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🔔 Conference Early Bird Registrations are now Open! Join us in London on Oct 1st for the 1st Getting Cilia Moving Conference Bringing together the key opinion leaders, UK Government, UKRI accelerators, industry KOLs, investors, experts in regulation and the patients to work together to leverage the progress made in cystic fibrosis into primary ciliary dyskinesia. If you have an interest in #respiratory #rare diseases, #brochiecstasis or #genetherapy, join us. Key highlights: 🌟 Focus on translational medicine for genetic respiratory diseases, and lessons learned from Cystic Fibrosis  🌟 Engage with experts and stakeholders in genomics, translational medicine, genetic therapies 🌟 Networking, expert panels, patient perspective and more more Save over £600! Register by 30th July for Early bird pricing 👉 https://lnkd.in/ezN7RDuz Ethris ReCode Therapeutics Skip Therapeutics SpliSense Vertex Pharmaceuticals Insmed Incorporated Parion Sciences Simbec-Orion LifeArc Prime Medicine, Inc. AXON Moderna Alexion Pharmaceuticals, Inc. FibroGen, Inc. Grifols Eloxx Pharmaceuticals CRISPR Therapeutics Weatherden #rarediseases #genetherapy #biotech #mRNA #PCDresearch #geneandcelltherapy #advancedtherapies

🎥 Exciting developments with the HSD17B13 humanized mouse model! This model is advancing MASLD and RNAi research by targeting metabolic dysfunction related to liver diseases. By inhibiting HSD17B13, we may unlock new therapeutic approaches for NAFLD and NASH, especially with the rise of RNA therapies. Curious about the potential impact of the HSD17B13 model in research? Share your thoughts below! ✨ #mousemodel #genetherapy #HUGOGT #YouTube #scienceandtechnology #researchmatters #biotechnology #biotech

Bristol Myers Squibb partners with Prime Medicine, Inc. Medicine to advance #celltherapy in #immunology and #oncology. The #collaboration includes a $110 million upfront payment to develop optimized Prime Editor reagents for specific targets, utilizing PASSIGE technology. With potential milestones totaling $3.5 billion, Prime aims to extend its cash runway through the first half of 2026. This innovative approach combines Prime Editing with enzyme technology, ensuring precise genome modifications without double-stranded DNA breaks or off-target edits. Keith Gottesdiener, CEO of Prime Medicine, highlighted the benefits of PASSIGE at a recent industry event. #CellTherapy #Innovation #BiotechCollaboration #collaboration #advancedtherapies #tcell #biotech #biotechnology #lifescience #lifesciences #milestones #110mupfront #primeediting #enzymetechnology

🚀 Targeting Regulatory Genome RNAs to Reprogram Disease Cell States - The HAYA Way to Fight Disease   The 2024 Nobel Prize in Physiology and Medicine recognizes the importance of RNA as more than just a messenger, highlights its critical role as an important regulator of cells and their function.   🧬 At HAYA, our mission is clear: bring to patients’ transformative medicines targeting Regulatory Genome RNAs, capable to reprogram disease cells into healthy ones and with the potential to stop and reverse disease-associated processes like fibrosis. 💡 We invite you to learn more about HAYA in our profile article at Biopharma Dealmakers, where you will find: - Insights into regulatory genome RNAs and their key role in driving disease - Details about HAYA's unique platform and approach - Upcoming milestones and our latest achievements - Collaboration opportunities - partnering with biopharma companies with a complementary disease or modality expertise. ➡ https://lnkd.in/efUGws24 #RegulatoryGenome #Biotech #HealthcareInnovation #HAYA #FutureofHealthcare #ReprogramCellStates

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> 2seventy bio, Bristol Myers end late-stage Abecma study in multiple myeloma: 2seventy bio and its partner Bristol Myers Squibb said Wednesday that they are discontinuing enrollment in the Phase 3 KarMMa-9 study investigating Abecma in patients with newly diagnosed multiple myeloma. 2seventy CEO Chip Baird wrote in a statement that the decision was made after a “rigorous review of the business case for the KarMMa-9 study” and that there’s been a “greatly improved” treatment landscape in the newly diagnosed setting. Chip Baird In a separate release, Bristol Myers added there were enrollment difficulties as there were fewer eligible patients for the study due to “the evolution of more intense and prolonged induction therapies.” The trial has been open for over a year in 18 countries but only 10% of the study population has been accrued so far, the company added. The trial had a target of 618 patients, according to ClinicalTrials.gov. For its part, the trial discontinuation would help 2seventy break even next year. “Consistent with our focus on capital allocation and creating value for all stakeholders, we anticipate this decision will conserve over $80 million in near-term expenditures and accelerate our path to breakeven in 2025,” Baird added. 2seventy and Bristol Myers were evaluating the CAR-T therapy in the trial alongside lenalidomide maintenance versus lenalidomide maintenance alone in patients with newly diagnosed multiple myeloma who had a suboptimal response to an autologous stem cell transplant. The companies scored an expanded approval from the FDA for Abecma in April in the third-line setting, following its initial approval in fifth-line patients, despite concerns over a higher rate of death in early treatment among patients who took the drug compared to those who received standard regimens. “Abecma continues to show encouraging signs of growth with an expanded label in the third line and a differentiated safety profile,” Baird added. In its Tuesday announcement, 2seventy wrote that it expects 2024 third-quarter Abecma revenue growth in the US of around 30% from second-quarter revenue of $54 million. #lucidquest #genetherapy #celltherapy

Gene&Cell Therapy >> 2seventy bio, Bristol Myers end late-stage Abecma study in multiple myeloma: 2seventy bio and its partner Bristol Myers Squibb said Wednesday that they are discontinuing enrollment in the Phase 3 KarMMa-9 study investigating Abecma in patients with newly diagnosed multiple myeloma. 2seventy CEO Chip Baird wrote in a statement that the decision was made after a “rigorous review of the business case for the KarMMa-9 study” and that there’s been a “greatly improved” treatment landscape in the newly diagnosed setting. Chip Baird In a separate release, Bristol Myers added there were enrollment difficulties as there were fewer eligible patients for the study due to “the evolution of more intense and prolonged induction therapies.” The trial has been open for over a year in 18 countries but only 10% of the study population has been accrued so far, the company added. The trial had a target of 618 patients, according to ClinicalTrials.gov. For its part, the trial discontinuation would help 2seventy break even next year. “Consistent with our focus on capital allocation and creating value for all stakeholders, we anticipate this decision will conserve over $80 million in near-term expenditures and accelerate our path to breakeven in 2025,” Baird added. 2seventy and Bristol Myers were evaluating the CAR-T therapy in the trial alongside lenalidomide maintenance versus lenalidomide maintenance alone in patients with newly diagnosed multiple myeloma who had a suboptimal response to an autologous stem cell transplant. The companies scored an expanded approval from the FDA for Abecma in April in the third-line setting, following its initial approval in fifth-line patients, despite concerns over a higher rate of death in early treatment among patients who took the drug compared to those who received standard regimens. “Abecma continues to show encouraging signs of growth with an expanded label in the third line and a differentiated safety profile,” Baird added. In its Tuesday announcement, 2seventy wrote that it expects 2024 third-quarter Abecma revenue growth in the US of around 30% from second-quarter revenue of $54 million. #lucidquest #genetherapy #celltherapy

📰 This is a follow up post - congrats to the teams at Orchard Therapeutics on the FDA approval of #Lenmeldy (atidarsagene autotemcel) - known as #Libmeldy in European markets!   💡 The most striking statement from the announcement for me was "All children with pre-symptomatic late infantile MLD who were treated with Lenmeldy were alive at 6 years of age, compared to only 58% of children in the natural history group." 🗓 For a disease where previous treatment was almost fully limited to palliative care - this is fantastic news. The future of #cellandgenetherapy is bright, and it is saving human lives.   💉Lenmeldy is an autologous HSC-derived treatment conferring a functional ARSA gene to treated cells, which then attach and multiply within patient bone marrow conferring long term benefit in patients suffering from metachromatic leukodystrophy - a debilitating rare disease I covered in a recent post. FDA Announcement: https://lnkd.in/gqRugpRt   My previous post with more scientific background on the treatment and disease: https://lnkd.in/gZ9BeyXw     Hi, I'm Connor Wies,   I'm an industry professional with experience helping developers through early R&D, preclinical, and clinical stages of their research. I do this through a consultative value added approach where I learn as much as I can about your process and needs before giving any recommendations.   I also post semi-regularly about CGT developments. If you'd like to hear more: ➕ Follow my profile 🤝 Connect with me and schedule a chat 👇 Interact with my post   Sources in comments below:     #biotechnology #lifesciences #MLD #celltherapy #cellandgenetherapy #cellandgenetherapies #Libmeldy #LVV #lentiviral #metachromaticleukodystrophy #rarediseasetherapies #rarediseases      

Not all RNAs are created equal. tRNAs have some incredible therapeutic potential, but also unique challenges in terms of sequencing and chemical synthesis. In this Q&A with Drug Discovery World, Caroline Köhrer, Alltrna's Senior Vice President of Discovery Platform, breaks down the biology and how Alltrna is harnessing it to create tRNA therapeutics that can unify the treatment of diseases based on shared genetic mutations and accelerate rare disease drug development for millions. Read the article here: https://lnkd.in/egAY6_nC #RNA #tRNAmedicines #tRNAbiology #rarediseases #geneticmedicines #programmablemedicines #AI #machinelearning #StopCodonDisease

"Helixomer: Pioneering RNA Origami to Revolutionize Safe and Effective Anticoagulation" Helixomer is pleased to announce the publication of our patent application, introducing the first-of-its-kind RNA origami aptamer platform. This is a breakthrough in precision medicines. Our groundbreaking anticoagulant therapy is designed for both safety and high efficacy, featuring specific reversal agents —addressing critical unmet needs in healthcare. This achievement not only showcases the power of RNA origami in transforming anticoagulation but also sets the stage for expanding into areas like oncology, neurodegenerative diseases, and antimicrobials. To explore how RNA origami is reshaping healthcare, we invite you to reach out and join us on this exciting journey. #PrecisionMedicine #Biotech #HealthcareInnovation #DrugDevelopment #InvestmentOpportunity #Anticoagulation #RNAOrigami #helixomer

GenieUs Genomics is delighted to announce our #partnership with Neurizon, to harness comprehensive #genomic #profiling to identify digital #biomarkers in the genome of patients most likely to benefit from their promising #ALS therapeutic, NUZ-001. 📰Read our EINPresswire announcement at https://lnkd.in/eFCyV86j 🎯ALS is not one disease. Our mission is to decode the genetics of ALS and #MND subtypes, to inform which groups will respond to a drug, and facilitate the next generation of ALS therapeutics. 🧬Only through a #precision #approach can we effectively address and treat #heterogeneous #CNS diseases like ALS and MND.