Roche's $3 billion increase, what breakthroughs are needed for gene therapy?

August 28, 2021

On August 24, Shape Therapeutics (ShapeTX) announced a cooperation agreement with Roche, under which the two parties will jointly develop gene therapies for certain targets in Alzheimer's disease, Parkinson's disease and rare diseases. Shape may receive more than $3 billion in upfront and milestone payments. On the same day, Vertex Pharmaceuticals and Arbor Biotechnologies announced that they will launch a new collaboration to use Arbor’s proprietary CRISPR gene editing technology to enhance the development of innovative cell therapies for the treatment of serious diseases. Arbor was co-founded by renowned scholar Dr. Feng Zhang. According to the terms of the agreement, Arbor will receive an advance payment and is eligible for potential milestone payments of up to US$1.2 billion.

The idea of gene therapy appeared as early as 1972. After nearly half a century, gene therapy has changed from a concept to a reality. In recent years, it has become more and more popular, the IPO market is booming, and it is also sought after by big pharma, with frequent mergers and acquisitions. According to Froster Sullivan, a well-known consulting agency, the global gene therapy market will reach 30.54 billion U.S. dollars by 2025, of which the Chinese market will reach 17.89 billion U.S. dollars. However, there are still many problems that need to be solved in gene therapy, such as safety and expensive treatment costs.

What is gene therapy?

According to the definition of "General Theory of Gene Therapy Products for Human Use" issued by the National Pharmacopoeia Commission, gene therapy products usually consist of vectors or delivery systems containing engineered gene constructs, and their active ingredients can be DNA, RNA, genetically modified viruses, Bacteria or cells can replace, compensate, block, and modify specific genes by introducing foreign genes into target cells or tissues to achieve the purpose of treating diseases. Gene therapy products are composed of two parts: vector and gene. The choice of vector will affect the genes that can be loaded and delivered. Commonly used vectors include adenovirus, adeno-associated virus (AAV), retrovirus, lentivirus and oncolytic virus. Different gene selection can achieve different therapeutic goals. According to the effect on genes in the body, there are three types: enhancement, inhibition and correction. In 1990, the United States approved the first gene therapy clinical trial, and so far more than 2,000 gene therapies worldwide have entered clinical trials. After nearly half a century, gene therapy has gone from concept to reality. Up to now, more than 20 gene therapy drugs have been approved for the market worldwide for the treatment of cancer, viral infections, genetic diseases and so on. In 2017, the US FDA approved the first in-person gene therapy Luxturna to treat children and adult patients with vision loss caused by pure and mutation RPE65 gene but retain a sufficient number of viable retinal cells, thereby restoring and improving their vision. The product is developed by Spark Therapeutics (the company has been acquired by Novartis), which is the first true gene therapy in the US market and also marks the official arrival of the era of gene therapy. In China, in January this year, Boya Gene announced that the Center for Drug Evaluation of the National Medical Products Administration of China had approved its clinical trial application for the CRISPR/Cas9 gene editing therapy product ET-01 for blood transfusion-dependent β-thalassemia. This is the first gene editing therapy product and hematopoietic stem cell product approved by the State Food and Drug Administration for clinical trials in China. In August, BBM-H901 injection (AAV gene therapy drug for preventing bleeding in adult male patients with hemophilia B) independently developed by Shanghai Xinzhi Pharmaceutical Technology Co., Ltd., a wholly-owned subsidiary of Faith Pharmaceuticals, was approved by the National Food and Drug Administration. Test approval. This is the first AAV gene therapy drug for hemophilia approved to enter clinical trials in China, and it is also the first gene therapy drug for rare diseases administered systemically in China.

The durability of gene therapy has been questioned, what are the ways to optimize it in the future?

At present, most gene therapy strategies are to use modified viruses as delivery vehicles, of which AAV vectors are the most common. However, because the genome of the AAV vector cannot be integrated into the human genome, the delivered therapeutic gene may be diluted with cell division, death or other reasons, making the therapeutic effect unsustainable. According to the GENEr8-1 data released by BioMarin Pharmaceuticals in January this year, we can see that in subjects who received valoctocogene roxaparvovec for at least 2 years, the endogenous FVIII expression level increased from 42.2 (SD 50.9, median at the end of the first year). Value 23.9) IU/dL decreased to 24.4 (SD 29.2, median value 14.7) IU/dL at the end of the second year, showing continuous hemostatic effect, ABR of 0.9 (median value 0.0) times. Although the level of factor VIII in the patient's body is declining, it is still within the effective range. In July, valrox received a marketing authorization application (MAA) from the European Medicines Agency (EMA) for use in adult patients with severe hemophilia A. It is expected to issue CHMP opinions in the first half of 2022. Dr. Jiang Ruhong, the founder and CEO of ASC Therapeutics in the United States, said that there are about three ways to optimize gene therapy: 1. Improve the transfection rate of viral vectors; 2. Build better genes for higher expression; 3. More protein after expression Mostly secreted from the cell. In addition, in vivo gene editing technology is also a direction to solve the durability problem.

How much room is there for price reduction in the future for gene therapy’s “sky price” treatment costs?

Dr. Jiang Ruhong said that the price of treatment drugs takes into account all costs from hair growth to production. First of all, the platform is very expensive to build, it takes a long time, and the first product is definitely expensive. But in the future, if you continue to use this platform to develop products, the price will come down. Maybe in the next 5 years or so, it may drop to about $100,000. Dr. Sun Xiulian, chairman and chief scientist of Immune Cell, said that in fact, the production of gene therapy products is not high. But for drugs, regardless of the production cost, the cost is more clinical costs. As a new technology product, gene therapy is reasonably expensive. However, with the advancement of technology and platform-based R&D and production, it is believed that it is entirely possible to achieve affordable treatment in the future.

Concluding remarks

Gene therapy was first developed to treat hereditary genetic diseases. After positive results have been achieved, gene therapy has developed faster and faster. The market size of the gene therapy industry is growing rapidly, and large pharmaceutical companies such as Roche and Novartis have deployed. . In China, a large number of excellent local gene therapy companies and scientific research institutions have emerged in the field of gene therapy. Many indications have been deployed, including hemophilia A/B, β-thalassemia, autoimmune deficiency diseases, and various Solid tumors and so on. I believe that with the efforts of many gene therapy companies, the spring of gene therapy will usher in.

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