February 24, 2024

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GSK abandons investment in gene and cell therapy and transfer to small nucleic acid drugs

GSK abandons investment in gene and cell therapy and transfer to small nucleic acid drugs


GSK abandons investment in gene and cell therapy and transfer to small nucleic acid drugs

Recently, Emma Walmsley , CEO of GlaxoSmithKline (GSK) , emphasized when summarizing GSK’s vision to investors that GSK has decided to end its investment in cell and gene therapy .

This is also a month after GSK canceled its cell therapy cooperation with Lycell and Immatics, it once again released a clear signal: GSK’s drug development direction is going against the trend of the industry.

GSK believes that instead of rushing to push “off-the-shelf” CAR-T to the clinic, or fully investing in the strategy of cell therapy for solid tumors , it is better to rely on its expertise in the field of genetics to achieve market success more smoothly.


Medical Dialogues GSK cancer drug JEMPERLI



About 70% of GSK’s entire product portfolio now has a strong human genetics base, according to Dr John Lepore , head of research and development at GSK .

Theoretically, a genetically validated target is at least twice as likely to end up being a drug. Although this is not a panacea, but based on GSK’s large investment in human genetics, functional genomics and artificial intelligence, if it can double the probability of drug success, it is of great importance to GSK itself and the entire industry, and GSK Significant progress has been made in this regard.


It sounds simple enough, but not every big pharma company is daring to bet on genetic targets. That’s because new potential targets are identified through genetics, and half of them cannot be targeted with small molecules or antibodies. Towards.

And that’s where oligonucleotides , short strands of artificially synthesized DNA or RNA, come in to reduce, restore, or regulate RNA through several different mechanisms .


Biotech companies such as Alynlam and Ionis are early pioneers in this field. The former has a market value of more than 27 billion U.S. dollars, and the latter has a market value of more than 5 billion U.S. dollars.

They have several siRNA or ASO drugs on the market, respectively. Today, however, the oligonucleotide platform is evolving faster than the two companies originally envisioned.


It is precisely because of its optimism about oligonucleotide technology that GSK invested US$170 million in cooperation with Wave Life Sciences in December last year .

Wave is the only company with an oligonucleotide platform for the three RNA-targeting modes of editing, splicing and silencing.

All three modes of RNA targeting are of interest to GSK when investigating genetics-based drug targets. Another point that Wave attracts GSK is that Wave’s delivery technology can target organs and tissues other than the liver (such as kidney and lung) , which is expected to solve the current extrahepatic targeting problem.


The cooperation between GSK and Wave involves up to 8 joint R&D pipelines, the most important of which is WVE-006 , a small nucleic acid drug in the preclinical research stage, which restores normal α1-antipancreatin by targeting disease-causing RNA Expression of proteases for the treatment of alpha 1-antitrypsin deficiency .

α1-antitrypsin deficiency is an inherited metabolic disease caused by α1-antitrypsin deficiency, which can lead to neonatal hepatitis, liver cirrhosis, liver cancer, and emphysema in infants and adults.


While exciting, WVE-006 is still late in GSK’s pipeline of small nucleic acid drugs. An antisense oligonucleotide (ASO) drug bepirovirsen developed by GSK and Ionis in cooperation is much ahead. Bepirovirsen is currently undergoing two phase 3 clinical trials and is expected to achieve a functional cure for hepatitis B.


Nucleosides/nucleoside analogues are the first-line treatment drugs for hepatitis B patients, they can inhibit the replication of hepatitis B virus. However, the hepatitis B virus cannot be eliminated, so the medicine must be continued for life.

The unique design of bepirovirsen can reduce the replication of hepatitis B virus and inhibit the production of HBsAg, so as to achieve a functional cure and free patients from life-long medication.

In June 2022, GSK announced the interim data of the phase 2b clinical trial of bepirovirsen at the International Liver Congress 2022. The data showed that after 24 weeks of treatment, nearly 30% of hepatitis B patients had undetectable levels of hepatitis B virus in their bodies. These data were subsequently published in the New England Journal of Medicine (NEJM) .


GSK abandons investment in gene and cell therapy and transfer to small nucleic acid drugs



Oligonucleotide technology has existed for decades, but more than a dozen drugs approved for marketing at this stage are all concentrated in the field of rare genetic diseases. And bepirovirsen is currently one of the leading small nucleic acid drugs for large common diseases, and the success of bepirovirsen will greatly promote the entry of small nucleic acid drugs into a wider range of diseases.

In addition, GSK4532990, a small nucleic acid drug jointly developed by GSK and Arrowhead , is about to enter Phase 2 clinical trials for the treatment of chronic nonalcoholic steatohepatitis (NASH) .



In fact, betting heavily on small nucleic acid drugs is not the only move against the trend of drug research and development by GSK. They are still vigorously developing new antibiotics .

In November 2022, GSK announced positive results for two new antibiotics for the treatment of tuberculosis and urinary tract infections, the latter of which will be submitted to the FDA this year.


Despite the growing problem of antibiotic resistance, the return on investment in developing antibiotics has been too low for decades .

There is a “double-ten law” in the field of new drug research and development (the average cost of an innovative drug from research and development to the market is more than 1 billion US dollars, and the research and development cycle is more than 10 years) , while the research and development of antibiotics requires an average of 1.6 billion US dollars.

More importantly, the long development process has led to the emergence of antibiotic resistance in bacteria before large-scale application of some antibiotics after they have been approved for marketing.

This has led even big pharmaceutical companies to stay away from developing antibiotics. But GSK believes that this is an area that absolutely needs someone to continue to develop, and antibiotic research and development cannot be allowed to disappear.




GSK executives believe it was the right decision to abandon the hot research field of cell and gene therapy.

While cell therapies such as CAR-T have had significant success in treating hematologic malignancies, the field is now too crowded, and the prospect of extending it to solid tumors is more challenging than we originally thought.

Therefore, continuing to invest in gene and cell therapy is not the best option for GSK, given all the development and manufacturing costs and risks.




References :

GSK abandons investment in gene and cell therapy and transfer to small nucleic acid drugs

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