Russia Legalizes 'Bespoke' Gene Therapy Without Registration to Save Hopeless Patients
The Ministry of Health has proposed allowing the use of individualized gene therapy drugs created in clinics for specific patients. This approach would give a chance to children with orphan diseases for which developing a mass-market drug is unprofitable, although experts demand strict safety controls.
An eight-year-old boy with an extremely rare genetic defect was dying because no standard drug existed for him—pharmaceutical companies find it unprofitable to develop a drug for a market of one. In April 2026, the Russian Ministry of Health published a draft law that legalizes the creation of gene therapy drugs for specific patients directly in clinics, bypassing the lengthy state registration procedure. If adopted, Russia will become one of the few countries where 'bespoke' gene therapy ceases to be a legal gray area and becomes a medical service.
A Drug That Doesn't Exist in Nature
The draft law amends the federal law 'On Circulation of Medicines' and adds gene therapy drugs to the list of products not subject to mandatory registration. These are drugs made from the genetic material of a specific patient and used only in the medical organization where they were produced.
The document has already undergone public consultation, which lasted until May 15, 2026. The planned effective date is March 2028. Meanwhile, the government is expanding the list of clinics authorized to perform such work: by a directive dated February 19, 2026, Pirogov Russian National Research Medical University (RNRMU) and Bashkir State Medical University were added to the list.
This is not an abstract initiative. Behind it is a concrete, solid project. Since late 2024, Pirogov RNRMU has been building a separate plant for the production of gene therapy drugs worth 1.36 billion rubles. Construction is expected to be completed by the end of 2026, with production launch in 2027.
Three Months Instead of Ten Years
The classic route to market for a gene therapy drug is 7–10 years of clinical trials and a budget of hundreds of millions of dollars. But when a disease occurs in one in a million children, no business plan works out. Denis Rebrikov, Vice-Rector for Research at Pirogov RNRMU, explains bluntly: 'With a limited number of patients, it is difficult for pharmaceutical companies to recoup investments.'
The new approach compresses the timeline to three months. This is the development cycle planned at Pirogov University: after necessary safety tests and ethics committee approval, application can begin. Streamlined regulation is not the abolition of control, but the replacement of an unaffordable procedure with targeted expert review of the scientific validity of a specific intervention.
Technologically, there are two directions. The first is classic gene replacement therapy, where a working copy of the broken gene is delivered to the patient. The second is genome editing based on CRISPR/Cas editors, used when a mutation causes pathological protein activity rather than simply its absence. 'Roughly speaking, the enzyme goes crazy and starts ruining everything. Meanwhile, the second copy of the gene may be healthy. Adding another healthy copy is pointless—you need to fix the defect itself,' explains Rebrikov.
Who Is on the Front Line
What is happening now in the Russian orphan sector is the result of several parallel processes launched long before this draft law.
The first factor is the 'Circle of Kindness' foundation, established by presidential decree. In five years, it has provided innovative treatment to 30,000 children, and its list includes 133 drugs for 110 orphan diseases. The foundation already purchases unregistered gene therapy drugs, including Zolgensma for spinal muscular atrophy and Elevidys for Duchenne muscular dystrophy. The latest purchase price for Zolgensma was 93.5 million rubles per package.
The second factor is surgeons who know how to administer these drugs. In November 2024, neurosurgeons at the Russian Children's Clinical Hospital (RCCH) became the first in Russia to inject a gene drug into the brain of a child with aromatic L-amino acid decarboxylase deficiency. By June 2025, five children had undergone such surgery. Microinfusion technology using frameless stereotaxis allows precise delivery of the drug to subcortical brain structures—a procedure that five years ago would have been called science fiction.
The third factor is the emergence of domestic drugs. Biocad filed for registration of a gene therapy for hemophilia B in April 2025, and Generium began clinical trials of a gene replacement therapy for Duchenne muscular dystrophy. The Kulakov National Medical Research Center for Obstetrics and Perinatology also has its own gene therapy product, Alfacrigen, based on adeno-associated viruses.
Who Wins and Who Is Nervous
The most obvious beneficiaries are children with ultra-rare mutations for which no registered drug exists and never will due to purely economic reasons. In Russia, there are hundreds, if not thousands, of such patients: about 7,000 genes are known to be associated with monogenic diseases.
Federal medical centers also win. Pirogov RNRMU gains not just a production facility worth 1.36 billion rubles—it becomes the operator of a new medical model where the clinic simultaneously treats, produces, and develops. RCCH has already accumulated unique clinical experience found nowhere else in the world.
But there are also those who are tense. Experts demand 'maximally strict' quality control requirements. Yuri Zhulev, Co-Chairman of the All-Russian Union of Patients, puts the problem bluntly: 'Gene therapy is a complex intervention where unexpected body reactions and long-term consequences are possible.' Ekaterina Zakharova, Head of the Department of Molecular Mechanisms of Hereditary Metabolic Disorders at the Bochkov Medical Genetic Research Center, insists that before starting production, 'all consequences and risks in each case must be discussed very clearly and in detail with the professional community.'
These concerns are not unfounded. A drug made for a specific patient cannot, by definition, undergo full clinical trials on thousands of volunteers—they simply do not exist. The ethical review, which the Ministry of Health proposed making mandatory back in August 2025, becomes the only filter between scientific adventurism and justified risk.
What Will Change by 2028
The first scenario: the law is adopted, bylaws are prepared promptly, and by March 2028 the system is launched. By that time, Pirogov RNRMU will have been operating its plant for a year and applying drugs at two clinical bases—RCCH and the Veltischev Research and Clinical Institute for Pediatrics. Academic gene therapy ceases to be an experimental field and becomes a standard option for patients with monogenic diseases.
The second scenario, more likely given Russian specifics, is partial implementation. Ethical review will stall, standardizing the quality assessment procedure for 'bespoke' drugs will prove more difficult than it seemed on paper. But even in this case, several dozen children per year will receive therapy under expanded access protocols—as is already happening with CAR-T cell products.
The main question is price. Deputy Minister Evgenia Kotova estimated in December 2024 that the cost of producing one personalized gene therapy drug is about 1 million rubles. This is two orders of magnitude lower than the $3 million for a foreign drug like the one the Circle of Kindness foundation purchases for patients with decarboxylase deficiency. If Russian production costs are confirmed in practice, the economics of orphan therapy will be turned upside down: treating 'bespoke' mutations will become cheaper than purchasing registered Western drugs.
For families who have been knocking on the closed doors of pharmaceutical companies for years, the draft law means one thing: their child now has a route. Not a place on a waiting list, but a specific production cycle—from mutation sequencing to infusion—lasting three months. In a world where an ultra-rare disease was still synonymous with a death sentence, this is the definition of a breakthrough.
— Editorial Team
