Is the era of gene therapy coming? Development trend of data inventory in recent 10 years

Earlier this month, Bluebird bio's gene therapy, zynteglo, was approved by the European Commission for the treatment of transfusion dependent beta thalassemia In mid May, zolgensma, a heavyweight gene therapy from Novartis, was approved by the US FDA to treat patients with spinal muscular atrophy (SMA) A number of large pharmaceutical companies are also in the form of M & A or R & D cooperation to layout the field of gene therapy There are various signs that the field of gene therapy has not only recovered after 20 years of setbacks, but also is entering a new era of vigorous development Recently, dealforma has made statistics on R & D cooperation, M & A, R & D authorization agreement and other investment and financing activities in this field in the past decade Today, the content team of Wuxi apptec will share insights from these data with readers With the rapid development of gene therapy, dealforma broke out in 2018, which made statistics on the number of transactions and cooperation in gene therapy in the past decade, including research and development cooperation carried out by non-profit institutions such as universities / foundations, research funds issued, and start-ups established according to academic research; research and development cooperation reached by the pharmaceutical industry, and M & A events This statistic focuses on gene therapy and vector technology for gene therapy Statistical data show that since 2008, the field of gene therapy has started to develop steadily, and the development speed has accelerated since 2013, and reached a leap in 2018 In 2019, it still maintains a strong momentum Since 2008, the number of cooperation in the field of gene therapy (data source: dealforma, content team mapping of Wuxi apptec) has been in the mainstream in the early stage of research and development Neurology, ophthalmology, hematology and cancer are concerned These activities are divided according to the field of disease It can be seen that neurology, ophthalmology and cancer are the three most active fields of gene therapy On the one hand, there is a great demand in these fields On the other hand, gene therapy may be more suitable for treating these diseases For example, many hereditary ophthalmic diseases are caused by single gene mutation, and the eyes are anatomically isolated organs, so the risk of using gene therapy is low This makes hereditary ophthalmic diseases one of the most suitable diseases for gene therapy Since 2008, the classification of cooperation projects in the field of gene therapy (according to the field of treatment, data source: dealforma, content team drawing of Wuxi apptec) Hematology Diseases, especially hemophilia, sickle cell anemia, and β thalassemia, has been another hot spot of gene therapy These diseases usually require patients to receive regular treatment for life, and gene therapy may make them achieve "curative" effect after one treatment Most of these collaborations occur at the patent and technology platform stage Even in the pharmaceutical industry, most of the cooperation takes place in the stage of technology platform development of gene therapy Classification of cooperation projects in the field of gene therapy since 2008 (by clinical stage field, data source: dealforma, content team mapping of Wuxi apptec) support of venture capital to gene therapy The development of gene therapy needs the help of a lot of funds Venture capital is an important source of funds for gene therapy development From this point of view, we can also indirectly see the industry's assessment of the potential of gene therapy Since 2010, dealforma has tracked 120 seed rounds and venture capital rounds in the field of gene therapy, with a total investment of US $5 billion More than $3 billion of that went to companies with only platform technology and preclinical products It is worth noting that in the past 18 months, the amount of financing reached US $2.5 billion, which is another indication of the outbreak of gene therapy in the last two years Since 2010, the amount of venture capital financing in the field of gene therapy (million US dollars, data source: dealforma, content team mapping of Wuxi apptec) in 2019, although less than half of it has just passed, the amount of venture capital in the field of gene therapy is close to the sum of 2018 We hope that with the help of capital, the innovative treatment mode of gene therapy can continue to flourish, providing patients with refractory diseases with real "healing" therapy, so that they can "benefit once and for all" Appendix: major events of gene therapy (incomplete list) approved: on June 4, 2019, zynteglo of Bluebird bio was approved by the European Union as the first gene therapy for β thalassemia On May 25, 2019, zolgensma of Novartis was approved by the US FDA as the first gene therapy for SMA On December 20, 2017, the spark therapeutics gene therapy luxturna was approved by the US FDA to treat hereditary retinal dystrophy This is the first in vivo gene therapy for genetic diseases Update of regulatory measures: on January 15, 2019, Dr Scott Gottlieb, former director of the FDA, and Dr Peter marks, director of the center for biological product evaluation and research jointly issued a statement It is expected that the FDA will approve 10-20 gene therapy and cell therapy every year by 2025 FDA will employ at least 50 new clinical reviewers to evaluate cell and gene therapy On August 15, 2018, NIH and FDA jointly issued a statement, marking a new stage of human gene therapy regulation Large pharmaceutical companies arrange gene therapy: on March 21, 2019, Pfizer announced a US $630 million R & D cooperation with vivet therapeutics, which develops gene therapy for rare diseases, and reserves the right to acquire vivet On March 4, 2019, Biogen announced that it plans to acquire nightstar therapeutics, a company developing gene therapy for ophthalmic diseases, for an amount of 800 million US dollars On February 26, 2019, Roche announced that it wanted to buy gene therapy star spark therapeutics for $4.3 billion On April 10, 2018, Novartis acquired avexis for $8.7 billion Avxs-101, developed by the company, became the recently approved zolgensma Reference: [1] gene therapy R & D deals turn red hot as big pharma steps up to play https://endpts.com/gene-therapy-rd-deals-turn-red-hot-as-big-pharma-steps-up-to-play/