BOSTON and SHANGHAI, Sept. 3, 2024 /PRNewswire/ -- Skyline Therapeutics, an innovation-driven gene therapy company committed to developing unique and novel solutions for rare and severe diseases, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108, a novel one-time intravitreally delivered gene therapy for the treatment of Retinitis Pigmentosa (RP). This designation reflects the recognition by the FDA of the potential of SKG1108 for treating RP. The ODD grants special status to SKG1108 and qualifies it for various benefits, which will greatly accelerate the development of the drug and enhance patient access to the leading-edge therapeutic solution.
SKG1108 is a recombinant adeno-associated virus (rAAV) vector with innovative design, utilizing the novel intravitreal capsid AAV.0106 to deliver single-stranded DNA encoding light-activatable proteins directly to the retina. The protein, regulated by specific genetic elements, aims to improve or restore visual function by generating new photo-sensing cells, thereby compensating for the loss of rod and cone photoreceptors in patients with late-stage RP, agnostic on the specific gene or genetic mutation responsible for the condition.
RP is a type of inherited retinal diseases (IRDs) linked to mutations in over 100 different genes or loci, all leading to progressive vision loss. While the age of onset is varied, most RP patients are classified as legally blind by their 40s. The disease typically begins with the degeneration of rod cells, followed by the gradual loss of cone cells, and ultimately leads to the near-complete loss of all photoreceptor cells, leaving the retina non-functional and resulting in blindness. Currently, there are no effective treatments worldwide that can halt or reverse photoreceptor degeneration. Existing therapies targeting specific mutant genes apply only to a small subset of RP patients. SKG1108, an innovative gene therapy that employs a novel approach to improve or restore patients' visual perception and acuity independent of the underlying genetic defects, holds great potential to benefit a broader range of RP patients.
About Orphan Drug Designation (ODD)
Orphan Drug Designation (ODD) is a status granted by the FDA to drugs or biological products that show promise in treating, preventing, or diagnosing rare diseases. The goal of granting ODD is to encourage innovation and research in the development of treatments for rare diseases and to make these treatments available more quickly. ODD qualifies drug sponsors for incentives including tax credits for qualified clinical trials, exemption from the prescription drug user fees, and potential seven years of market exclusivity after approval.
About Skyline Therapeutics
Skyline Therapeutics is an innovation-driven, clinical-stage gene therapy company focused on developing unique and novel solutions to address unmet needs in rare and severe diseases. Leveraging our cutting-edge adeno-associated virus (AAV) platform, which includes multiple proprietary technologies for capsid discovery, transgene design, and vector engineering, as well as robust in-house process development and GMP manufacturing, we are advancing a diverse pipeline of gene therapies for ocular, neurological, and cardiovascular disorders. Our lead programs are rapidly progressing through clinical trials with regulatory approvals and special designations from the US FDA and China CDE, bringing us closer to providing life-changing therapies to patients in need. Skyline Therapeutics has a global presence for research, development, regulatory, and manufacturing in Shanghai and Hangzhou, China, and Boston, MA, USA. www.skytx.com
References |
1. Daiger, S. P., Sullivan, L. S., & Bowne, S. J. (2013). Genes and mutations causing retinitis pigmentosa. Clinical Genetics, 84(2), 132–141. |
2. O'Neal, T. B., & Luther, E. E. (2018). Retinitis pigmentosa. |
3. Hamel, C. (2006). Retinitis pigmentosa. Orphanet Journal of Rare Diseases, 1(1). |
4. Heckenlively, J. R. (1987). RP cone-rod degeneration. Transactions of the American Ophthalmological Society, 85, 438–470. |
5. Dalkara, D., Goureau, O., Marazova, K., & Sahel, J.-A. (2016). Let There Be Light: Gene and Cell Therapy for Blindness. Human Gene Therapy, 27(2), 134–147. |
6. Cross N, van Steen C, Zegaoui Y, Satherley A, Angelillo L. Retinitis Pigmentosa: Burden of Disease and Current Unmet Needs. Clin Ophthalmol. 2022 Jun 20;16:1993-2010. |
