Scienza e Tecnologia
HUIDAGENE THERAPEUTICS RECEIVES FDA RARE PEDIATRIC DISEASE DESIGNATION FOR HG004 TO TREAT INHERITED BLINDNESS
"We are pleased to have received another significant regulatory feedback from the US FDA underscoring the dire need for a treatment option of this devastating inherited blindness," said Linyu Shi , Ph.D., Co-founder and Chief Scientific Officer of HuidaGene. "Data from our preclinical studies have shown that HG004 demonstrates significant superiority than AAV2- (LUXTURNA like) in the recovery of retinal function of the mice. We are committed to delivering transformative genetic medicines for rare genetic diseases globally."
Inherited retinal disease (IRD) is a group of rare blinding conditions caused by mutations in any 1 of more than 250 genes. Leber's congenital amaurosis (LCA), severe early childhood-onset retinal dystrophy (SECORD), early-onset severe retinal dystrophy (EOSRD), and retinitis pigmentosa (RP), which may all be grouped under the heading of inherited retinal disease caused by
mutations ( -IRD), are considered to represent a phenotypic continuum of the same disease. The
-IRD with a typical onset between birth and five years of age exhibits several common clinical findings, chiefly night blindness, progressive loss of visual fields, and loss of central vision. Given the often severe and early visual loss associated with IRD, other areas of development, including speech, social skills, and behavior, may also be delayed.
HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in
Shanghai and New Jersey , HuidaGene is committed to addressing patients' needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. We are currently advancing clinical programs in inherited retinal disease caused by
mutations and our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy, and MECP2 duplication syndrome. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine.
For more information, please visit http://www.huidagene.com
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