07.08.2023 – 17:10
Huidagene Therapeutics
SHANGHAI AND CLINTON NJ, April 20 /PRNewswire/
HG004 is a one-time, direct-to-RPE treatment for inherited retinal disorders caused by mutations in the RPE65 gene~10-fold lower vector doses in the multi-region, multi-center HG00402 study than other AAV2 gene therapy clinical studies HG004 previously went orphan Drug Designation (ODD) for RPE65-IRD granted on March 31, 2023
Huidagene Therapeutics (辉大基因，”HuidaGene”), a clinical-stage biotechnology company focused on the development of CRISPR-based programmable genomic drugs, announced today that the US Food and Drug Administration (FDA) has granted HG004 designation as a Rare Pediatric Disease Designation (RPDD) for the treatment of inherited retinal diseases caused by RPE65 mutations (RPE65-IRDs).The FDA grants RPDD to encourage the development of new treatments for severe or to create life-threatening diseases that primarily affect children ages 18 and younger and affect fewer than 200,000 people in the U.S. Under the RPDD program, a sponsor who receives approval can apply for a Priority Review Voucher (PRV), which serves as an accelerated six-month priority review for any subsequent application to commercialize or may be sold or transferred. Historically, PRVs have sold for over $100 million, with the highest price paid for a PRV being $350 million. For a large company bringing a multi-billion dollar drug to market, the six-month speedup in regulatory review can be of significant commercial value. Most recently, Sarepta Therapeutics sold a PRV for $102 million.
“This US FDA pediatric rare disease designation underscores the significant unmet medical need that HuidaGene is seeking to address with HG004 for RPE65 IRDs,” said Alvin Luk, Ph.D., MBA, CCRA, co-founder and chief executive officer of HuidaGene “HG004 received both RPDD and ODD (Orphan Drug Designation) status from the US FDA in March, allowing for clinical development tax credits, waivers of certain FDA filing fees, seven-year market exclusivity, and assistance in the drug development process. We.” We look forward to advancing HG004 in the clinic and remain true to our goal of helping patients worldwide.”
“We are pleased to have received further important regulatory feedback from the US FDA, underscoring the urgent need for a treatment option for this devastating inherited blindness,” said Dr. Linyu Shi, Co-Founder and Chief Scientific Officer of HuidaGene. “Data from our preclinical studies have shown that HG004 demonstrates clear superiority over AAV2-hRPE65 (like LUXTURNA) in restoring retinal function in Rpe65-/- mice. We are committed to providing innovative genetic medicines for rare genetic diseases worldwide.”
Information on hereditary retinal diseases caused by RPE65 mutations
Inherited retinal diseases (IRDs) are a group of rare blinding diseases caused by mutations in 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), all included under the term of by RPE65 mutations causing hereditary retinal disease (RPE65-IRD) are considered as a phenotypic continuum of the same disease. RPE65-IRD, which typically occurs between birth and five years of age, has several common clinical findings, most notably night blindness, progressive visual field defects, and loss of central vision. Given the often severe and early vision loss associated with RPE65-IRD, other areas of development, such as language, social skills, and behavior, may also be delayed.
Informationen zu HuidaGene – Huida Gene
HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focused on the discovery, engineering and development of CRISPR-based genetic medicines to reshape the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene aims to serve the needs of patients worldwide with various preclinical therapy programs in the fields of ophthalmology, otology, myology and neurology. We are currently advancing clinical programs in inherited retinal diseases caused by RPE65 mutations, as well as our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy and MECP2 duplication syndrome. The company’s CRISPR-based therapeutics offer the potential to heal patients with life-threatening diseases by addressing the cause of the disease. HuidaGene aims to change the future of genome editing medicine.
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