Andelyn Biosciences’ Post

Cell and gene therapies (CGT), also known as “Advanced Therapy Medicinal Products” (ATMP), address diseases by directly repairing genetic defects within the body or introducing tissues or cells with new functions. These innovative therapies can correct or cure a wide range of conditions. The development and manufacture of these medicines are complex and novel, and they have only recently started gaining traction in the commercial sphere over the past decade. What sets them apart is their remarkable potential to offer permanent cures rather than temporary symptom reduction. Get an overview in the latest article by VTU experts Cornelia Haas and Birgit Krenn, which appeared in the magazine “botenstoff” of the Human.technology Styria GmbH (February 2024 issue, Precision Medicine special edition). #VTU #CellandGeneTherapy

Our CEO Jean-Philippe Combal had a fantastic time at the 10th Annual LSX World Congress where he joined a panel of key industry leaders for a captivating discussion on investing in #CGT clinical strategy to guarantee funding success.   The remainder of the day was spent enjoying productive #networking opportunities, catching up with familiar faces, and making new connections.   Find out more about #Vivet and its novel and long-lasting #rAAV based gene therapies for rare inherited liver metabolic disorders including #WilsonDisease and #CTX in the link in the first comment below!   #AAVGeneTherapy #GenomicMedicines #GeneTherapy #GenomeEditing 

🌍✨ Cell and gene therapies (CGTx) hold incredible promise for treating Cancer and rare genetic diseases, but global access and operational challenges still stand in the way. By expanding trial sites, embracing patient-centric technology, and prioritizing patient diversity, we can accelerate advancements and make transformative treatments accessible to all. Read the opinion of our Founder & CEO, Harsha K Rajasimha, Ph.D. from this Healthcare Business Today's Article: https://hubs.la/Q02M9SRs0 #clinicalresearch #genetherapy #clinicaltrials #medicalresearch #cellandgenetherapy

Osteoarthritis, one of the most common and debilitating chronic diseases, affects more than 30 million Americans, a number that is expected to increase exponentially as the population ages. Therapeutic options are limited. Current approaches start with nonpharmacological treatments such as walking aids, weight loss, and physical therapy, followed by acetaminophen and NSAIDS. Pioneers working on gene therapies for osteoarthritis and other chronic joint conditions won the prestigious OREF Award for 2024 at this year’s AAOS. One of the winners, Christopher Evans, PhD, discusses 30 years of research leading up to the founding of Genascence Corporation, which is now in clinical trials for a gene therapy for OA: https://bit.ly/3VDAh3R #medtech #osteoarthritis #chronicdisease #jointcondition #genetherapy

#podcast Nolan Townsend, CEO of Lexeo Therapeutics, and Jen Farmer, CEO of FARA, the Friedreich's Ataxia Research Alliance, are working together to better understand and treat Friedreich's Ataxia. This rare genetic condition includes cardiomyopathy and scoliosis. Lexeo Therapeutics is studying gene therapy as a potential treatment for the cardiovascular component of the disease. The Friedreich's Ataxia Research Alliance (FARA) plays a role in funding research, understanding the natural history of the disease, and advocating for treatments. #FriedreichsAtaxia #CureFA #FAAwarenessMonth #FAAwarenessDay #RareDiseases  LexeoTX.com CureFA.org https://lnkd.in/g94V2-Pz

Gene therapy has the potential to transform the lives of individuals with haemophilia, but should it be accessible to everyone? During #EHA2024, I attended an engaging debate where experts presented compelling arguments both in favour of and against expanding access to these innovative treatments. Here are some of my key takeaways from both sides of the debate, illustrating the many factors that must be weighed when considering these cutting-edge therapies for patients. Please reach out if you would like to learn more about our insights on gene therapy in rare blood disorders.

⚕️ Project Recomb: A New Hope for SCID Treatment Project #Recomb is an ambitious initiative that aims to develop a new therapy for #SCID, a severe genetic disease that compromises the immune system. The main focus of this clinical trial on RAG1-SCID, that is unique worldwide, is to carry out curative stem cell-based gene #therapy. This #project involves the collaboration of various stakeholders, including researchers, doctors, patients and scientific institutions, all working together to achieve a common goal: improving the lives of those living with SCID. We look forward to sharing the successes of this innovative journey. Watch here 👇

Cell and gene therapies are novel forms of medical treatment that require experienced teams with a deep understanding of the science behind these clinical trials. Ask how we can manage your trials and provide flexible and tailored support per your site and country guidelines. Learn how here: https://hubs.la/Q02m535C0 #genetherapy #celltherapy #clinicaltrials #cellandgenetherapy

Blood Transfusions for Thalassemia: A Lifesaving Therapy Blood transfusions are a crucial part of managing thalassemia, a genetic blood disorder. In this video, we'll delve into the reasons why thalassemia patients need blood transfusions, how often they are typically needed, the process involved, and the potential risks and benefits. We'll also discuss alternatives like iron chelation therapy and emerging treatments like gene therapy. Our Channel Name] is committed to providing comprehensive and accurate health information. Join us as we explore the role of blood transfusions in thalassemia management and empower you with knowledge. Blood transfusions play a vital role in managing thalassemia. Watch our full video to understand why they are needed, how they work, and the potential risks and benefits for patients. If you or someone you know has thalassemia, understanding blood transfusions is essential. Watch the full video on our YouTube channel and share it with others to spread awareness. #ThalassemiaAwareness #Thalassemia #BloodTransfusion #ThalassemiaTreatment #BloodDisorder #GeneticDisorder #ThalassemiaAwareness #HealthEducation #MedicalTreatment

Gene Therapy: A New Hope for Rare Diseases Gene therapies are leading the charge in treating rare and life-threatening conditions. From spinal muscular atrophy to hereditary transthyretin amyloidosis, these breakthrough therapies are offering new hope by addressing diseases at their genetic root. Dive into the latest clinical trial successes and see how these advancements are reshaping the future of medicine. 🧬 #RareDiseases #GeneTherapy #FutureOfMedicine