Ascidian Therapeutics’ Post

🧬 Exciting Developments in Gene Editing! 🌐 Intellia Therapeutics, Inc. , founded by Jennifer Doudna, joins the ranks of gene editing biotechs undergoing strategic changes. The CRISPR company is streamlining operations, focusing on advancing its lead assets, NTLA-2001 and NTLA-2002, following a pause in exploratory research. 🚀💊 🌍 This move aligns with industry trends as several gene editing biotechs, including Beam Therapeutics, Editas Medicine, and CRISPR Therapeutics, underwent downsizing in the past year. It underscores the dynamic nature of the genetic medicine sector and the need for strategic recalibration. 🧬🔄 👩🔬 Intellia will concentrate on its phase 3 trials for NTLA-2001, targeting ATTR amyloidoses with cardiomyopathy, and initiate a phase 3 trial for NTLA-2001 in patients with ATTR amyloidosis with polyneuropathy. The race to address ATTR with cardiomyopathy presents an opportunity for Intellia amid recent developments in the field. 💡🏃♂️ 🌟 Meanwhile, Aera Therapeutics, founded by Feng Zhang, is another player in the genetic medicine arena making strategic adjustments. While Aera recently emerged from stealth with $193 million, it is now reshaping its workforce. 🔄💼 🌐 The genetic medicine landscape continues to evolve, showcasing the resilience and adaptability of biotech companies. The new year brings fresh challenges and opportunities for innovation in the genetic medicine sector. Stay tuned for more updates! 🧬🌈 #GeneEditing #CRISPR #BiotechInnovation #GeneticMedicine #HealthcareTrends #BiotechNews #InnovateForHealth 💉🔬

This paper in BioDrugs reviews the recent advances in preclinical studies demonstrating in vivo targeted gene insertion for therapeutic benefits, targeting somatic solid tissues through systemic delivery, and highlights hemophilia as a prominent disease model. #genetherapy #hemophilia #CRISPR #openaccess https://lnkd.in/ghg3Ecze

Are you "in tune" with the S12 guideline? The objective of this guideline is to provide harmonised recommendations for the conduct of nonclinical biodistribution (BD) studies in the development of gene therapy (GT) products. BD is the in vivo distribution, persistence, and clearance of a GT product at the site of administration and in target and non-target tissues, including biofluids (e.g., blood, cerebrospinal fluid, vitreous fluid). At TATAA Biocenter, we bring over two decades of expertise in extraction and assay design: - Sample extraction is crucial for biodistribution studies, and we test and optimize extraction efficiency for each tissue and target since target modifications, lengths, and secondary structures can significantly impact extraction efficiency. - Our qPCR and dPCR assays are designed and optimized to precisely identify the test item, whether it’s to detect a short target like siRNA or miRNA using our TATAA-invented Two-Tailed primer approach or to differentiate between transgenes and highly homologous endogenous sequences at the DNA or transcribed (mRNA) level. The assay optimization also includes tests to detect and mitigate matrix interference on the assay. Our approach provides reliable, high-quality data that supports robust scientific conclusions so reach out to further discuss! Read more in our blog, link below: https://lnkd.in/dJ9bfDY9 #biodistribution #research #medicine #drugdevelopment #shedding #proteomics #drugdevelopment #biopharma #pharma

Do you need help for biodistribution studies? Are you aware how at Tataa we can help you complying with S12 guidelines ? If you are interested, drop me a message and we can meet to discuss

🔬🧬 Once again, the pharmaceutical industry has topped its existing astronomical prices with Lenmeldy, an FDA-approved gene therapy for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), which has a wholesale acquisition price tag of $4.25 million. The one-time gene therapy treatment is derived from a patient’s hematopoietic stem cells (HSCs) that are genetically modified to include a functional copy of the arylsulfatase A (ARSA) gene and transplanted back into the patient. 🌟 #GeneTherapy #MLD #MedicalBreakthrough #FDAApproval #HealthcareInnovation #RareDisease #TreatmentOptions #HopefulFuture #MedicalResearch #ScienceAdvancements #BioTech #HealthTech #ClinicalTrials #MedicalScience #PatientCare

We are excited to share that our Director of Business Development, French Lewis, III will be attending the 2nd Gene Therapy Potency Assay Summit from 16th-18th July. With the prediction of up to 17 gene therapies potentially being approved in the US & EU in 2024, staying ahead of the curve is crucial. This summit is the perfect opportunity to deepen our understanding of the latest FDA guidelines, innovative potency assay approaches, and emerging technologies that are shaping the future of gene therapy. At DefiniGEN, we are committed to aiding researchers bring drugs and therapies to market by providing iPSC-derived human hepatocytes and disease models. By conducting drug screening and disease modelling on these phenotypically relevant cells, researchers can make crucial insights and select the right compounds/therapies going forward. For more information on our iPSC-derived human hepatocytes and disease models, check out our website: https://lnkd.in/eRVXmF4U #GeneTherapy #Biotech #DefiniGEN #liver #liverdisease #FDA #Innovation #Healthcare #Biopharma

Big news, as Genentech have recently announced a significant partnership with GenEdit to develop and commercialize nanoparticle-based gene therapies. This collaboration is projected to be valued at up to $644 million and has the potential to revolutionize the field of gene editing. The innovative use of nanoparticles in gene therapy holds great promise for addressing a wide range of genetic disorders and could significantly impact the future of medicine 👏🏼📊🔬 This strategic deal underscores Genentech's commitment to staying at the forefront of cutting-edge technologies and further solidifies their position as a key player in the biotech industry. By harnessing the power of nanoparticle-based gene therapy, Genentech are demonstrating their dedication to advancing medical innovation. Let's hope to see this newly formed partnership push the boundaries of what is possible in the realm of genetic medicine 💉💊 It will be interesting to see the future impact of this collaboration and the potential it holds for bringing about meaningful change in the lives of patients around the world - watch this space! 🚀🌍 #GeneTherapy #BiotechInnovation #MedicalBreakthroughs #Genentech #GenEdit #Biotechnology #Pharmaceuticals #ClinicalResearch #ClinicalTrials #LifeScience #ElixirAssociates #StaffingPartner

🚀 Exciting News Alert! 🚀 Today, Epitor Therapeutics proudly unveils a groundbreaking innovation: CasNano, an ultracompact Cas protein.   As the next frontier in genetic medicine unfolds, CRISPR/Cas technology is revolutionizing how we target specific genes and modulate their expression—without altering the DNA itself. However, to effectively administer these tools to diverse organs, we require epigenetic editors of smaller proportions.   Enter CasNano—an innovation of engineering, boasting a mere 399 amino acids in size. It may well be the smallest engineered Cas protein developed to date. At Epitor, we are honored to spearhead the development of these transformative tools, poised to catalyze innovation in genetic medicine.   Our mission? To pave the way for groundbreaking therapies that tackle a multitude of severe, hard-to-treat diseases head-on.   #EpitorTherapeutics #CasNano #GeneticMedicine #Innovation #DiseaseTherapies #CRISPRCas #Biotechnology https://lnkd.in/ekB5z7Xv

Thoughts on this? >> ASGCT 2024: Beverly Davidson Unlocks AAVs for Brain Gene Therapies - Genetic Engineering & Biotechnology News >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #healthcare #biotech #pharmaceutical #competitivemarketing

Which ionizable lipid can be used for DNA delivery? Are there any differences to RNA? Find out! 👇 Happy to share a new paper from our collaborators Claudia Lotter and Prof. Jörg Huwyler from Universität Basel. They compare different ionizable #lipids for #lipidnanoparticle mediated #DNA delivery. 💥 The highlights? 👉 DNA delivery via lipid nanoparticle (#LNP) formulations is underexplored. 👉 Our study combines DNA expression plasmids with clinically used ionizable lipids. 👉 In vivo #biodistribution and expression are not reliably predicted by physicochemical and in vitro characteristics. 👉 #Zebrafish were used as a vertebrate bridging model to extrapolate from in vitro to the in vivo situation. 👉 LNP formulations were identified, which lead to luciferase gene expression in wildtype mice. #pharma #science #drugdelivery