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𝐈𝐧𝐭𝐞𝐥𝐥𝐢𝐚 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐏𝐮𝐛𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧 𝐨𝐟 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐈𝐧𝐭𝐞𝐫𝐢𝐦 𝐏𝐡𝐚𝐬𝐞 1 𝐃𝐚𝐭𝐚 𝐟𝐨𝐫 𝐍𝐓𝐋𝐀-2002 𝐢𝐧 𝐇𝐞𝐫𝐞𝐝𝐢𝐭𝐚𝐫𝐲 𝐀𝐧𝐠𝐢𝐨𝐞𝐝𝐞𝐦𝐚 Intellia Therapeutics, Inc. has shared interim clinical results from the Phase 1 portion of the ongoing Phase 1/2 trial of NTLA-2002 in hereditary angioedema (HAE). NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for HAE, and the results were were published online in the New England Journal of Medicine. https://lnkd.in/eDPEHfDJ #crisprmedicine #crisprmedicinenews #crispr #geneediting #crisprtrial #raredisease

#Celltherapies and #genetherapies hold enormous potential to transform how we address traditionally difficult-to-treat diseases. Unlocking this potential, however, has proven challenging due to heterogeneity in drug response across patients, as well as potential adverse events related to delivery modalities. In this #webinar, we will discuss: 📈 How small molecule biomarkers can provide dynamic measures of target engagement, drug response, and drug-induced toxicity 🔎 How nontargeted, high throughput mass spectrometry enables discovery of the most biologically relevant biomarkers in circulation that associate with therapeutic efficacy and safety 🧬 A framework for when and how small molecule biomarkers can be applied across the phases of cell and gene therapy development to optimize and advance these personalized treatments Register to join us today!: https://lnkd.in/e5nxp8N3 Hosted by American Association of Pharmaceutical Scientists (AAPS) | @aapscomms

Anticodon-Engineered tRNA (ACE-tRNA) Therapy for Hemophilia A Nonsense mutations in the critical coagulation protein Factor VIII (FVIII) cause severe hemophilia A. Researchers at hC Bioscience, Inc. have reported the development of anticodon engineered tRNAs (ACE-tRNAs) designed to read through nonsense mutations in FVIII mRNA by inserting the appropriate amino acid, thereby restoring full-length protein expression. A DNA construct encoding ACE-tRNAs targeting TGA stop codons was encapsulated in lipid nanoparticles (LNPs) and tested both in vitro and in vivo. Results showed that LNP-delivered ACE-tRNAs restored full-length FVIII expression in a dose-dependent manner, with the protein being biologically active as evidenced by coagulation assays. In mice, LNP-encapsulated ACE-tRNAs were effectively delivered to liver endothelial cells, the natural production site of FVIII, demonstrating functional rescue of premature termination codon mutations. These findings highlight the potential of ACE-tRNAs as a therapeutic option for severe hemophilia A by enabling the production of functional, full-length FVIII. Other methods, such as recombinant FVIII therapy, AAV-mediated gene therapy, and monoclonal antibody bypass therapy, do not restore normal FVIII levels. https://lnkd.in/gjdqWeAq? #genetherapy #hemophilia #tRNA #AAV #hemophiliaA Suchul Jang, Alan Leggett, Stefania Lenna, Deena Silton, M.S. Ruan Zhang, Sara Evke, Ph.D., Sagi Ravid, Lingyue Yan, Conlin O'Neil, Chris Katanski, Philip McGilvray, Monika Tasak, Ph.D., Gautam Goel, Leslie J Williams, Jose Lora

Tomorrow, the FDA will be reviewing Vertex Pharmaceuticals’ CRISPR-based gene therapy, Exa-cel, which is currently in clinical trials for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) 🧬🔬 To learn more about this development, read my post on Goodwin’s Big Molecule Watch blog here: [https://lnkd.in/gEUjdHTp] #CRISPR #genetherapy #medicalresearch #innovation #FDAreview #VertexPharmaceuticals #ExaCel #sicklecelldisease #thalassemia #biotech #healthcare #science #medicaladvancements

Intellia Therapeutics, Inc. has announced positive long-term data from the Phase 1 portion of their ongoing Phase 1/2 study of NTLA-2002. This single-dose in vivo CRISPR-based gene editing therapy has led to 98% mean reduction in monthly attacks for patients suffering from hereditary angioedema (HAE). And, 8 out of 10 patients remained attack-free following the 16-week observation period. These promising results indicate that NTLA-2002 could change the lives of people afflicted with HAE. #genetherapy #CRISPR https://lnkd.in/evkqTURF

#PlasmidDNA CGT (Cell and Gene Therapy) is considered one of the important industry for future medical research. It offers the possibility of providing cures for cancer, genetic diseases, and rare disease patients. In recent years, the research and development pipeline for CGT has been growing rapidly. According to the statistics, there are a total of 5,814 CGT research and development projects globally, with 4,881 of them being actively in development. The outlook for the commercialization of CGT products is also quite optimistic, with a total of 41 CGT products having been launched between 2015 and 2022. Plasmid DNA is the highest-cost component in CGT production, with GMP-grade plasmids priced as high as $10,000 to $30,000 per gram. Most downstream CGT companies choose to directly purchase ready-made plasmids and collaborate with CDMO companies that offer fast production, high yields, and excellent quality. Some representative CDMO companies in China include GenScript, Wuxi AppTec, Obio, and WuXi Biologics, among others. The key to further reducing costs while ensuring plasmid quality lies in domestic production substitutes and process optimization. The CGT industry is transitioning from a focus on customized therapies for blood cancers, rare diseases, and specialized applications towards more generalized treatments for solid tumors and common diseases. Some related products have already entered Phase II clinical trials. As the scope of indications broadens and more universal CGT products become available, the market demand is expected to increase, which should lead to further cost reduction in the large-scale GMP-grade plasmid production.

Cerba Research is answering the call for CAR-T patient monitoring solutions by constantly innovating and investigating new technologies and modalities for patient safety in clinical trials. The cell and gene therapy (CGT) clinical trial landscape in general and CAR-T cell clinical trials in particular are a special focus for the FDA, EMA, and other regulatory agencies. The whole industry is thus aware of the recent FDA safety investigation and requirements for labeling CAR therapy products. Read the article: https://ow.ly/czkJ50RY47z. #CerbaResearch #FDAregulations #CAR-T #RegulatoryCompliance #ClinicalResearch #ClinicalTrialSafety

Cerba Research is answering the call for CAR-T patient monitoring solutions by constantly innovating and investigating new technologies and modalities for patient safety in clinical trials. The cell and gene therapy (CGT) clinical trial landscape in general and CAR-T cell clinical trials in particular are a special focus for the FDA, EMA, and other regulatory agencies. The whole industry is thus aware of the recent FDA safety investigation and requirements for labeling CAR therapy products. Read the article: https://ow.ly/h5qe50RUiWE. #CerbaResearch #FDAregulations #CAR-T #RegulatoryCompliance #ClinicalResearch #ClinicalTrialSafety

Really fascinating research brief on the success of Cell and Gene Therapies getting through trial phases up to launch. #CART #Pharma #Pharmacology #oncology #cellandgenetherapy "CAR-T/TCR therapies for blood cancers are 3X as likely to be approved when entering Phase I as the average oncology drug and over 2X as likely as the average hematological oncology drug. Orphan gene therapies are 2 – 3.5X as likely to be approved when entering Phase I as the average drug in clinical trials, outperforming in every clinical development phase. Orphan gene therapies are 2X as likely to be approved when entering Phase I as the average drug in similar therapeutic areas*, outperforming in every phase."

Lexeo Therapeutics stock is down almost 30% this afternoon on promising, but early data for its cardiomyopathy treatment for Friedreich's ataxia patients. The gene therapy reduced markers of the disease, including left ventricular mass index, or LVMI, and troponin I. Elevated LVMI is tied to cardiovascular morbidity and mortality. Troponin I is an enzyme that, when increased, can indicate heart muscle injury. Leerink Partners analyst Mani F. sees a "compelling" bull argument for Lexeo's approach. "Progressive LVMI improvement through 18 months and high proportion of patients achieving greater than 10% LVMI improvement offer enough evidence of (effectiveness) for Lexeo to design a pivotal study with a reasonable (number of patients)," he said in a report. Lexeo is currently enrolling more patients in another cohort. It expects to have additional results this fall. https://lnkd.in/g3HDp9tZ