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news.Skyline Therapeutics has announced that the US Food and Drug Administration (FDA) granted orphan drug designation (ODD) to its gene therapy, SKG1108, for the treatment of Retinitis Pigmentosa (RP), an inherited retinal disease.
Retinitis Pigmentosa is an inherited retinal disease linked to mutations in over one hundred different genes or loci, leading to progressive vision loss. Credit: Marina Vitale on Unsplash.
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SKG1108 is a one-time intravitreally delivered therapy, which uses a recombinant adeno-associated virus (rAAV) vector to target the condition.
It employs the AAV.0106 capsid to deliver a single-stranded DNA that encodes light-activatable proteins directly to the retina.
These proteins, which are regulated by particular genetic elements, are designed to create new photo-sensing cells, potentially improving or restoring visual function.
This approach aims to compensate for the loss of rod and cone photoreceptors in late-stage RP patients, regardless of the specific gene or genetic mutation that is responsible for the disease.
The ODD offers various benefits to SKG1108, potentially accelerating its development and enhancing patient access to this therapeutic solution.
The US regulator grants ODD to drugs or biological products that demonstrate potential in treating, preventing, or diagnosing rare ailments.
It qualifies drug sponsors for incentives such as tax credits for clinical trials, exemption from prescription drug user fees, and possibly seven years of market exclusivity post-approval.
RP is linked to mutations in over one hundred different genes or loci, leading to progressive vision loss.
Utilising its AAV platform, which includes technologies for transgene design, capsid discovery, and vector engineering, along with in-house process development and GMP manufacturing capabilities, the company is progressing a diverse pipeline of gene therapies for neurological, ocular and cardiovascular disorders.
Its lead programmes are advancing through clinical studies and have received regulatory approvals and special designations from the and China’s Center for Drug Evaluation (CDE) and the US FDA.
