Biallelic RPE65 mutation associated retinal dystrophy

COLUMBUS, Ohio, Oct. 9, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by Hubble Therapeutics, LLC, to manufacture adeno-­associated virus (AAV) using its suspension AAV Curator™ Platform to manufacture clinical grade AAV for the treatment of Leber Congenital Amaurosis 16 ("LCA16"). LCA16 is a severe, early-onset retinal dystrophy resulting from mutations in the KCNJ13 gene, which expresses a critical ion channel in the retinal pigment epithelium cells in the retina. Children typically present with visual abnormalities within five years after birth, followed by progressive retinal degeneration during the next two decades of life, ultimately resulting in blindness. HUB-101 has received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA for this program. Andelyn will optimize and scale-up the HUB-101 process, generate GMP-grade plasmids, and produce GMP vectors that include normal copies of the KCNJ13 gene. From this partnership, Hubble Therapeutics LLC ("HubbleTx") will be able to conduct Phase 1/2 clinical trials with the intention to demonstrate safety and efficacy in an adult patient population. "Over the past 18 months we have identified best-in-class partners including Andelyn Biosciences to establish a gene therapy development program for adult and pediatric patients suffering from Leber Congenital Amaurosis 16 (LCA16)," said Jeff Sabados, President of Hubble Therapeutics LLC. Sabados continued, "Andelyn Biosciences has become a global leader in gene therapy development for ultra-rare and orphan diseases and we are grateful for their unwavering commitment toward helping Hubble Therapeutics realize our steadfast promise to support families throughout the world impacted by this devastating genetic disease." Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said, "We are extremely grateful for the opportunity to produce the clinical grade material for HUB-101 and be a critical part of the pathway for Hubble Therapeutics to demonstrate clinical benefit for the LCA16 program. We are thrilled that our establishment as a top clinical and commercial CDMO is helping to accelerate so many programs like HUB-101 which offer real hope to patients stricken with debilitating diseases." With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three facilities located in and near Columbus, Ohio, Andelyn continues to partner with premier innovator organizations like Hubble Therapeutics to enable the development of life-altering gene therapies for rare and prevalent diseases. About Andelyn Biosciences, Inc. Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator™ Platform, or tech transfer in an established client program. Capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A rigorous quality system, regulatory support and supply chain vertical integration further support clients in bringing their critical therapies to market. For more information, visit andelynbio.com. About Hubble Therapeutics, LLC Hubble Therapeutics LLC ("HubbleTx") is a patient-focused biotechnology company developing research out of Professor Bikash Pattnaik's lab at University of Wisconsin-Madison. Our primary focus is on developing a novel gene therapy for Leber Congenital Amaurosis Type 16 (LCA16), a debilitating retinal disease that leads to severe vision loss in adult and pediatric patients. Our mission is to restore vision and improve the quality of life for affected individuals and their families by harnessing the power of gene delivery technology to target the underlying genetic mutation responsible for the disease. Our team of leading scientists, industry experts and best-in-class partners are committed to rigorous research and clinical development, ensuring that our therapies are safe, effective, and accessible. Through collaboration and innovation, HubbleTx strives to bring hope to those affected by this condition. Together, we aim to illuminate the path to a brighter future for patients battling LCA16. SOURCE Andelyn Biosciences WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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

SHANGHAI and REDWOOD CITY, Calif. , May 29, 2024 /PRNewswire/ -- HuidaGene Therapeutics ("HuidaGene"), a global clinical-stage biotechnology company focused on advancing CRISPR-based genomic medicines, and Synthego Corporation ("Synthego"), a leading provider of innovative CRISPR solutions for developing cell and gene therapies, proudly announce a partnered licensing agreement for the high-fidelity Cas12 CRISPR nuclease (hfCas12Max). This partnership underscores the significant clinical utility of hfCas12Max, naturally complementing Synthego's focus on advanced GMP manufacturing capabilities. Under this licensing agreement and pursuant to undisclosed financial terms between HuidaGene and Synthego, HuidaGene grants Synthego manufacturing and commercialization rights for the hfCas12Max nuclease and optimized gRNA for research; HuidaGene also grants Synthego the right to sublicense the nuclease for therapeutic use. This partnership will streamline the development of CRISPR-based therapeutic applications, providing developers with access to highly precise, efficient, and next-generation genome editing tools. Xuan Yao, Ph.D., Co-founder and President of HuidaGene, emphasized the significance of this agreement, stating, "HuidaGene's expansive CRISPR intellectual property portfolio distinguishes it as a vanguard in genomic medicine, with a versatile pipeline targeting critical neurological and ophthalmological conditions. Together with Synthego, we are poised to significantly accelerate the advancement of CRISPR-based therapies and deliver life-altering genomic medicines to patients worldwide." Craig Christianson, CEO of Synthego, expressed enthusiasm about the partnership with HuidaGene on hfCas12Max, stating, "The combination of our advanced CRISPR GMP production capabilities and regulatory expertise with HuidaGene's next-generation nuclease technology is a crucial step toward advancing transformative cell and gene therapies. This partnership underscores Synthego's commitment to CRISPR-based therapeutics, bolstered by our focused investments in GMP manufacturing and comprehensive CRISPR solutions that support therapeutic development. The integration of hfCas12Max naturally aligns with our initiative to enhance the accessibility and efficiency of CRISPR tools." hfCas12Max, a novel CRISPR gene-editing system developed through HuidaGene's HG-PRECISE platform, stands out for its superior on-target editing efficiency and reduced off-target editing activity in mammalian cells. This novel CRISPR gene-editing system also offers the advantage of packaging into a single viral vector, an important requirement for many cell and gene therapies. The commercialization of hfCas12Max will significantly enhance the accessibility of this CRISPR gene-editing system, providing increased freedom-to-operate and facilitating the development of CRISPR-based cell and gene therapies. This partnership reinforces both companies' positions as catalysts for innovation, advancing their shared mission of transforming genomic-based cell and gene therapies and enabling unprecedented therapeutic outcomes. For partnership inquiries, please contact [email protected] and [email protected]. hfCas12Max is a registered trademark in China. About HuidaGene HuidaGene Therapeutics utilizes its proprietary CRISPR-based HG-PRECISE® platform to discover, engineer, and develop potentially curative genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted both ODD and RPDD), HG202 CRISPR RNA-editing in neovascular age-related macular degeneration, and the preclinical pipeline, including HG204 CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted both ODD and RPDD by U.S. FDA and ODD by EMA), HG302 CRISPR DNA-editing for Duchenne muscular dystrophy (granted both ODD and RPDD), and HG303 CRISPR DNA-editing for Amyotrophic Lateral Sclerosis (ALS). The Company's extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicine. Learn more at huidagene.com or on LinkedIn. About Synthego Synthego is a pioneering provider of genome engineering solutions, offering a comprehensive suite of products and services to accelerate CRISPR-based cell and gene therapy development. With a commitment to empowering researchers and innovators, Synthego's cutting-edge CRISPR technologies and expertise drive progress from preclinical to clinical application. Learn more at Synthego.com or on LinkedIn. SOURCE HuidaGene Therapeutics