RNA Leaders’ Post

The April issue cover of Cell Press Trends in Pharmacological Sciences (TIPS) is online now. On the cover: Siyu Chen, Albertas Navickas,and Hani Goodarzi discuss how changes in the mRNA translation machinery are tailored to specific sequence and structure information encoded in the mRNA, and interconnect with additional regulatory layers to drive breast cancer progression and metastasis. Metastasis is the process where cancer cells disseminate from their original site to other body parts and is a key contributor to cancer-related mortality. Also, they explore how understanding metastasis mechanisms can be harnessed for developing novel breast cancer treatment strategies, particularly those aimed at overcoming drug resistance and inhibiting the spread of cancer cells. Other reviews in the April issue focus on promising opportunities in leveraging macrophage metabolism for anticancer therapy (Piyal Saha, Paul Ettel, Thomas Weichhart), advancements in drug delivery strategies aimed at overcoming the technical barrier in targeting mitochondria for CVD treatment (Mirza Ahmar Beg, Yiliang C. and colleagues), recent advances and applications of genetically engineered loaded extracellular vesicles for drug delivery(Zuriñe Eraña Pérez, Manoli Igartua Olaechea, Edorta Santos Vizcaíno, Rosa Maria Hernandez),  the potential of targeting Fks1 for antifungal drug development (Ge-Fei Hao and colleagues), and the latest pharmaceutical treatments for Steatotic liver diseases (Mari Jokinen & Panu K. Luukkonen). Do not miss the two Forum articles in this issue: the first focuses on the role of NMR in advancing small molecule drug discovery (Leszek Poppe), and the second analyzes recent research to understand the sensitivity of senescent cells to BH3 mimetics (David Bernard and colleagues). Reach out to me on here or at the Trends in Pharmacological Sciences website if you have an idea for a Drug of the Month, Scientific Life, Opinion, or Review article you wish to publish. Take a look at the full issue. https://lnkd.in/e4Jh2hcY

GRANT NEWS We are delighted announce that we have been awarded a non-dilutive grant from TARGET ALS FOUNDATION, INC in collaboration with Biospective Inc., to support our discovery proof-of-concept studies in our mitochondrial permeability transition pore (#mPTP) inhibitor drug discovery programme for a treatment for Amyotrophic Lateral Sclerosis (#ALS) (also known as Lou Gehrig’s disease or Motor Nuerone Disease, #MND).   The In Vivo Target Validation Program aims to generate high-quality data on candidate therapeutics in Biospective’s modified ALS TDP-43 mouse model, provide access to in-kind funding for a resource-intensive step of drug discovery, and accelerate the progress of candidate therapeutics from preclinical testing towards clinical trials.   NRG Therapeutics’ co-founder and CEO Dr Neil Miller said, “Preliminary experiments using a mouse model of ALS which contains the human gene for TDP-43 showed that our mPTP inihibitor candidate therapeutic reduced brain cell death. However, due to the rapid disease progression in that mouse model, a full assessment of the therapeutic potential was not possible. This project aims to evaluate NRG’s small molecule inhibitor, in the modified ALS TDP-43 mouse model that progresses more slowly. This will allow for a comprehensive assessment of the molecule’s ability to protect mitochondria, reduce brain cell death, and decrease immune system activation. Additionally, the study will measure improvements in muscle function and strength. If successful, this project will provide crucial support for advancing our mPTP inhibitor into clinical trials for ALS.”   We are one of five recipients of the 2024 In Vivo Target Validation grants. Details of the Target ALS 2024 In Vivo Target Validation grant awardess can be found HERE https://lnkd.in/ebpKCN4H See our full press release HERE https://lnkd.in/emNuMGtF   #BioTech #DrugDiscovery #Grant #NeurologicalDisorders #CNS Vad Lazari

AbbVie announced today the acquisition of Celsius Therapeutics, Inc. ("Celsius"), a privately held biotechnology company pioneering new therapies for patients with inflammatory disease. Celsius' lead investigational asset is CEL383, a potential first-in-class anti-TREM1 antibody that has completed a Phase 1 clinical study for the treatment of IBD. TREM1 has been identified as a key disease driver gene in IBD, where it is expressed on inflammatory monocytes and neutrophils. In these cell types and others, TREM1 is upstream of multiple known inflammatory pathways and acts as an amplifier of inflammation. "Given the potential relevance of TREM1 as a key driver of inflammation and pathology in IBD and other conditions, we are eager to advance the development of CEL383 with a goal of helping more patients with IBD achieve remission," said Kori Wallace, MDPhD, vice president, global head of immunology clinical development, AbbVie. "AbbVie shares our excitement about the potential of TREM1 inhibition for patients with inflammatory disease," said Tariq Kassum, M.D., chief executive officer, Celsius. "I'd like to thank the Celsius team for their relentless efforts in the discovery of CEL383. We look forward to the further development of this promising program, which we hope will offer a new approach to the treatment of IBD." Under the terms of the agreement, AbbVie has acquired all outstanding Celsius equity for $250 million in cash, subject to certain customary adjustments. #inflammatorydisease #immunology #IBD #acquisition #AbbVie #CelsiusTherapeutics 👉 https://lnkd.in/dJCqX8PW

𝐌𝐨𝐝𝐞𝐫𝐧𝐚’𝐬 𝐦𝐑𝐍𝐀-𝟑𝟕𝟎𝟓 𝐒𝐞𝐥𝐞𝐜𝐭𝐞𝐝 𝐟𝐨𝐫 𝐅𝐃𝐀’𝐬 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐏𝐫𝐨𝐠𝐫𝐚𝐦 Moderna Moderna announced that the FDA has selected its investigational methylmalonic acidemia (MMA) therapy, mRNA-3705, for the agency’s START pilot program. Launched in September 2023, START is designed to offer drugmakers “regular ad-hoc communication with FDA staff to address product-specific development issues,” such as trial design and patient population choices. Companies also receive guidance on non-clinical information and product characterization. Eligible companies must be developing gene or cell therapies for rare neurodegenerative or genetic metabolic diseases likely to cause significant disability or death within the first decade of life. Moderna’s mRNA-3705, which targets MMA, a life-threatening metabolic disorder, fits these criteria. MMA is characterized by the accumulation of toxic substances in the body, leading to metabolic crises and multi-organ damage. It is often diagnosed within the first few months of life, with no approved treatments currently available. mRNA-3705 delivers a MUT-encoding mRNA molecule via a proprietary lipid nanoparticle, addressing the underlying disease pathway in MMA. The candidate, which has Orphan Drug, Rare Pediatric Disease, and Fast Track designations, is being evaluated in a Phase I/II open-label study for patients aged one year and older. Kyle Holen Kyle Holen, head of development for therapeutics and oncology at Moderna, stated that the company is “excited” and “proud” to be selected for START, which will “enable enhanced communication with the U.S. FDA, resulting in the acceleration of our development program.” Moderna plans a pivotal MMA study for mRNA-3705 this year. “This selection highlights the promise of Moderna’s mRNA platform beyond vaccines and the potential of this novel medicine to address the unmet medical needs of MMA,” Holen said. Moderna joins four other biopharma companies selected for START: Denali Therapeutics Denali Therapeutics, Grace Science Grace Science, LLC, Larimar Therapeutics Larimar Therapeutics Inc., and Neurogene Neurogene Inc., each developing therapies for other rare diseases. #Moderna #mRNA3705 #RareDiseases #FDA #STARTProgram #Biotech #ClinicalTrials #MedicalResearch #PharmaInnovation #Healthcare #DrugDevelopment

Catch the hottest mRNA clinical updates and new data in July! https://ter.li/03nqzd ➡️ Download the program for the 4th mRNA-Based Therapeutics Summit Here's just a snapshot of hot clinical updates and new data being shared: 🚀 Next generation self-replicating RNA drives substantial improvement in infectious disease vaccines - with Replicate Bioscience, Inc. 🚀 Leveraging the LUNAR platform to develop RNA medicines for diseases with significant unmet medical needs and acclerated clinical paths - with Arcturus Therapeutics 🚀 Clinical safety and impacy of RNA cell therapy in patients with Myasthenia Gravis - with Cartesian Therapeutics Download the event guide to see the comprehensive 3-day, 4-track, 65+ speaker agenda ➡️ https://ter.li/03nqzd #mRNA #mRNATherapeutics #mRNAVaccines #RNA #RNATherapeutics #srRNA #selfreplicatingRNA

RNA-based therapeutics and vaccines are having a major moment, but while these approaches hold great promise for disease treatment and prevention, they still face a major hurdle to widespread use: delivery. "If an RNA drug cannot enter the right target cell, it will not work," Sangeeta Bhatia and James Dahlman write in an introduction to a special PNAS feature on RNA drugs (https://lnkd.in/emzrAzhN). "Since RNAs are large and anionic, they cannot efficiently cross the cell bilayer on their own and therefore usually require a drug delivery system."  Also in PNAS, Liangliang Hao, Natalie Boehnke, Susanna Elledge, Bhatia, and colleagues detail efforts to develop an RNA-loaded nanoparticle for treating and monitoring treatment response in ovarian cancers (https://lnkd.in/ep5JvU2h), while Namita Nabar, Paula Hammond Cunningham, and colleagues discuss the design of layered lipid nanoparticles (https://lnkd.in/eMTP8UeT) for tunable delivery of RNA to a wide variety of cell and tissues. And Victoria Madigan, Yugang Zhang, Rumya Raghavan, Feng Z., and colleagues show how a human protein, PNM2, can self-assemble in a test tube and can be engineered for RNA packaging and delivery (https://lnkd.in/eKDwSwfJ). #BroadInstitute #Science #ScienceNews #Research #ScientificResearch

Good collection of #RNA packaging & delivery articles in a special feature of PNAS. Thanks Broad Institute of MIT and Harvard for highlighting this.  Grab a coffee and give these pieces a read! #mRNA #LipidNanoparticles #CellTherapy #GeneTherapy #Vaccines #Oncology #PrecisionMedicine #RareDisease #ADCY5

AbbVie Acquires Celsius Therapeutics AbbVie has acquired Celsius Therapeutics, a privately held #biotechnology company developing new therapies inflammatory diseases, for $250 million in cash. The transaction is subject to certain customary adjustments. Celsius' lead investigational asset is CEL383, a potential first-in-class anti-TREM1 #antibody that has completed a Phase 1 clinical study for the treatment of I inflammatory bowel disease (IBD). TREM1 has been identified as a key disease driver gene in IBD, where it is expressed on inflammatory monocytes and neutrophils. In these cell types and others, TREM1 is upstream of multiple known inflammatory pathways and acts as an amplifier of #inflammation. "Given the potential relevance of TREM1 as a key driver of inflammation and #pathology in IBD and other conditions, we are eager to advance the development of CEL383 with a goal of helping more patients with IBD achieve remission," said Kori Wallace, MDPhD, vice president, global head of immunology clinical development at AbbVie. Read more on #CMI online: https://lnkd.in/e5SGCyiT

AbbVie announced today the acquisition of Celsius Therapeutics, Inc. ("Celsius"), a privately held biotechnology company pioneering new therapies for patients with inflammatory disease. Celsius' lead investigational asset is CEL383, a potential first-in-class anti-TREM1 antibody that has completed a Phase 1 clinical study for the treatment of IBD. TREM1 has been identified as a key disease driver gene in IBD, where it is expressed on inflammatory monocytes and neutrophils. In these cell types and others, TREM1 is upstream of multiple known inflammatory pathways and acts as an amplifier of inflammation. "Given the potential relevance of TREM1 as a key driver of inflammation and pathology in IBD and other conditions, we are eager to advance the development of CEL383 with a goal of helping more patients with IBD achieve remission," said Kori Wallace, MDPhD, vice president, global head of immunology clinical development, AbbVie. "AbbVie shares our excitement about the potential of TREM1 inhibition for patients with inflammatory disease," said Tariq Kassum, M.D., chief executive officer, Celsius. "I'd like to thank the Celsius team for their relentless efforts in the discovery of CEL383. We look forward to the further development of this promising program, which we hope will offer a new approach to the treatment of IBD." Under the terms of the agreement, AbbVie has acquired all outstanding Celsius equity for $250 million in cash, subject to certain customary adjustments. #inflammatorydisease #immunology #IBD #acquisition #AbbVie #CelsiusTherapeutics

Published this week: Check out the 2024 New Drug Modalities Report, which explores the incredible expansion of opportunity over this past year. The #biopharma industry is witnessing a wave of innovation, with new drug modalities projected to reach a pipeline value of $168 billion by 2024, up 14% since 2023. Breakthrough therapies such as #gene therapies, #microbiomes, and expanded #mRNA uses are leading this growth, but investor caution around ADCs and CAR-T remains. The report offers key insights into how companies are adapting to these changes and positioning for future success. Congrats especially to my colleagues, Lu Chen & Brian Bush, who co-authored the work! Explore the full report to learn more: #Biopharma #antibodies #cagt #genetherapy #GLP1 #BCG