RPE65

Retinitis pigmentosa can eventually lead to complete blindness. Credit: GBJSTOCK via Shutterstock. Gene therapy company Skyline Therapeutics has secured an orphan drug designation from the US Food and Drug Administration (FDA) for its retinitis pigmentosa gene therapy, named SKG1108. There are currently no approved treatments that can halt or reverse photoreceptor degeneration in retinitis pigmentosa, a group of inherited eye disorders that cause progressive loss of vision due to the degeneration of the retina, specifically affecting the photoreceptor cells. This typically begins with night blindness and loss of peripheral vision and can eventually lead to complete blindness. SKG1108 uses a recombinant adeno-associated virus to deliver DNA directly to the retina. This DNA encodes light-sensitive proteins that can help restore vision by creating new photo-sensing cells, which replace the lost rod and cone cells in patients with advanced retinitis pigmentosa. Skyline said the treatment is effective regardless of the specific gene mutation causing the condition. Existing therapies for this rare disease mainly target specific mutant genes that apply only to a small subset of patients. The only approved retinitis pigmentosa therapy on the market is Spark Therapeutics ’ Luxturna (voretigene neparvovec), a retinoid isomerohydrolase activator, indicated for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. See Also: BR-6002 by Boryung Pharmaceutical for Duodenal Ulcer: Likelihood of Approval BR-6002 by Boryung Pharmaceutical for Gastric Ulcers: Likelihood of Approval One of the main players in this field is US-based Beacon Therapeutics. Its lead asset, AGTC-501 (laruparetigene zovaparvovec) is currently in Phase II/III of development for X-linked retinitis pigmentosa (XLRP) in the VISTA trial (NCT04850118) and has shown good efficacy in previous clinical trials. AGTC-501 is highly anticipated as it has the potential to become the first gene therapy specifically indicated for XLRP, according to a GlobalData analyst . Initial data with AGTC-501 showed an improvement in microperimetry measurements, which was also seen in patients who received the highest dose of the therapy in a Phase II study. In July 2024, Beacon secured $170m in a Series B funding round to advance AGTC-501. China-based Skyline has other ocular disease treatments under its belt, including the gene therapy SKG0106 for neovascular age-related macular degeneration (nAMD). The one-time intravitreally delivered candidate is being evaluated in a Phase I/II clinical trial (NCT05986864) in patients with nAMD. The nAMD market is expected to reach $27.5bn across the seven major markets (US, UK, France, Germany, Italy, Spain, and Japan) by 2031, according to a GlobalData report . GlobalData is the parent company of Pharmaceutical Technology. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

– Dr. Wright has three decades of experience in the research and development of viral vectors and has been involved in the development of multiple approved gene therapies globally – PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., July 08, 2024 (GLOBE NEWSWIRE) -- Kriya Therapeutics, Inc. ("Kriya"), a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world, today announced the appointment of J. Fraser Wright, Ph.D., to the position of Chief Gene Therapy Officer. Dr. Wright is a Scientific Co-Founder of Kriya and has served as a senior advisor to the company since its founding in 2019. During his career he has contributed to numerous gene therapy development programs, including those leading to the regulatory approvals of multiple FDA approved commercial products: Luxturna® for RPE65-/- Leber congenital amarosis Type 2, the first gene therapy for a genetic disease; Kymriah®, the first CAR T-cell therapy for lymphoma and leukemia indications; and Beqvez™ for the treatment of hemophilia B. Most recently, Dr. Wright was a Professor of Pediatrics at the Stanford University School of Medicine and Director of Technology Innovation at Stanford’s Center for Definitive and Curative Medicine. His research aimed to advance the design, characterization and production of AAV-based medicines for rare and common diseases, including addressing key immunological barriers to the successful translation of AAV gene therapies. “Fraser has been instrumental in advancing the field of gene therapy, including through his contributions to the approval and commercialization of multiple gene therapy products globally,” said Shankar Ramaswamy, M.D., Co-Founder and CEO of Kriya. “Since helping to launch Kriya in 2019, Fraser has played a crucial role in accelerating our efforts by bringing his expertise in immunology, vector biology and CMC to the development of our gene therapy pipeline for common diseases. We are thrilled to have Fraser join our leadership team as Chief Gene Therapy Officer as we prepare to advance multiple gene therapies into the clinic for highly prevalent diseases.” Dr. Wright added, "Over the past decades, the field of gene therapy has evolved from an exciting concept facing many clinical translational challenges, to the current landscape of multiple successful regulatory approvals and commercialization of products that provide definitive therapeutic benefit. I am privileged to have been deeply immersed in the gene therapy revolution, and I am optimistic about the future of gene therapy as a new paradigm of therapeutics across multiple fields of medicine. I am impressed that Kriya has built the critical infrastructure and expertise needed to be a fully integrated gene therapy company with collective dedication, innovation and pursuit of excellence to bring better medicines to patients in need. I look forward to the opportunity provided by this leadership role to help address serious healthcare challenges facing society today.” Dr. Wright began his industry career as a Scientist at Sanofi Pasteur, contributing to the development of viral vector-based vaccines and cancer immunotherapies, and subsequently as Director of Development and Clinical Manufacturing at Avigen, a gene therapy company that pioneered AAV-based investigational gene therapies for hemophilia and Parkinson’s disease. After Avigen, Dr. Wright returned to academia where he established and directed the Clinical Vector Facility at the Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia and gained a faculty appointment at the University of Pennsylvania Perelman School of Medicine. Dr. Wright is also a Co-Founder and the founding Chief Technology Officer of Spark Therapeutics. He is an author of over 100 peer reviewed publications and an inventor of many patents used widely in human gene therapy. Dr. Wright received his Ph.D. (Biochemistry and Molecular Immunology) and B.Sc. (Biology and Physiology) from the University of Toronto, and completed post-doctoral studies at INSERM/CENG Grenoble, France. About Kriya TherapeuticsOur mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease. Kriya is a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world. With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $600 million, which will be used to advance a broad pipeline of gene therapies for ophthalmology, metabolic disease and neurology. For more information, please visit www.kriyatx.com and follow us on LinkedIn and X (Formerly Twitter).

Pictured: Illustration of a gene therapy showing a syringe injecting a treatment into a DNA strand/iStock, Ilya Lukichev Nanoscope Therapeutics claimed a hit on the changed primary endpoint of its Phase IIb vision loss gene therapy trial Tuesday, as the biotech looks to FDA approval of the candidate this year. The study compared two doses of MCO-010, a mutation-agnostic gene therapy, to a sham control in 27 people with retinitis pigmentosa. The chronic genetic eye disease is characterized by degeneration of the retina. Spark Therapeutics, part of Roche, won FDA approval for Luxturna (voretigene neparvovec) in the condition in 2017 but the authorization only covers patients with mutations in the RPE65 gene. Nanoscope designed MCO-010 to improve the vision of all patients with retinitis pigmentosa, regardless of the genetic basis of their condition. The gene therapy consists of an adeno-associated virus vector that delivers multi-characteristic opsins to re-enable retinal bipolar cells to detect light. Last year, an investigator in the clinical trial said the study did not meet its primary endpoint, which, at that time, was assessing the efficacy of MCO-010 using the multi luminance y- mobility test at 52 weeks. Spark tracked the change in multi-luminance mobility test scores over 52 weeks to show the efficacy of Luxturna. The test measures changes in functional vision, as assessed by the ability of a subject to navigate a course accurately and at a reasonable pace at different levels of light. Nanoscope changed the primary endpoint of its Phase IIb trial in January 2024 and released a statement about its talks with regulatory agencies the next week. According to Nanoscope, the FDA said the change from baseline in a measure of visual acuity in low vision patients could be an appropriate primary efficacy endpoint to support a filing for approval. Tuesday, Nanoscope presented data on the change in best-corrected visual acuity (BCVA) at week 52. The Phase IIb study linked both doses of the gene therapy to statistically significant improvements in BCVA, a vision test, causing the trial to hit its new primary endpoint. Nanoscope changed the primary endpoint after publishing 52-week results on the original measure of efficacy, although the biotech said it finalized and submitted a revised statistical analysis plan before the completion of week 76. The low dose failed to beat control on BCVA at week 76 but the high dose was significantly better than the sham treatment. The biotech tracked reductions in logMAR from baseline of 0.539 and 0.374, respectively, in the high and low dose arms after 76 weeks. Nanoscope said in January that the Icelandic Medicines Agency endorsed a 0.3 logMAR change as being clinically meaningful for patients with severe vision loss. The biotech is now preparing to FDA approval in the second half of 2024. Nick Paul Taylor is a freelance pharmaceutical and biotech writer based in London. He can be reached on LinkedIn.