CAI’s Post

🔬✨ Transforming #Healthcare with Cell & Gene Therapies! ✨🔬 Pharma R&D is pushing boundaries with cell and gene therapies, offering new hope for genetic disorders like cancer and autoimmune diseases. These innovative treatments use living cells and #DNA/#RNA to replace faulty genes or cells, changing lives. 🌿🧬 🔍 How do Cell & Gene Therapies (#CGT) work, and what makes them so effective? 🌟 Our article explores the crucial role of #AncillaryMaterials (AMs) in these groundbreaking treatments. 🚀 Technologies like #CRISPR, CAR-T, and #AI-driven personalised medicine are pushing the boundaries of medical science, promising more precise and effective treatments. Learn more about these revolutionary therapies in Đurđa Zdravković's and Dr. Hürter's article: https://lnkd.in/epNZJj5h #innovation #cellandgene

Approval of Casgevy(Vertex & CRISPR Therapeutics) and Lyfgenia( Bluebird Bio), two first gene editing treatments by the USFDA is great news not only for SCD patients but also provides hope for patients with other genetic diseases. The lucky break behind the discovery of Casgevy is captured in : https://lnkd.in/gHWEfi8J. However there are still practical hurdles for SCD patients before this breakthrough can benefit them. For one, the cost is prohibitive : List price of Casgevy and Lyfegnia is 2.2 mio USD and 3.1 mio USD respectively. https://lnkd.in/gAXQgTDt Landmark approval of the first CRISPR therapy paves the way for treatments based on more efficient and more precise genome editors. https://lnkd.in/g-bKtjFP

The future of drug development is here! Last year, the FDA approved two milestone treatments, Casgevy and Lyfgenia, for the treatment of sickle cell disease (SCD) in patients 12 years and older. What's exciting is that Casgevy is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy. This is an exciting development and we can't wait to see what's next! #drugdevelopment #genetherapy #SCD #innovation

#AdenoAssociatedVirus, known as AAVs, have begun emerging as a vector for providing therapeutic genes to target cells which can show significant potential in treating #GeneticDisorders and #ChronicDiseases. These advancements have led to improved safety, efficacy, and scalability. While there are challenges, such as packaging capacity and opportunities, innovation has begun in cell and gene therapy to overcome specific barriers and optimize treatment outcomes.

As I analyze spending trends within the Sickle Cell community and the correlation with Emergency Department (ED) visits, reading this article 'FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease' instills hope for the community. Despite the prevailing stigma and racial stereotypes affecting their access to care, I am optimistic about the positive impact gene therapy could have on their quality of life, potentially reducing ED costs associated with their illness. I'm a proponent of prioritizing immediate care when needed. #SickleCellAwareness #HealthcareOptimism #GeneTherapies https://lnkd.in/g3xpZegx

🚀 Big News in #SickleCellDisease (SCD) #Innovation - FDA Approved First Gene Therapy 🌟 THIS is a game changer for patients in a disease that has a high burden of disability and mortality. 🌟 The data is astounding: SCD occurs in about 1 out of every 365 Black or African-American births, and about 1 out of every 16,300 Hispanic-American births. About 1 in 13 Black or African-American babies is born with sickle cell trait (SCT). 🌟This is the BEST holiday gift for many of us who have treated patients with #sicklecelldisease - we now have the power to rewrite the narrative and transform outcomes. #SickleCell #MedicalInnovation https://lnkd.in/ezNXpCXg

Reflecting on the advancements in #genetherapy throughout 2023, we observe: ✔ multiple approvals, ✔ an expanding clinical trial pipeline, ✔ and increased regulatory support, signaling the early maturation of the field. Despite progress in understanding gene therapy mechanisms and improving delivery systems, achieving financially sustainable and commercially viable therapies remains a challenge. Unlocking the true potential of gene therapies demands a united effort across the industry, especially in enhancing biomanufacturing practices. Let's collaborate to make gene therapies not just accessible, but a transformative reality 🚀🧬! #enzymes #bioprocessing #cgt #efficiencyboost #innovativesolutions Explore the exciting highlights of 2023 in CGT by clicking here: https://lnkd.in/eHCvhkq5.

Cell and gene therapies are revolutionizing modern medicine, offering new hope and treatment options for a variety of diseases. Check out this e-book called "Cell and Gene Therapies: From the Lab to the Clinic" that dives into recent advancements, challenges, and clinical applications in this field. From breakthroughs to tackling rare diseases, it's a must-read for anyone interested in the future of healthcare. Download the e-book below 👇 #CellTherapy #GeneTherapy #RareDiseases

Cell and Gene Therapy: Modern Solutions for Modern Health Challenges  Dr Nilanjan Guha, PhD - Country Academia and Research Manager, Agilent Technologies From #personalizedtreatments to tackling #geneticdisorders, this article sheds light on how these #moderntherapeutic approaches are reshaping the landscape of healthcare. It's amazing to see science and technology converge to bring hope to patients and pave the way for a healthier future. #CellTherapy #GeneTherapy #HealthcareInnovation #ScienceAndTechnology #MedicalAdvancements #HealthcareFuture

This. Is. Huge! It's like the AI moment in Medicine, an era defining event The @FDA has approved not 1 but 2 gene based therapies fro #SickleCellDisease. The first uses the CRISPR gene editing technique (which earned its inventor Nobel prize in 2020) According to the studies cited by the FDA press release, 94% of patients achieved desired clinical outcome. The cost of the treatment? 2.3m usd per patient (KES 356M), not including costs of hospitalization, laboratory testing and ancillary services related to the treatment. Will this ever be affordable in our side of the world? For now, I'm celebrating this breakthrough for millions of SCD patients. Hopefully it will become affordable in our lifetime! https://lnkd.in/dd4c8v4X