Helix Biotech, Inc.’s Post

Europe's Gene-Editing Milestone - European Commission Approves CRISPR/Cas9 Therapy, CASGEVY™, for severe sickle cell disease SCD and transfusion-dependent beta thalassemia TDT CRISPR Therapeutics announced the European Commission's conditional approval of CASGEVY for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The therapy, targeting patients 12 years and older, potentially benefits over 8,000 individuals. CASGEVY is the first genetic therapy approved for SCD and TDT in the EU, offering hope to patients with limited treatment options. CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy that enhances fetal hemoglobin production, potentially reducing symptoms and eliminating transfusion requirements. Vertex Pharmaceuticals leads global development and commercialization efforts, ensuring early patient access and establishing treatment centers across the EU. The approval of CASGEVY reinforces CRISPR/Cas9 technology's potential to revolutionize treatment for severe genetic diseases, providing hope to patients and paving the way for really transformative therapies. #CRISPRTherapeutics #GeneTherapy #RareDiseases #CRISPR #CRISPRTechnology #biotechnology Source: https://lnkd.in/eu95Qtuc

The final « Optimus » guidance is available !

👉 Discover the Latest Innovations in Disease Treatment - New Article on the Mabion website! Last year was a landmark year for innovative biologic medicines that combined the latest scientific discoveries with dynamic developments in applied technologies to fight the most challenging diseases. The European Medicines Agency (EMA) approved a total of as many as 18 new biological drugs and the Food and Drug Administration (FDA) approved a record 34 biopharmaceuticals. We have selected and described for you the top 10 drugs that address untapped medical needs and have the greatest potential to transform medical practice. Among them are: 🔸 the first-ever protein-based vaccines against RSV, 🔸bispecific antibodies to treat therapy-resistant cancers, 🔸CRISPR/Cas9-based gene therapy for sickle cell anaemia. Read our latest article on the Mabion S.A. website, where we detail these new innovations that have the potential to change the face of medicine. Find out more and stay up to date with the latest medical developments. Article here 👉 https://lnkd.in/dnJrND6S #MedicalInnovations #BiologicalDrugs #Mabion #EMA #FDA #MabExpert

Hot off the grill! The FDA has just posted the final guidance for industry on "Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases"! This guidance is intended to assist sponsors in identifying an optimized dosage(s) for human prescription drugs or biological products for the treatment of oncologic diseases during clinical development prior to submitting an application for approval for a new indication and usage. This guidance does not address selection of the starting dosage for first-in-human trials. In addition, this guidance does not address dosage optimization for radiopharmaceuticals, cellular and gene therapy products, oncolytics, microbiota, or cancer vaccines, nor does it specifically address pediatric drug development. Check this out! https://lnkd.in/ek2G27xk

Wonderful, the final FDA guidance of Optimus has been released. If you are a company looking for a fgroup of clinical pharmacologists and paharmacometricians to support you in this strategy, reach out to us. We have >20 years of expertise in the field , always adapting our mindset to this evolving landscape Talk to you soon #optimus #dosingoptimization #innovativedesigns #clinicalpharmacology #pharmacometrics

📣FEBRUARY'S FDA ROUNDUP - ACDN-01: Stargardt Disease On January 29, 2024, Ascidian Therapeutics announced that the FDA has cleared its investigational new drug (IND) application and granted Fast Track designation for ACDN-01.6 According to the report, “ACDN-01 is the first-ever clinical-stage RNA exon editor and the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease.”6 “This open IND for ACDN-01 by the FDA – the first regulator to have cleared ACDN-01 for clinical development – represents an important milestone for Ascidian and the broader field of RNA editing,” said Michael Ehlers, MD, PhD., President and Interim Chief Executive Officer of Ascidian Therapeutics. “We chose to go to the FDA first because we have conviction in the rigor of our data, and that by editing RNA and not DNA, the Ascidian approach brings unique advantages with potential to transform the lives of people living with Stargardt disease and, more broadly, to dramatically expand the reach of genetic medicine.”6 ⭐The FDA Roundup is bringing you up to speed with the latest announcements and approvals in #Ophthalmology. Read here ➡️: https://brnw.ch/21wHc8w #ACDN01 #StargardtDisease #InvestigationalNewDrug #INDApplication #FastTrack #Ophthalmology #ClinicalTrials #EyeHealth

What’s new this week in the #cellandgenetherapy sector? 🧬The first CRISPR gene editing therapy approved in the UK 🔬Cell therapies for heart and autoimmune conditions advance in the pipeline 🤝$620m deal for genetic medicines manufacturer Read the full news roundup. ⬇ Vertex Pharmaceuticals and CRISPR Therapeutics announced the approval of the first CRISPR/Cas9 gene-edited therapy, CASGEVY™, by the United Kingdom MHRA for treating sickle cell disease and transfusion-dependent beta-thalassemia. https://lnkd.in/gG89yk6R BioCardia, Inc. announced the FDA cleared Phase III clinical trials of its CardiAMP autologous cell therapy for treating patients with ischemic heart failure. https://lnkd.in/g9FGi2a7 Kyverna Therapeutics announces FDA clearance of Phase 2 IND for KYV-101, a fully human CD19 CAR T-cell therapy to treat myasthenia gravis. https://lnkd.in/gre4YtyS Ajinomoto Co. and Forge Biologics announced that they have entered into a definitive agreement by which Ajinomoto Co. will acquire Forge in an all-cash deal for $620 million. https://lnkd.in/e4fq76Yf

🤷♀️ What to know about a versatile LNP production process for mRNA vaccines, therapeutics, and gene editing? 📣 Excited to announce our next #webinar! Andreas Wagner from Polymun Scientific Immunbiologische Forschung GmbH will tell us more. 📅 Join the #online #seminar on April 24, 2024, 3:30 pm CEST. 💡 #RNA-based therapeutics, which function by either silencing pathological genes through delivery of #siRNA or expressing therapeutic proteins through the delivery of exogenous #mRNA to cells, hold great potential for the treatment of various diseases, like #Covid-19 related diseases. However, mRNA molecules are large, fragile and easily degrade. They do not readily cross plasma membranes to enter target cells and so a delivery solution is required. 💡 #Lipid #nanoparticles (#LNPs) are the leading delivery systems for enabling the therapeutic potential of siRNA, mRNA for systemic applications or #CRISPR. LNPs, currently represent the most advanced platform for RNA delivery, which have now advanced into human clinical trials and their mRNA delivery safety profiles have been evaluated in human and non-human primates. 💡 Lipid nanoparticle delivery platforms have been extensively investigated and optimized for the formulation of oligonucleotide drug products and provide a good basis for mRNA-based systems. However, mRNA containing LNPs need to be treated differently than oligonucleotide containing LNPs, as particle structure has an impact with respect to stability upon processing conditions. 🔔 Data will be presented, which describe hurdles and solutions throughout these processes. JOIN! #research #pharma #forschung #LNP #geneediting

📣 Slides available for download! 💻 Last week, Andreas Wagner from Polymun Scientific Immunbiologische Forschung GmbH gave a #webinar on the versatile #LNP production process from #mRNA vaccines, therapeutics, and gene editing. You missed this online #seminar? Slides of the presentation are now available for download! 👉 Check our website. #education #lipidnanoparticles #geneediting #vaccine #RNA

On December 5th, I will be presenting Levatio's Circular RNA platform at the ‘mRNA Technologies, Therapeutics & Vaccines Digital Week’ hosted by TIDE. Title:   A Novel Approach to Circular RNA Synthesis and Its Therapeutic Potential Here's the table of contents for my talk. 1.    Company Overview 2.    Levatio Circular RNA Platform Technology 3.    Engineering the Internal Ribosome Entry Site (IRES) of Circular RNA 4.    Immunogenicity Assessment for Circular RNA 5.    Circular RNA Delivery System: LPX-a 6.    Circular RNA-Based Drug Development - Neoantigen-Cancer Vaccine - Autoimmune Diseases - BIO-PROTAC - Cas-9   You can join the webinar via the link below. https://lnkd.in/ehjrTkzi