Lundbeckfonden / Lundbeck Foundation’s Post

Induced pluripotent stem cells (iPSCs) have revolutionized the way we explore human disease mechanisms and develop therapies. However, challenges like genetic instability, heterogeneity, and quality control remain a concern. At Syngene, we address these challenges by generating high-quality iPSCs followed by extensive characterization to ensure the quality of the final clones and their derivatives.   With our deep experience and expertise in this area, we provide comprehensive solutions to generate feeder-free and viral integration-free iPSC clones specifically designed for cellular assays and screening. Our end-to-end services include iPSC reprogramming, characterization, CRISPR and advanced nuclease-based gene editing, differentiation of iPSCs into multiple lineages, and in vitro assays using iPSC derivatives or primary cells.   Furthermore, our advanced CAR-T platform complements our iPSC capabilities, offering a comprehensive suite of services for cell and gene therapy research. Partner with Syngene to accelerate your drug discovery. For more information, visit the link in comments section 🔗   #iPSCs #DrugDiscovery #Syngene #discoveryservices

🔍 𝐄𝐱𝐩𝐥𝐨𝐫𝐢𝐧𝐠 𝐭𝐡𝐞 𝐅𝐫𝐨𝐧𝐭𝐢𝐞𝐫𝐬 𝐨𝐟 𝐑𝐍𝐀 𝐃𝐞𝐥𝐢𝐯𝐞𝐫𝐲 𝐰𝐢𝐭𝐡 𝐋𝐢𝐩𝐢𝐝 𝐍𝐚𝐧𝐨𝐩𝐚𝐫𝐭𝐢𝐜𝐥𝐞𝐬 🧬 Exciting developments in nanomedicine continue to shape our approach to disease treatment, particularly through the use of lipid nanoparticles (LNPs) in RNA delivery. It's crucial to understand the foundational aspects that make LNPs an effective tool in gene therapy and beyond. This insightful review dives deep into the mechanics and applications of LNPs, highlighting the meticulous process of lipid component selection and encapsulation strategies that form the foundation of LNP development. It further elaborates on the importance of characterizing and optimizing these particles to ensure effective delivery, while also detailing the transition from in vitro studies to human clinical trials. Whether you're a seasoned researcher or new to the field of RNA therapeutics, this review offers a comprehensive look at the potential and challenges associated with LNPs, making it a crucial read for anyone involved in the development of new treatments. Attached is the full publication for those interested in a more detailed read. 🎯 𝐊𝐞𝐲 𝐓𝐚𝐤𝐞-𝐚𝐰𝐚𝐲𝐬: • 𝐵𝑖𝑜𝑐𝑜𝑚𝑝𝑎𝑡𝑖𝑏𝑖𝑙𝑖𝑡𝑦 𝑎𝑛𝑑 𝑉𝑒𝑟𝑠𝑎𝑡𝑖𝑙𝑖𝑡𝑦: LNPs provide a safe and adaptable platform for RNA delivery, crucial for treatments across various diseases. • 𝐶𝑟𝑢𝑐𝑖𝑎𝑙 𝑆𝑡𝑎𝑔𝑒𝑠 𝑜𝑓 𝐷𝑒𝑣𝑒𝑙𝑜𝑝𝑚𝑒𝑛𝑡: From the formulation of LNPs to their characterization and in vivo evaluation. • 𝑃𝑟𝑜𝑚𝑖𝑠𝑖𝑛𝑔 𝐴𝑝𝑝𝑙𝑖𝑐𝑎𝑡𝑖𝑜𝑛𝑠: Beyond their success in COVID-19 vaccines, LNPs are being explored for a wide range of therapies, including cancer immunotherapies and genetic disorders. • 𝐹𝑢𝑡𝑢𝑟𝑒 𝑂𝑢𝑡𝑙𝑜𝑜𝑘: The field is set to expand with ongoing research into enhanced delivery mechanisms and broader therapeutic applications. #Nanomedicine #RNATherapeutics #LipidNanoparticles #GeneTherapy #Biotechnology #DrugDelivery #mRNA #InnovativeMedicine #HealthcareInnovation #ScientificResearch Yutian Ma Shiyao Li Xin Lin & Yupeng Chen

🌟 Company Spotlight: Ascidian Therapeutics 🌟 Led by Michael Ehlers At the forefront of #RNA therapeutics, Ascidian Therapeutics is revolutionizing the treatment of genetic diseases. Inspired by the unique biology of sea squirts (ascidians), the company has developed a pioneering RNA exon editing platform that offers a novel approach to gene therapy. 🔬 Innovative Technology: Ascidian’s platform enables precise post-transcriptional editing of RNA, allowing for the correction of genetic mutations at the exon level. This technology ensures the production of full-length, functional proteins, addressing diseases that current gene editing technologies cannot. 🧬 Clinical Impact: The company’s lead program, #ACDN-01, targets Stargardt disease and other #ABCA4 #retinopathies. Currently in Phase 1/2 clinical trials, this program aims to provide a one-time treatment that could significantly improve patients’ lives. 🤝 Strategic Partnerships: Ascidian has also entered a groundbreaking collaboration with Roche to develop RNA exon editing therapeutics for neurological diseases. This partnership combines Ascidian’s innovative technology with Roche’s advanced #CNS delivery capabilities. 🌍 Vision: By harnessing the power of RNA biology, Ascidian Therapeutics is committed to delivering life-changing treatments to patients worldwide. Their work represents a significant leap forward in the field of genetic medicine. #Fierce15 #FiercePharma #Ascidian #Roche #CRO #ContractResearch #clinicaldata

Exciting News from EditCo Bio, Inc.! 🌟 I am thrilled to announce the launch of our groundbreaking Knockout CD4+ T-cell Pools! This latest innovation is set to transform cancer and autoimmune research, offering unprecedented precision in CRISPR-mediated gene editing. 🔬 Why is this a game-changer? 🚀 Rapid Results: 7-day protocol with knockout efficiencies beyond 80%. This enables us to deliver these edited pools to researchers in less than three weeks, 💪Dependable Performance: Our CD4+ T-cell Pools maintain over 90% viability post-editing and post-cryopreservation and achieve more than 60-fold expansion after 14 days in culture. 🎯Optimal Functionality: edited CD4+ T-cell Pools are highly functional We are proud to be at the forefront of this scientific breakthrough, offering tools that empower researchers to achieve faster, more reliable results. Congratulations to all the team that has made it possible Alexander Brown, Piazza Paolo and Daniel Orozco 🔗 Learn more about how our Knockout CD4+ T-cell Pools are revolutionizing the field: https://lnkd.in/dcBcBhHy #EditCoBio #CRISPR #GeneEditing #Immunology #CancerResearch #AutoimmuneResearch #Innovation #ScienceNews https://lnkd.in/gNmVMW-U

📢 The Danish Pharma Giant Novo Nordisk, is taking control of a hemophilia program from 2SeventyBio. ✨ Looking to diversify its portfolio with Cell and Gene therapy assets Novo Nordisk acquired 2seventy bio’s Hemophilia A program and gene editing technology for autoimmune diseases in a deal worth up to $40 million. The 2seventy bio team involved in the program will join Novo Nordisk to advance the technology. Meanwhile, 2seventy bio will focus exclusively on developing and commercializing Abecma, a CAR-T cell therapy for multiple myeloma, in collaboration with Bristol Myers Squibb. ✨ The deal will also give Novo rights to 2seventy’s in vivo gene-editing technology—dubbed megaTAL—outside of oncology and gene editing for cell therapies of immune cells for the treatment of autoimmune disease, #genetherapy #2seventybio #Novonordisk #HemophiliaA #Geneediting #M&A #Autoimmune

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Edgewood's $20M Series A; Nanoscope to pursue FDA approval for eye gene therapy later this year: Plus, news about bluebird bio, Q32 Bio and Aquestive Therapeutics: Edgewood Oncology’s $20M Series A: The Brookline, MA-based biotech plans to use the money to support Phase 2a studies in acute myeloid leukemia and targeted breast cancer. It’s financed by Alta Partners. — Kyle LaHucik Nanoscope to seek FDA approval for eye gene therapy: The Dallas-based biotech said its mutation-agnostic gene therapy MCO-010 passed a Phase 2b study in retinitis pigmentosa, significantly improving vision measured on an eye chart at one year at both the high and low doses compared to a sham control. It expects to submit an application for MCO-010 to the FDA in the second half of this year. — Lei Lei Wu Bluebird bio projects 85 to 105 patient starts for its gene therapies in 2024: So far, the company has reported seven patient starts for beta thalassemia treatment Zynteglo and two for cerebral adrenoleukodystrophy therapy Skysona. Bluebird said that the first patient start for its sickle cell disease therapy is “imminent.” In its Tuesday earnings announcement, the company also shared that it signed an outcomes-based agreement with Michigan Medicaid, marking its first such agreement with a government payer. — Lei Lei Wu Q32 Bio begins trading: The mid-stage immune biotech completed its reverse merger with Homology Medicines and announced a $42 million private placement. Its shares $QTTB were down about 4% on Tuesday morning, its first day of trading on the Nasdaq. — Kyle LaHucik Aquestive Therapeutics closes $75M offering: The New Jersey biotech, which is developing an oral film to deliver medications, raised the money via a sale of 16.6 million shares at $4.50 apiece. — Kyle LaHucik #lucidquest #genetherapy #celltherapy

As we reflect on 2023, it's clear that it has been a landmark year for CCRM Nordic and the field of cell and gene therapy. After securing significant financing early in the year, we have in just six months since our inception expanded our dedicated team to around ten members. Many significant milestones have been reached in the ATMP field during 2023, including the approval of several therapies, including the first CRISPR-based therapy and the first cell therapy for type 1 diabetes. Closer to home, Skånes universitetssjukhus (Skåne University Hospital) treated the first patient in the Nordics with Libmeldy, a live-saving gene therapy used to treat children with metachromatic leukodystrophy developed by Orchard Therapeutics. The Swedish biotech landscape has faced some challenges, however notable investments have been made with for example Anocca who raised SEK 400 million (~USD 40 million) for the development of next-generation cell therapies for cancer. We look forward to seeing them excel in 2024 and beyond! As we gear up for 2024, we are not just celebrating our achievements but also the groundbreaking advancements in our field. These strides are not just milestones but stepping stones toward a future where cell and gene therapies transform lives globally. #CCRMNordic #ATMP #LifeSciences #Innovation #HealthcareInnovation #GoCo

Gene&Cell Therapy >> Edgewood's $20M Series A; Nanoscope to pursue FDA approval for eye gene therapy later this year: Plus, news about bluebird bio, Q32 Bio and Aquestive Therapeutics: Edgewood Oncology’s $20M Series A: The Brookline, MA-based biotech plans to use the money to support Phase 2a studies in acute myeloid leukemia and targeted breast cancer. It’s financed by Alta Partners. — Kyle LaHucik Nanoscope to seek FDA approval for eye gene therapy: The Dallas-based biotech said its mutation-agnostic gene therapy MCO-010 passed a Phase 2b study in retinitis pigmentosa, significantly improving vision measured on an eye chart at one year at both the high and low doses compared to a sham control. It expects to submit an application for MCO-010 to the FDA in the second half of this year. — Lei Lei Wu Bluebird bio projects 85 to 105 patient starts for its gene therapies in 2024: So far, the company has reported seven patient starts for beta thalassemia treatment Zynteglo and two for cerebral adrenoleukodystrophy therapy Skysona. Bluebird said that the first patient start for its sickle cell disease therapy is “imminent.” In its Tuesday earnings announcement, the company also shared that it signed an outcomes-based agreement with Michigan Medicaid, marking its first such agreement with a government payer. — Lei Lei Wu Q32 Bio begins trading: The mid-stage immune biotech completed its reverse merger with Homology Medicines and announced a $42 million private placement. Its shares $QTTB were down about 4% on Tuesday morning, its first day of trading on the Nasdaq. — Kyle LaHucik Aquestive Therapeutics closes $75M offering: The New Jersey biotech, which is developing an oral film to deliver medications, raised the money via a sale of 16.6 million shares at $4.50 apiece. — Kyle LaHucik #lucidquest #genetherapy #celltherapy

RNA-editing therapies pick up steam The first few treatments using RNA editing have entered clinical trials or have gained approval to do so, raising hopes for safer and more flexible gene-editing therapies. One treatment involves swapping a single base to correct a mutation that causes alpha-1 antitrypsin deficiency (AATD), which can damage the lungs and the liver. Another changes thousands of genetic letters in anRNA molecule at once to treat the group of mutations that cause Stargardt disease. Unlike CRISPR genome editing, RNA editing doesn’t change genes. Nor does it introduce permanent changes, because RNA molecules are transient. Both characteristics could cut the risks associated with genome editing, but could mean treatments have less lasting effects. Gene Edit Biolab CRISPR genome editing by IDT CRISPR Therapeutics CRISPR Medicine News CRISPR-Cas9

What's next for CRISPR in 2024, and what are its main challenges coming into the New Year? Jasper Morley discusses below: “Challenges in this space include how to develop a fair pricing strategy, the logistics of offering broad access to the most needy populations in underserved markets (such as sickle cell patients in Africa), and potential unfamiliar adverse events associated with this novel modality of therapy. Vertex and CRISPR’s Casgevy will run $2.2 million for a one-time treatment for both SCD and TDT. That sticker price is in line with other novel gene therapies, such as bluebird bio’s Lyfgenia, which was also approved by the FDA for SCD and will cost $3.1 million. Yet one study of SCD patients found the total cost of disease over a patient’s lifetime was over $5 million." Read more below: