100 mutations,” the researchers said.
Of course, this will not happen soon, and there will still be limitations on the number of diseases that can be treated with this method – a universal therapy for genetic disorders will never exist.
Genome‑editing technology was first approved in 2023. It was cleared for use in Britain to treat sickle‑cell anaemia and beta‑thalassaemia – specifically the CRISPR method, which was also authorised for the same indications in the United States and Europe. Worldwide, CRISPR is being tested for conditions including hepatitis B and Down syndrome.
Attempts to create CRISPR‑based drugs are also underway in Russia, with Sechenov University among those involved.
The Pirogov RNRMU and the Kulakov National Medical Research Centre for Obstetrics, Gynaecology and Perinatology are also planning to develop gene therapy.
The press service of Pirogov RNRMU told GxP News that construction of its own manufacturing facility for gene therapy drugs – such as Zolgensma for spinal muscular atrophy and Elevidys for Duchenne muscular dystrophy – is scheduled for completion by the end of 2026. These expensive drugs are currently purchased by the Circle of Good foundation for children under 18. Bringing similar technology to Russia would immediately solve many problems, the university said.
RNRMU clarified that production is scheduled to begin in 2027. The entire drug development cycle will take three months, after which the medicine will be administered to the patient following a series of necessary safety tests and approval by the Health Ministry’s ethics committee.
The university also explained that it will produce gene replacement therapy using the classical approach – delivering a working copy of the gene into the patient’s cells – as well as the newer genome‑editing technology based on CRISPR/Cas editors.
