The world’s first ‘gene-editing therapy’ has been approved, heralding a new era where diseases can be cured by editing the genes that cause them.
Vertex Pharmaceuticals and CRISPR Therapeutics announced on the 16th (local time) that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has conditionally approved ‘Casgevy’, a gene-editing therapy for sickle cell disease (SCD) and beta thalassemia (TDT). The company estimates that approximately 2,000 patients in the UK will be able to receive treatment through Casgevy.
With this, Casgevy has become the world’s first approved gene-editing therapy. The therapy, developed under the name ‘Exa-Cell’, is also under review by the U.S. Food and Drug Administration (FDA) for SCD indications, with a deadline of the 8th of next month. Reshma Kewalramani, CEO of Vertex, emphasized that “Today is a historic day in the field of medical science” and that “the approval of Casgevy is the world’s first approval of a CRISPR-based therapy”.
Casgevy is a ‘one-shot’ therapy that can cure the disease with a single dose. By extracting stem cells from the patient, editing the problematic genes using ‘CRISPR-Cas9’ technology, and then administering them back to the patient, these stem cells take root in the bone marrow and reconstitute the patient’s immune system. In fact, patients who participated in the clinical trials have maintained the effects of the drug for nearly three years after administration.
SCD is a fatal disease where the average age of death is 40, caused by anemia resulting from red blood cells, which should be round, becoming sickle-shaped and failing to properly transport oxygen. TDT is also a disease caused by a problem with beta globin in the red blood cells, with no cure other than stem cell donation, and patients typically die at an average age of 55. Both diseases had no cure other than bone marrow (hematopoietic stem cell) transplantation, but now they can also be treated with medication.
Casgevy is expected to become the most expensive drug in the world upon its release. The market currently estimates the launch price of Casgevy to be between $4 million and $6 million (approximately $4.4 million to $6.6 million), which far exceeds the $3.5 million (approximately $3.7 million) price tag of ‘Hemlibra’, the most expensive drug to date from CSL Behring for the treatment of Hemophilia B.
This approval is a breakthrough 12 years after the discussion on gene scissors technology began in earnest. Although there were first and second-generation technologies, the development of the third-generation technology, CRISPR-Cas9, in 2011 opened the floodgates for development. This is when Professor Emmanuelle Charpentier of the Max Planck Institute first announced its existence, and through joint research with Professor Jennifer Doudna of UC Berkeley, they successfully reproduced gene scissors in a test tube. Both of them jointly won the Nobel Prize in Chemistry in 2020.
With the emergence of the world’s first gene-editing therapy, the related market is expected to grow rapidly. The Korea Biotechnology Industry Organization predicts that the global gene-editing market, which is currently worth $5 billion (approximately $5.4 billion), will grow at an average annual rate of about 15% and double to $10 billion (approximately $10.8 billion) by 2028.
In addition to Vertex and CRISPR, Verve Therapeutics is also growing rapidly overseas, and interest from big pharma is increasing, as evidenced by Eli Lilly’s acquisition of a cardiovascular treatment being developed by Verve for up to $600 million in June. In Korea, ToolGen, founded by Professor Jin-Soo Kim, who succeeded in correcting human genes through gene scissors for the first time in the world in 2013, is developing ‘TGT-001’, a treatment for the rare disease Charcot-Marie-Tooth (CMT).
However, safety issues remain a stumbling block. The FDA Advisory Committee recommends tracking the status of patients for about 15 years even if Exa-Cell is marketed. Although the value of Exa-Cell’s efficacy is clear for diseases that previously had no treatment, the recommendation reflects concerns that gene-editing technology could cause unintended genetic changes.
Reporter Lee Chun-hee spring@asiae.co.kr
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