Business Wire

CYTOO’s Partner Astellas Gene Therapies Has Presented Results of an AGT/CYTOO Collaboration on Clinical Candidate Selection to Treat DMD at the 2 nd Gene Therapy for Muscular Disorders Summit

Share

CYTOO and Astellas Gene Therapies (AGT) entered into a research collaboration in 2020 to select AGT’s AAV gene therapy clinical candidate to treat Duchenne Muscular Dystrophy (DMD). The results of this collaboration have been presented at the 2nd Gene Therapy for Muscular Disorders Summit in Boston.

DMD is a rare and life-threatening genetic disorder that affects children–approximately 1 in 3,500 to 5,000 boys–and families. It is caused by mutations in the dystrophin gene that results in progressive muscle degeneration and weakness. By the early teens, most individuals with DMD have lost the ability to walk unassisted and their heart and respiratory muscles have also weakened. Without therapy, individuals with DMD usually die from cardiomyopathy and respiratory failure in their second decade of life.

CYTOO pioneered MyoScreen™, an in vitro R&D platform that enables the evaluation of therapies in patient-derived primary skeletal muscle cells under physiological conditions. The MyoScreen platform is compatible with most AAV-based gene therapies and facilitates the development of quantitative, image-based assays monitoring the activity and mechanism of action of these therapies. The collaboration between AGT and CYTOO assessed the activity of several AAV-mediated exon skipping candidates using a quantitative cell profiling assay developed at CYTOO. This assay employs AI-assisted comparisons of images from myotubes from healthy and disease donors to establish profiles that phenotypically distinguish healthy and patient cells. These profiles are then used to determine a Health-Score™ (% phenotypically healthy cells out of total) that evaluates the ability of therapeutic candidates to revert disease-associated phenotypes in patient-derived myotubes.

AGT’s investigational exon skipping gene therapy approach to treat DMD uses an AAV vector, encoding modified U7 small nuclear RNAs (snRNA), to deliver an antisense sequence designed to induce cells to skip over faulty or misaligned sections of genetic code in the dystrophin gene, with the goal of restoring meaningful levels of a functional dystrophin protein. For the treatment of DMD, this approach has the potential to provide significant advantages over microdystrophin gene replacement strategies that produce a substantially truncated protein, which may limit the degree and durability of disease correction, as well as existing antisense oligonucleotide (ASO) therapies whose efficacy is limited by poor biodistribution to muscle tissue.

Luc Selig, CYTOO’s CEO, said, “The data to be presented today show that MyoScreen is a unique platform to demonstrate and quantify the functionality of restored dystrophins in DMD myotubes treated with AGT’s AAV-mediated exon sipping.” Luc added: “We strongly believe that the ability to evaluate muscle therapies in patient-derived cells with quantitative and functional assays provides a much-needed alignment of preclinical discovery with clinical endpoints.”

About CYTOO

CYTOO is a drug discovery biotech company focusing on disorders affecting muscle health. CYTOO pioneered MyoScreen™, an in vitro R&D platform that enables testing muscle therapies in primary patient-derived skeletal muscle cells. Using innovative, image-based, high-throughput assays and quantitative functional assays, MyoScreen aids therapeutic discovery at all stages, from target identification to the development of clinical candidate potency assays. Through R&D partnerships, CYTOO has worked on more than 50 projects comprising RNA and gene therapies. To better support CMC related activities, CYTOO is establishing a GMP environment that will be operational in 2023.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Press : Florence Portejoie, FP2COM, fportejoie@fp2com.fr

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Biognosys Announces Appointment of Beat Lüthi, Ph.D., as independent Board Director19.5.2022 14:00:00 CEST | Press release

Biognosys, a leader in next-generation proteomics solutions for life science research and drug development, announced today the appointment of Beat Lüthi, Ph.D., as Board Director. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005079/en/ Beat Lüthi, Ph.D. – Biognosys' Newly Appointed Board Director. (Photo: Business Wire) Dr. Lüthi is Chief Executive Officer of CTC Analytics AG, a privately owned Swiss company leading in automation and advanced laboratory sample handling solutions for the pharmaceutical, life science, chemical, environmental, and food & flavor industries. Additionally, he holds several Board of Directors mandates in public firms, including Chairman of Inficon Group, Vice Chairman of Straumann Group, and Board Member of Skan AG. Dr. Lüthi holds an M.Sc. in Electrical Engineering and a Ph.D. in Business Administration from ETH Zurich and completed an International Executive Program at the INSEAD Busines

Inversago Pharma Presents Data on INV-202 in Diabetic Nephropathy at the ERA 2022 Congress19.5.2022 14:00:00 CEST | Press release

Inversago Pharma Inc. (“Inversago”), a clinical stage biotech company with a unique portfolio of CB1 inverse agonists, announces the presentation of preclinical data at the 59th Congress of the European Renal Association (ERA), the largest annual nephrology meeting in Europe, currently held in Paris, France. These data show that INV-202, the company’s lead peripheral CB1 blocker, reduced renal injury in a murine model of Streptozotocin (STZ)-induced Diabetes. More specifically, INV-202 reduced glomerular injury, renal fibrosis and injury to proximal tubular epithelial cells, and also preserved podocyte structure and function. In particular, there was a marked, dose-dependent reduction in albuminuria in this model of diabetes and associated kidney injury. “The study showed that treatment with INV-202 reduced the progression of nephropathy in STZ-induced diabetic mice. Improvements in kidney size, function, and renal matrix remodeling, with corresponding gene expression were noted with b

Grovara Partners with Dubai Global Connect, Establishes Middle East HQ and Showroom19.5.2022 13:00:00 CEST | Press release

Grovara, the easy global wholesale experience, announces it has signed a lease agreement and become formally incorporated to do business in Dubai as part of a partnership with Dubai Global Connect (DGC), a permanent wholesale market that facilitates business between international buyers and sellers. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005017/en/ Grovara and Dubai Global Connect cemented their partnership on April 26, 2022 at DGC's new Visitor Centre in Dubai. Standing, from left are Grovara Sales Director Eugenia Schlitter, Grovara Executive Chairman David Pottruck, Grovara Chief Innovation Officer/Co-founder Peter Groverman, Dubai Global Connect Food Business Development Lead Badreddine Soukarie, and Grovara VP of Global Supply Chain Juliana Rogenski; Seated, from left are Dubai Global Connect Head of Business Development Paul Boots and Grovara CEO/Co-founder Abu Kamara. (Photo: Business Wire) The partnersh

Zilliant Launches Quick Start Program to Speed Time to Value and First Quick Start Package, Transforming How Companies Manage Pricing in Response to Inflation19.5.2022 13:00:00 CEST | Press release

Zilliant, the industry leader in intelligent end-to-end pricing solutions and sales guidance software, today announced its Zilliant Quick Start Program, which enables significantly faster deployment times for its software and faster time-to-value for customers. The first Quick Start package of this transformative program is Zilliant Quick Start for Global and Country Price Lists, which is available beginning today. In the past, deploying price optimization and management software may have seemed time intensive or difficult to implement. As a direct result, pricing and commercial teams rely on spreadsheets to manage pricing, sales operations and commercial functions, a method that is insufficient to respond to today’s volatile market conditions. The Zilliant Quick Start program flips this paradigm, offering packaged solutions to address specific pricing and sales challenges, each of which can be up and running within the span of three to eight weeks, work seamlessly with each other on t

Eaton Introduces DC-DC Converters for 48-Volt Commercial Vehicle Architectures19.5.2022 12:30:00 CEST | Press release

Power management company Eaton today introduced a family of 48-volt DC-DC converters for diesel-powered commercial vehicles that can be used to power accessories such as antilock brakes and lighting. Unlike competitive offerings, Eaton’s DC-DC converters are operational in ambient temperatures up to 85 degrees Celsius and boast 97% design efficiency. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005093/en/ Eaton’s family of 48-volt DC-DC converters can be used to power accessories such as antilock brakes and lighting and are operational in ambient temperatures up to 85 degrees Celsius. (Graphic: Business Wire) “DC-DC converters are an essential part of our eMobility product portfolio and are used in a variety of vehicle applications,” said Ben Karrer, director, Technology Development, Eaton’s eMobility business. “Many commercial vehicle manufacturers are transitioning to 48-volt architectures, so having the ability to