Alvin Luk, CEO of HuidaGene, is using his experience in big pharma to lead the small Shanghai and New Jersey-based biotech on a path towards creating one-time CRISPR gene-editing treatments for rare medical conditions.
In November 2024, the U.S. FDA cleared the company’s drug HG202, a treatment for a rare eye condition called neovascular age-related macular degeneration, for a phase 1 clinical trial to evaluate its safety and efficacy. The first few patients who received the treatment in clinical tests in China starting in 2023 have reported improvements in their vision and no adverse effects.
A month later, the company announced it had administered its treatment to the first patient in a Duchenne muscular dystrophy trial and also for its trial for a rare and fatal neurodevelopmental disorder called MECP2 duplication syndrome. Based on preliminary data presented in April, the 9-year-old boy with MECP2 duplication syndrome showed improvements in motor and social skills after treatment, though many hurdles remain before full approval.