The global medical community is abuzz with anticipation as the first gene-editing therapy nears potential FDA approval in December.
According to industry watchers, the gene-editing therapy Exa-cel, co-developed by CRISPR Therapeutics and Vertex Pharmaceuticals, is nearing potential FDA approval in December. Exa-cel, based on the CRISPR-Cas9 system, has been developed as a drug candidate for treating transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD).
Gene editing is a technology that selectively removes specific genes or changes the base sequence to cause positive mutations. The essence of this technology is to selectively remove disease-causing genes or neutralize viruses. The most representative gene-editing technology is the "CRISPR gene scissors," derived from cutting and pasting disease-causing DNA with scissors.
Amid the growing anticipation for the birth of the first gene-editing therapy globally, domestic companies such as ToolGen and GenKOre are also taking on the challenge of developing gene-editing therapies.
Notably, ToolGen is the only company in Asia with original patents for gene-scissor technology. ToolGen is developing treatments for wet macular degeneration and Charcot-Marie-Tooth (CMT) disease using gene-editing technology.
The development for a CMT treatment is ahead, with the company planning to enter phase 1 clinical trials in 2024.
On the other hand, GenKOre is focusing on developing treatments for three diseases—muscular, brain, and eye disorders.
Among the candidates, the ophthalmic disease treatment is progressing most rapidly, as the number of cells that need to be treated is more limited compared to brain and muscle disorders.
GenKOre's technological capabilities have also garnered interest from overseas pharmaceutical companies.
In January, the company signed a joint research agreement with a global pharmaceutical company to develop in-vivo gene therapies.
Although the contract does not disclose the company name and target disease, GenKOre has received an upfront payment and research funding, and upon successful commercialization, it is set to receive up to $350 million in options and milestones, as well as royalties on sales.
However, while these two companies are leading in developing gene-editing therapies in Korea, industry officials state that the situation in Korea is significantly behind compared to other advanced countries.
The main targets of gene-editing technologies are mostly rare disease treatments that are difficult to apply for insurance reimbursement after commercialization, and the market for these treatments is not large, presenting financial limits.
"Given the domestic situation, the likelihood seems low for high-cost drugs like gene-editing therapies to be covered by insurance, given that no approved treatments are available in Korea," an industry official told Korea Biomedical Review. "Also, the fact that the Ministry of Food and Drug Safety has not much experience in reviewing treatments using gene-editing technology can make things additionally difficult to develop such treatments."