Apertura Gene Therapy’s Post

🔦 Spotlight on our #MultiomicsSolutions: #sCircle   Did you know that besides single cell transcriptomics, Singleron has a range of multi-omics solutions? Today we would like to put sCircle in the spotlight. Our solution for single cell sequencing of the entire transcriptome plus sensitive full length TCR or BCR!     Full length single cell TCR or BCR sequencing has previously shown its incredible potential, for instance in the field of CAR-T cell treatment. Our sCircle can be used to identify novel CAR-T target, to monitor the function and effectiveness of CAR-T therapy and much more!   How does it work?: Using our microwell chip, single cells are separated, lysed and their mRNA is captured on barcoding beads using poly(dT), including the full length transcripts of TCR or BCR. After reverse transcription, the now barcoded immunoreceptors are circularized and the cDNA is used for a 3-round nested-PCR enrichment.   ⚕ Applications: ·      Immune microenvironment ·      Autoimmune Diseases ·      Transplantation ·      Therapeutics Development   Advantages: High capture efficiency & specificity: capture of TCR/BCR prior to amplification! High throughput: up to 10k cells in a single run. High mapping rate: increased number of cells with full length V(D)J sequences à high specificity and sensitivity. Comprehensive: transcriptome with full length TCR or BCR.   And of course, automation is possible using our Matrix NEO! …Pssst! For the quick deciders: this week our Matrix NEO is still in #promotion!   More info?: https://lnkd.in/eKPH3sAu #singlecell #highthroughput #mRNAsequencing #precisionmedicine

PANDA trial : small trial with interesting approach Evaluating of neoadjuvant Atezolizumab in non metastatic gastric and GOJ adencarcinoma Phase II small number (21 patients) pCR was seen in 45% What is the interesting difference in this trial ? 🧐 The approach: Giving one cycle monotherapy of Atezo before start the 4 cycles of chemotherapy + Atezo What is the rationale for this approach? They want to to study the tumour micro environment TME after immunotherapy 1) providing the opportunity to specifically capture the contribution of anti-PD-L1 and the subsequent combination with chemotherapy 2) they found that increasing in CD8+ TCI and immune-related gene expression following atezolizumab monotherapy were most evident in responders, indicating that analysis of the TME following the initial cycle of ICB may provide valuable predictive insights about the efficacy of this treatment strategy in individual patients. Although small trial however it touched the base of personalalization and individualisation of using of ICIs depending on study of tumour micro environment (TME) Deeper looking to TME is a convinced approach and could be used in other malignancies like TNBC when ICIs are studied in neoadjuvant setting

In this publication, we showed that the #PDGFD gene is reversibly demethylated in gemcitabine-resistant #PDAC cells, making PDGF signaling a possible target for overcoming gemcitabine resistance. However, clinical trials of TKIs failed to improve the outcome in PDAC patients. What is the disconnect here, difficulty in delivering #TKIs into the stroma-dense PDAC tissues? https://lnkd.in/gDk5X5X3

Li 53: Our recent Publication: Thorough examination of the potential biological implications of the cuproptosis-related gene LIPT2 in the prognosis and immunotherapy in pan-cancer https://lnkd.in/gppdbCNU

https://lnkd.in/dKJ8Zt75 Anti-tumor effects of bemiparin in HepG2 and MIA PaCa-2 cells İhsan Alur et al. Gene. 2016.

𝐈𝐧 𝐜𝐚𝐬𝐞 𝐲𝐨𝐮 𝐦𝐢𝐬𝐬𝐞𝐝 𝐢𝐭, 𝐡𝐞𝐫𝐞 𝐚𝐫𝐞 𝐭𝐡𝐞 𝐥𝐚𝐭𝐞𝐬𝐭 𝐜𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐮𝐩𝐝𝐚𝐭𝐞𝐬 𝐟𝐫𝐨𝐦 𝐭𝐡𝐞 𝐠𝐞𝐧𝐞-𝐞𝐝𝐢𝐭𝐢𝐧𝐠 𝐟𝐢𝐞𝐥𝐝! ⛳ Intellia Therapeutics, Inc. obtains UK clearance to begin gene insertion trial in alpha-1 antitrypsin deficiency (https://shorturl.at/Ivwjz) 💉 Beam Therapeutics doses first patient with BEAM-302 in alpha-1 antitrypsin deficiency trial 📈 Intellia Therapeutics shares first-ever redosing data for a gene-editing therapy 🩸Editas Medicine announces new clinical data from 18 patients treated with sickle cell disease therapeutic candidate 💉 KSQ Therapeutics, Inc. announces dosing of the first patient with its CRISPR-edited TIL therapy for cancer https://lnkd.in/eVbFU_rz #crisprmedicinenews #crisprmedicine #crisprtrial #geneediting #clinicalnews #crispr #baseediting

4 important approvals by FDA this week: - expanded approval for Sarepta Therapeutics' #Elevidys, a gene therapy to treat ambulatory and non-ambulatory individuals aged four and older suffering from Duchenne muscular dystrophy (DMD). Of note, the primary endpoint of improving motor function failed, but the secondary endpoints were met.https://https://lnkd.in/eyyS8XNh. - Merck's #Keytruda was authorized in combination with chemotherapy followed by the PD-1 inhibitor as a monotherapy in patients suffering from primary advanced or recurrent endometrial cancer (40th U.S. indication approved).https://lnkd.in/eXZSnayr. - accelerated approval for GENFIT / Ipsen 's #Iqirvo (elafibranor) as a first-in-class treatment for primary biliary cholangitis (PBC). a rare liver disease. https://lnkd.in/e725xksQ. - approval for AbbVie's #Skyrizi (anti-IL23 mA ) to treat moderately to severely active ulcerative colitis in adults. https://lnkd.in/erC6Ga2z

Autologous cell therapy uses a patient’s own genetically modified immune cells to treat disease. In this personalized form of therapy, cells are removed from a patient, modified to achieve a specific therapeutic effect, and then input back into the patient. When creating autologous therapies, such as chimeric antigen receptor (CAR) T and T cell receptor (TCR) cell therapies, we combine target selection, cell and vector engineering, and gene editing expertise to modify cells and enhance performance.

I am thrilled to see that the #2024_Nobel_Prize_in_Physiology_Medicine has been awarded to the discoverers of the role of microRNAs (miRNAs) in regulating gene expression post-transcription. My doctoral thesis focused on the role of cellular microRNAs in inhibiting or suppressing HCV replication in vitro, and I am glad I chose this cutting-edge approach at the time. It’s incredibly rewarding to see this method now being recognized with a Nobel Prize. I would also like to reference five of my own related articles here. - Liver micro-RNAs and their role in different HCV genotype infections by Shafaati et al., 2018. - Luciferase Assay for Demonstrate the Competence of Selective MicroRNA of let-7b in Suppressing HCV Replication by Shafaati et al., 2020. - Downregulation of hepatitis C virus replication by miR‐196a using lentiviral vectors by Shafaati et al., 2021. - MicroRNA let-7b inhibits hepatitis C virus and induces apoptosis in human hepatoma cells by Shafaati et al., 2022. - Revolutionizing HCV Therapy: microRNA Approaches in New Era by Shafaati et al., 2024.