The latest approval by the U.S. Food and Drug Administration for Luxturna, a new gene therapy to treat hereditary retinal degeneration, will prompt other gene therapies to commercialize, an analyst said.
“Starting with Luxturna, the gene therapies market will open,” said Seo Keun-hee, an analyst at KB Securities, in a recent report on global R&D trends in 2018.
Developed by Spark Therapeutics, a U.S. firm, Luxturna obtained the FDA approval in December, after testing the drug on patients with congenital blindness.
BioMarin’s hemophilia treatment BMN270 and Bluebird’s treatment Lenti-D for childhood cerebral adrenoleukodystrophy (CCALD) are on the phase-3 clinical trials.
Local biotech firms such as ViroMed, Kolon Life Sciences, and Sillajen are also actively developing gene therapies.
ViroMed is conducting clinical trials on VM202 pipelines, targeting diabetic neuropathy and ischemic chronic foot ulcers. Among them, a global phase-3 trial is underway to treat diabetic neuropathy and diabetic ischemic ulcers.
Also, Kolon Life Science’s novel drug Invossa, gene therapy for osteoarthritis and the 29th locally developed new drug, is going through a global phase-3 clinical trial to obtain approval of the FDA, despite its Japanese partner’s decision to rescind the technology import deal,
Sillajen’s genetically engineered oncolytic virus Pexa-vec, which drew attention in the combination therapy with cancer immunotherapy, also entered a global phase-3 clinical trial for patients with terminal liver cancer, and a trial on combo therapies to treat solid cancer and breast cancer.
“Gene therapies have merits that a single treatment can prolong its effectiveness,” Seo said. “This is why companies are focusing on one-time treatments for genetic disorders to ease burdens of repeated treatments for patients.”
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