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



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

Canadian Tire Bank Selects Temenos Open Platform to Gain Best-In-Class Digital Banking Capabilities18.5.2022 09:00:00 CEST | Press release

Temenos (SIX: TEMN) today announced that Canadian Tire Bank (CTB), the financial arm of Canadian Tire Corporation (CTC) – one of the largest Canadian retail companies – has selected Temenos open platform for composable banking to replace its legacy systems. Temenos will help power Canadian Tire Bank’s growth by delivering faster to market and richer digital experiences, boosting customer engagement and bottom-line revenue. The new banking platform is part of CTC’s Better Connected strategic growth plan to invest in its omnichannel customer experience. Temenos open platform will help Canadian Tire Bank (CTB) drive its digital-first strategy to enhance customer acquisition, loyalty and engagement. Canadian Tire Bank is one of the largest card issuers in Canada, with more than 2 million customers, over $6B in receivables and was recently ranked second in JD Power’s Canada Credit Card Satisfaction Study. With CTB focused on launching a new set of digital-only offerings, Temenos will help e

Temenos Delivers Digital Banking Solutions on AWS18.5.2022 08:30:00 CEST | Press release

Temenos (SIX: TEMN) and Amazon Web Services (AWS), have extended their global relationship to run Temenos onboarding and origination solutions on AWS. The technology collaboration aims to allow all retail and commercial banks - incumbents and challengers - to provide digital onboarding and origination solutions with higher performance, scalability, and security. It will give banks greater agility while reducing time to market and improve cost efficiency. The two companies will go-to-market with solutions for retail, business, corporate and private banking, and wealth. This multi-year agreement aims to offer the full suite of Temenos banking solutions on AWS as-a-service. Temenos and AWS started working together in 2019 to integrate Temenos open platform onto AWS and have demonstrated success with joint banking customers since then. With Temenos digital banking solutions, Temenos and AWS are making it easier for banks to access the technology they need to meet rising customer expectatio

Collecting Sensitive Data Without User Consent: 24 Leading Online Pharmacies From Nordic Countries Fail GDPR Requirements – Usercentrics Reports18.5.2022 08:01:00 CEST | Press release

Analysis by Usercentrics shows: Over 80% of scanned domains from Denmark, Sweden and Norway are not GDPR compliant, setting at least one non-necessary cookie without user consent. Sweden ranks highest with an average of 26 non-necessary cookies set. In total 89% of the most popular online pharmacies in the EU do not meet GDPR requirements. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220517006345/en/ Lack of Data Protection on EU Pharma Webshops: The majority of websites do not operate in a privacy-compliant manner - analysis by Usercentrics shows. (Graphic: Business Wire) Pharmacy webshops constantly process personal data of their visitors, while selling privacy-sensitive products such as antidepressants, diabetes medicines or addiction treatments. Usercentrics scanned the 150 most-used pharmacy webstores in the EU, analyzing the extent to which website operators are complying with the General Data Protection Regulation (

Banks Shift to Eco-friendly Solutions Supported by Innovative Thales Cards, Certified Sustainable by Mastercard18.5.2022 08:00:00 CEST | Press release

The amount of plastic used to produce payment cards every year is equivalent to the weight of 125 Statues of Liberty1. With environmental challenges now a growing, global concern, Thales, the leader in high tech security, has developed solutions for a greener world, recently certified by Mastercard’s Sustainable Card Program. To date, Thales has deployed 30 million eco-friendly payment cards for banks and card issuers that address consumer concerns without compromising the user experience. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220517005012/en/ (Photo: Thales) All eco-friendly cards offered by Thales are made with innovative and sustainable components. For example, one solution is a card made from 85% PLA (polylactic acid), which is produced from corn. Another innovative approach reduces the amount of new plastic while fighting sea pollution, with a card made of Ocean Plastic® collected by Parley for the Oceans. This

Anterra Capital: Pandemic and Food Price Inflation Fuel Investor Interest to Accelerate Transformation of the Food and Ag Sector18.5.2022 07:00:00 CEST | Press release

A crushing pandemic and, more recently, the Ukraine war have convinced investors more than ever that the world needs to radically change how it produces, distributes and consumes food, says Adam Anders, managing partner of the international food and agriculture specialist venture capital firm Anterra Capital (“Anterra”). Anders: “The pandemic highlighted the fragility and adverse health impacts of our current food system: soaring prices, empty shelves in stores; the fact that unhealthy diets are a major risk factor for getting Covid; that the way we treat farm animals could trigger the next pandemic. Post our final closing, the devastating impact of the Ukraine War on the global food supply has increased the awareness among investors even further. This, plus our focus on deploying proven biotech and digital solutions to shake up the lagging Food and Ag-sector, go a long way in explaining the higher than expected interest in our second fund.” Anterra is proud to announce the closing of

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom