CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), the new gene-editing technology that claims to reduce blood cholesterol levels by more than half, has revolutionised the landscape of biomedical research and gene therapy. The good news is, it is expected to make an entry in the market in the next five years or so, according to Dr Sam Kulkarni, the CEO of CRISPR Therapeutics, the biotechnology company headquartered in Switzerland that is conducting the research. With the technology having been successfully administered to a small phase-1 trial group of four individuals, doctors and researchers are now working on making the therapy scalable and affordable.
For India, the therapy may prove particularly path-breaking, given the significant burden of cardiovascular issues and heart diseases prevailing in the country. While doctors are optimistic about the potential of CRISPR tech in Indian healthcare, they say that it can be seen in action perhaps five to ten years from now, with mass administration still being a few more years away. On November 19, the Union Ministry of Science and Technology launched BIRSA 101, India’s first CRISPR-based gene therapy for curing sickle cell disease, on the 150th anniversary of freedom fighter Birsa Munda. Now that the ball has already been set to rolling, inventions and advances in new genome therapies can also be expected, with the newly inaugurated research and translational facility, Council of Scientific and Industrial Research-Institute of Genomics and Integrative Biology (CSIR-IGIB).
A potential ‘game changer’
Doctors estimate that CRISPR technology in India is currently at its nascent stages, but can be expected to see fast-paced growth over the next few years in the country. “The government has approved frameworks for gene therapy and the ICMR (Indian Council of Medical Research) is working towards regulatory frameworks for safe and ethical trials,” says Dr Sagar Shah, consultant interventional cardiologist at Apollo Hospitals, Sheshadripuram, Bengaluru. However he adds, “But this requires a lot of capital and research funding which are the partly unmet needs currently.”
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Dr Shah goes on to explain the role of CRISPR gene therapy in reducing LDL cholesterol and triglycerides. “ANGPTL3 (angiopoietin-like protein) is a gene that provides instructions for making a protein which inhibits the enzyme lipoprotein lipase,” he explains. “A discovery in a specific population showed that individuals with a dysfunctional ANGPTL3 gene had significantly lower LDL and triglyceride levels, translating into protection against cardiovascular disease.”
“Based on this aforementioned discovery, researchers developed a CRISPR-Cas9 approach to disable the ANGPTL3 gene, with the goal of regulating cholesterol by turning this gene off,” he simplifies. This approach was tested in a phase 1 trial including four participants where it was proved to be both safe and successful. Dr Shah calls the possibility of a CRISPR-based solution a potential ‘game changer’ for India. “A single shot of the gene-editing treatment, which acts as a vaccine to permanently lower cholesterol, could be a ‘game-changer’ for certain high-risk and familial conditions that increase the cardiac issues. This addresses the root cause of cardiovascular disease and eliminates the risk due to elevated cholesterol levels,” he says, adding, “This approach is especially significant because one of the biggest challenges today is the non-adherence of daily medications.
Dr Prakhar Garg, principal consultant, internal medicine at Yatharth Super Speciality Hospital in Noida, too, is optimistic about CRISPR gene therapy in the country. “India has a very high burden of heart disease, so this area is important for us. With proper approvals, partnerships, and funding, Indian hospitals and research groups can run similar trials. But it must be done safely, with long follow-ups and strong monitoring,” he opines. He says that this treatment will be particularly useful for people suffering from high cholesterol or cardiac complications from genetic causes. “If CRISPR therapy becomes safe, affordable, and accessible, it can reduce long-term risks of heart attacks in such patients,” he adds.
However, Dr Garg also says that while CRISPR can significantly reduce risk, managing lifestyle and early screening will still matter the most. “It naturally cannot replace basic care like diet control, exercise, quitting smoking, regular checkups, and affordable medicines.” He adds, “CRISPR works at the gene level. It targets specific genes in the liver that control how the body makes or clears fats. By switching off or adjusting these genes, the liver starts to produce less ‘bad’ cholesterol and fewer triglycerides. This is why the drop in levels can be fast and strong in some patients,” he elaborates.
According to the Cardiological Society of India, almost 5 million Indians suffer from cholesterol imbalances. The CSI also holds that across the board, the first lipid profile screening of an individual should be done at 18 years without fail. In 2024, noting the unique lifestyles and diet patterns of Indians, the CSI released a set of lipid guidelines for India. This was a shift from the 2019 guidelines issued by the European Cardiological Society which are globally accepted, and were accepted by India too until 2024. A 2024 survey by health-tech company Healthians shows that 31% of Indians struggle with high cholesterol, with Kerala at the top of the list with a whopping 63% prevalence. Both Dr Garg and Dr Shah are in agreement on the massive future implications of CRISPR therapy in reducing the burden of cardiovascular issues and cholesterol imbalances in India.
A wait ahead
“Based on current progress, India could see the first CRISPR lipid-lowering trials or limited clinical use within five to ten years. This depends on successful global phase 2 and phase 3 data along with Indian regulatory support,” says Dr Shah. He says that for on-ground results to emerge and mass administration to be considered, a few more significant and possibly time-consuming hurdles are yet to be overcome. “Widespread clinical availability in major hospitals may take closer to ten to fifteen years, especially because long-term safety data and cost reductions are essential for adoption,” he adds. Mass availability of CRISPR-based treatment is still a far-sighted goal, Dr Garg agrees.
“We need more proof of long-term safety, lower treatment costs, trained staff, and strong regulatory systems. Before it reaches large numbers of patients, many years of research and development are still needed.” CRISPR Therapeutics’ Dr Kulkarni has gone on record in early November, when announcing the success of the phase 1 trial, saying, “The company plans to take its data to US regulators with the aim of starting phase 2 studies in 2026. We hope to have a product on the market in the next four or five years.”
Also, the first order of business is to target people with genetics-related high cholesterol, before it can eventually be appropriate for mass distribution. “We may see small clinical trials in India within the next few years. Wider use in hospitals will take longer because of cost, approvals, and training needs,” says Dr Garg, adding: “Once safety data becomes strong and the treatment becomes more affordable, we can expect gradual adoption. Overall, it is coming – but it will not happen overnight.”
Dr Shah says for the time being, CRISPR research in India has not advanced or progressed enough to be launched at a mass scale in India. The advantage with the CRISPR-Cas9 approach is that whenever it is rolled out in India, it will serve as a single-dose, long-lasting therapy. “CRISPR may eventually be the best option for patients who do not respond to standard therapies or who have severe genetic lipid disorders,” says Dr Shah.
Potential for other specialities
Apart from cholesterol imbalances and inherited heart disorders CRISPR technology can also be used in other specialities and complex treatments such as blood disorders like sickle cell anaemia, muscular degeneration, neurological conditions , as well as cancer treatment via CAR-T therapy which is already being used in India, informs Dr Shah. Dr Garg adds: “CRISPR will be most useful in conditions where one faulty gene is the main cause. This includes genetic cholesterol disorders, thalassemia, sickle cell disease, some liver diseases, certain eye conditions, and a few cancers. “
“This could work wonders in India, given the high burden of cardiovascular disease in India and the non-compliant nature of the Indian population,” predicts Dr Shah. “The current framework for regulating precision biotherapeutics is a path to future research and development of Indian products.”
BOX 1 – Existing treatments for high LDL cholesterol
- Statins
- Ezetimibe
- PCSK9 inhibitors
- siRNA therapies
- Lifestyle changes including managing diet, quitting smoking
BOX 2 – Top 5 states with high cholesterol prevalence
- Kerala
- Karnataka
- Telangana
- Maharashtra
- Punjab
