Lexeo Therapeutics’ Post

Arrhythmogenic cardiomyopathy (ACM), a genetic heart disease, is characterized by changes to the heart muscle, which affect its electrical activity. This condition leads to scarring, heart dysfunction and, in severe cases, sudden death. In the US, an estimated 130,000 individuals are affected, and more than half having a genetic cause, primarily associated with mutations in the PKP2 gene.     At Lexeo, we are developing a number of disease-modifying gene therapy candidates to treat cardiovascular diseases with significant unmet need. Our investigational AAV-based gene therapy LX2020, is being evaluated to deliver a fully functional PKP2 gene to heart muscle for the treatment of PKP2-ACM.    Learn more about our AAV-based gene therapy candidate, LX2020:   https://lnkd.in/gNWDX9aQ  

To commemorate the contributions of Mr. Frank Schnabel, the founder of the World Federation of Hemophilia, April 17 has been designated as 𝐖𝐨𝐫𝐥𝐝 𝐇𝐞𝐦𝐨𝐩𝐡𝐢𝐥𝐢𝐚 𝐃𝐚𝐲 since 1989. To learn more about World Hemophilia Day 2024, visit the World Federation of Hemophilia website: https://lnkd.in/eG4VKVAj   To enable hemophilia research, #GemPharmatech has developed a B6-F8-KO hemophilia A mouse model by knocking out certain exons of the C57BL/6JGpt FVIII gene, which causes a frameshift mutation that results in a FVIII protein deficit. This model can simulate the pathophysiology and main symptoms of hemophilia. It can also be used to evaluate the efficacy and safety of gene therapies and screening for other types of treatments for hemophilia A. For more information on this model, visit our hemophilia A model page: https://lnkd.in/gcAqtnDU Currently, we also have an F9-KO mouse model associated with hemophilia B under development. #WorldHemophiliaDay #mousemodel #Hemophilia #hemophiliaA #knockout #biotech

Hemophilia, a genetic disorder primarily affecting males, is characterized by deficiencies in clotting factors VIII (Hemophilia A) or IX (Hemophilia B). This deficiency impairs the blood's ability to clot and effectively seal wounds. Recent advances in treatment, including gene therapy, long-acting clotting factor replacements, and small molecule drugs, are revolutionizing care for hemophilia patients. Gene therapy shows exceptional promise by using viral vectors to introduce normal clotting factor genes into patients, potentially providing long-lasting therapeutic effects. Further research is targeting additional regulatory factors in the coagulation pathway, aiming to further enhance treatment outcomes. To aid in these advancements, we have carefully curated a list of over 20 essential targets and related reagents specifically for hemophilia research. https://bit.ly/3Q5Es4Z

🧬What are the differences between gene fusions and other types of oncogenic transformation? How are fusions identified and what are the implications for treatment? In this clinical topic update, pathologist Prof. Fernando López-Ríos gives an overview of gene fusions in solid tumours, methods for identification and the different tumour types harbouring gene fusions. He highlights the need to search for actionable fusions and the implications for treatment. Clinical takeaways - Several fusions are now actionable in solid tumours - Most of them were initially studied in NSCLC (ALK, ROS1 and RET), but many are now being developed in a tumour-agnostic manner (NTRK, RET, FGFR) - The gold standard for detecting them is RNA-based NGS, but IHC, FISH and real-time PCR can also be used Visit https://ow.ly/viSt50QoUn1 to watch the video or download the free accompanying slide set. Alternatively, you can watch the video on YouTube: https://ow.ly/Ous950QoUn2

🧬What are the differences between gene fusions and other types of oncogenic transformation? How are fusions identified and what are the implications for treatment? In this clinical topic update, pathologist Prof. Fernando López-Ríos gives an overview of gene fusions in solid tumours, methods for identification and the different tumour types harbouring gene fusions. He highlights the need to search for actionable fusions and the implications for treatment. Clinical takeaways - Several fusions are now actionable in solid tumours - Most of them were initially studied in NSCLC (ALK, ROS1 and RET), but many are now being developed in a tumour-agnostic manner (NTRK, RET, FGFR) - The gold standard for detecting them is RNA-based NGS, but IHC, FISH and real-time PCR can also be used Visit https://ow.ly/rY6w50QkhpB to watch the video or download the free accompanying slide set. Alternatively, you can watch the video on YouTube: https://ow.ly/G8hM50QkhpA

Immune recognition of therapeutic cells is a significant barrier for developing cell therapies. While B2M and CIITA, which regulate HLA -I and HLA-II expression, respectively, are key players in the immune response, additional factors are involved. A new animal study from the University of Arizona (https://hubs.ly/Q02qMLYD0) identifies factors that regulate NK cells, phagocytosis, and complement that may be significant for the immune response to hESCs, in addition to B2M and CIITA. These factors represent new targets for gene editing of cell therapies to reduce immune rejections. Find out how REPROCELL can help with your gene editing program: https://hubs.ly/Q02qMV6q0

Sacha, 8 years old, diagnosed with Duchenne muscular dystrophy, has benefited from gene therapy developed by Genethon, which is currently in the clinical trial phase. Discover the exceptional testimony of his parents on the occasion of World Duchenne Awareness Day! 💛 🌐 World Duchenne Awareness Day is an opportunity to observe the acceleration in the development of innovative therapies for this neuromuscular disease, which is the most common in children. Gene therapy is very promising. Genethon, the AFM-Téléthon laboratory, is currently developing a drug candidate that has demonstrated its effectiveness in the first patients treated. These advancements mark a major milestone in the development of the treatment, as Genethon, in collaboration with the European Medicines Agency, prepares the final evaluation phase of the drug candidate. More details 👉 https://lnkd.in/eaV_kuzy #WDAD2024

#Commentary now available! Comparative analysis of #nucleicacid delivery systems for #genetherapy: assessing viral and non-viral approaches with emphasis on #extracellularvesicles The potential of #nucleicacidtherapeutics lies in their ability to address genetic disorders by directly targeting and manipulating the underlying genetic material. However, the translation of nucleic acid therapeutic concepts into effective clinical interventions faces challenges. This Commentary #article navigates the complexities associated with nucleic acid therapeutics, with a particular focus on gene therapy, and explores the recent emergence of extracellular vesicles as a potential solution to overcome the hurdles in nucleic acid delivery. Access below:

Single-cell omics and CRISPR technologies are two topics that are given plenty of attention in the current market, but haematology as a whole doesn't get quite the same level of consideration. The latest review on erythropoiesis gives an interesting overview on where we stand in the mechanisms of red blood cell formation. Aside from looking into findings around transcription factors and epigenetic modifications relating to erythropoiesis, the review highlights how this knowledge could result in novel approaches to rare diseases like Diamond-Blackfan anemia. Gene therapy for β-thalassemia is highlighted as one example of a potential way forwards, but as of February 2024 there are only 38 FDA approved CGT products on the market. This review gives me hope that innovations in single-cell omics and CRISPR technologies could mean more CGT products getting approval and seeing more haematology-focused businesses in the world. Do you see CGT as the future of biotech too? Are there any other therapeutic areas you'd want to see more focus on? Let's connect to talk about all things Omic!