Courtney Rice’s Post

The ATTC Networks webinar 'Evaluation of the Patient Referral Pathways for Gene Therapy and CAR-T in the UK' which was hosted last week is now available to view on-demand! Tune in to learn more about the existing patient referral pathways for advanced therapies in the UK and how these can be improved. In this webinar, speakers provided an expert overview of two projects that assessed patient referral pathways for different advanced therapies and the resulting best practice recommendations. Watch it here: https://buff.ly/49guJA0 #ATTC #PatientReferral #CellTherapies #GeneTherapies

Interesting Review in the field of DNA/RNA Therapeutics! Our product, in vivo-jetPEI®, has been featured prominently in the recent peer-reviewed paper titled "Polyethylenimine (PEI) in gene therapy: Current status and clinical applications," authored by J. Casper et al. 📑 This comprehensive review delves into the Research & Development and Clinical applications of PEI, illustrating the use of in vivo-jetPEI®, as a reference delivery tool for developing localized DNA-based treatments for solid tumors and therapeutic vaccination. The paper also showcases the clinical use of in vivo-jetPEI® as a successful and proven excipient for the delivery of DNA/RNA therapeutics through a detailed table summarizing numerous clinical trials over the years. To read the article : https://lnkd.in/ejc2XhGs. #Polyplus #Sartorius #invivo

Today’s publications share some of the latest health-related news. The first article is from the U.S. Food and Drug Administration, which just approved Tecelra (afamitresgene autoleucel), a gene therapy indicated for the treatment of adults with unresectable or metastatic synovial sarcoma. Other conditions are indicated. https://lnkd.in/e9_Mh_av The next article looks at a new multicenter international study led which found that people with the B-cell precursor subtype of acute lymphoblastic leukemia (BCP-ALL), who also lacked a genetic abnormality known as the Philadelphia chromosome and were in remission with no trace of cancer, showed significantly higher survival rates when blinatumomab was added to their chemotherapy treatment. https://lnkd.in/gK6CpZZU The last article states how a mathematical method, validated with experimental data, provides a fast, reliable, and minimally invasive way of determining how to treat critical blood pressure changes during surgery or intensive care. https://lnkd.in/g_XyT64f #manhattanlife #medicalscienceandtech #healthcarefindings #healthrelatednews

Here is our recent article on long term follow up of metastatic pancreatic cancer patients that have been treated with HFHS developed gene therapy vector. Benjamin Movsas David Kwon #genetherapy #henryford #pancreaticcancer

💡 Exciting News from SparingVision at the Macula Society 47th Annual Meeting!   We are proud to announce that we have presented clinical data for our pioneering gene therapy, SPVN06, for the first time. This represents a significant milestone in our journey towards transforming the treatment of retinal disease.   Our abstract provides information on the preliminary safety data of SPVN06, our gene-agnostic investigational gene therapy, which shows that no severe adverse events were reported in the initial groups of patients who received treatment. Most adverse events were procedural, transient, and quickly resolved.   This achievement demonstrates our commitment to advancing genomic medicines and represents a step forward in our mission to lead the way in bringing new hope to millions affected by retinal diseases, for which there are currently no viable treatments.   👁️ To learn more about our findings and the impact of our research, please read the full press release here: https://lnkd.in/dN4ZTSMY   #GenomicMedicines #Ophthalmology #GeneTherapy

If large biopharma clinical development programs are shifting away from COVID-19 and immuno-oncology, what areas are they focusing on? They are focusing on hot spots in oncology and a range of therapies across large population and rare diseases, including: ⚕️Cell and gene therapies ⚕️Antibody-drug conjugates (ADCs) ⚕️Radioligand The annual report dives into the specifics: https://lnkd.in/eNHKxEgv. #RandD

EMA recommended approval of First gene editing therapy to treat beta thalassemia and severe sickle cell disease - #Casgevy (exagamglogene autotemcel) #EMA recently recommended approval of the first medicine using #CRISPR/Cas9, a novel #gene-editing technology. Casgevy (exagamglogene autotemcel) is indicated for the treatment of #transfusion‑dependent beta #thalassemia and severe sickle cell disease in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate and a suitable donor is not available. This new therapy may free patients from the burden of frequent transfusions and painful vaso-occlusive crises that occur when sickled red blood cells block small blood vessels, and has the potential to significantly improve their quality of life.

Cell and Gene Therapies (CGT) hold the key to transforming healthcare for patients and the industry at large. And, with the potential to treat illnesses including cancers, ALS, and more, their demand is only growing. At Mass General, 400 researchers are dedicated to studying these treatments for which over 7,000 applications have been discovered and are contending for FDA-approval. For many health plans and employers, however, the exponential growth of CGTs and their high cost in the marketplace can mean an overhaul of pharmacy benefits. From cost containment to risk management, ELMCRx Solutions’ specialty Rx services equip clients with the analytical and clinical expertise to navigate CGT’s rapidly changing effects on the pharmacy benefit industry.   Keep Learning at https://lnkd.in/eWjbR7QV #CGT #CellAndGeneTherapy #SpecialtyPharmacy

Cell and gene therapies (CGTs) present significant potential across a wide range of diseases including cancer, genetic disorders, and autoimmune conditions, if you want to know more about this topic please read Alira Health withe paper , in the link below. #cgt #cellandgenetherapyd

"Commercializing CGTs in non-orphan indications will be fundamentally different than the experience to date." If you want to learn more on the key commercial challenges and considerations check out this Alira Health white paper: https://lnkd.in/exyD4K2x #CGT #advancedtherapies #ATMP #cellandgenetherapy