Skyline Therapeutics Receives FDA Orphan Drug Designation for Gene Therapy for Retinitis Pigmentosa

BOSTON & SHANGHAI, September 2, 2024 – Skyline Therapeutics, a gene therapy company focused on innovative treatments for rare and severe diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108. SKG1108 is a novel, one-time gene therapy delivered intravitreally for the treatment of Retinitis Pigmentosa (RP), a progressive retinal disease that leads to vision loss.

The FDA’s designation recognizes the potential of SKG1108 in addressing an unmet need for patients with RP. The ODD status grants special benefits to SKG1108, such as tax credits, exemption from certain fees, and the possibility of seven years of market exclusivity upon approval, which could significantly accelerate the drug’s development and patient access.

SKG1108 utilizes a recombinant adeno-associated virus (rAAV) vector with an innovative design featuring the intravitreal capsid AAV.0106. This therapy delivers single-stranded DNA encoding light-activatable proteins directly to the retina. These proteins are intended to improve or restore visual function by generating new photo-sensing cells, compensating for the loss of rod and cone photoreceptors commonly seen in patients with advanced RP, regardless of the specific genetic mutations involved.

Retinitis Pigmentosa encompasses a group of inherited retinal diseases (IRDs) linked to mutations in over 100 genes, resulting in progressive vision loss. The condition typically begins with the degeneration of rod cells, followed by cone cells, leading to near-complete photoreceptor loss and blindness. Currently, no treatments effectively halt or reverse this degeneration. Existing gene-targeted therapies only address a small subset of RP patients. SKG1108 offers a broader therapeutic approach by aiming to improve visual perception and acuity independent of the underlying genetic defects.

About Orphan Drug Designation (ODD)

ODD is a status granted by the FDA to drugs or biological products that show promise in treating, preventing, or diagnosing rare diseases, with the goal of encouraging the development of treatments for conditions with significant unmet medical needs. Benefits of ODD include tax credits for clinical trials, exemption from prescription drug user fees, and potential seven-year market exclusivity after approval.

About Skyline Therapeutics

Skyline Therapeutics is a clinical-stage gene therapy company dedicated to developing novel solutions for rare and severe diseases. The company leverages an adeno-associated virus (AAV) platform with proprietary technologies in capsid discovery, transgene design, and vector engineering.

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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