Biomedcode’s Post

🔔 First clinical evaluation of an inhaled mRNA-based therapy designed to restore ciliary function in people with PCD ! 👏 ReCode Therapeutics’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform was used to develop RCT1100, an inhaled mRNA therapy designed to deliver and express DNAI1 mRNA in target cells. 🔎 Interested in monitoring mRNA therapeutic related IP like recently published patent related to SORT LNP? Take advantage of KnowMade's patent monitoring service that is focused on therapeutic mRNA https://lnkd.in/eCfnR-ct #mrna #LNP #patent #innovation

Exploring RNA delivery strategies for cardiovascular disease therapeutics: in this extensive review article, Elisa Garbayo Atienza, Maria Blanco-Prieto and collaborators at the University of Navarra provided a comprehensive overview of RNA-based therapeutic strategies investigated for cardiovascular diseases, including non-viral delivery systems such as extracellular vesicles, lipid-based carriers, polymeric and inorganic nanoparticles, and hydrogels https://lnkd.in/eYz35FkZ Additionally, they discussed the current status of these therapies and the challenges impeding their clinical implementation. An article also authored by Paula Gil Cabrerizo and Teresa Simón-Yarza #extracellularvesicles #exosomes #LNP #nanomedicine #drugdelivery #cardiovasculardiseases #Vesiculab

🌟 Exciting Times Ahead in #lupus Treatment! 🌟 The realm of autoimmune diseases, particularly Lupus, is witnessing groundbreaking advancements thanks to CAR-T CD19 therapies. A multitude of companies are spearheading this innovative approach, aiming to revolutionize patient care and outcomes. Here's a shoutout to the trailblazers in this space: Century Therapeutics, Inc, Cartesian Therapeutics, Sana Biotechnology, Inc., Bristol Meyers Squibb, Novartis, Autolus Therapeutics, iCell Gene Therapeutics, Kyverna Therapeutics, Cabaletta Bio, Galapagos, Luminary Therapeutics, Inc, Interius BioTherapeutics, Intellia Therapeutics, Inc. Credit Andrew Pannu for the amazing infographic on the latest Cell Therapy Landscape chart shown below. We've highlighted some of the key players in Lupus.

Today is #RareDiseaseDay, dedicated to raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and caretakers. At ARMGO, we are committed to making a difference for rare disease patients by developing our proprietary Rycal® drugs. Rycals are novel small molecule therapeutics that repair leaky ryanodine receptor (RyR) channel proteins. These drugs have the potential to become a powerful new treatment option for patients suffering from Catecholaminergic Polymorphic Ventricular Tachycardia (#CPVT), a rare, genetic #arrhythmia and RYR1-related myopathy (RYR1-RM), a rare muscle disease. To find out more about Rare Disease Day, visit: https://lnkd.in/gk6hbAF. #RareDiseaseDay #CPVT #RYR1-RM #RYR-1Foundation

We are delighted to announce today, on Rare Disease Day, that Coave Therapeutics has received a grant from the The ALS Association to advance its investigational CTx-TFEB program as a potential treatment for all forms of ALS. CTx-TFEB is a novel, targeted genetic medicine approach designed using Coave’s unique ALIGATER™ platform to promote autophagy and reduce the accumulation of toxic protein aggregates in neurons in the brain and spinal cord of people with ALS and that lead to nerve degeneration, paralysis, respiratory failure and ultimately death. There is no cure. With this funding and invaluable support from the ALS Association, we are poised to move our CTx-TFEB program forward. Our goal is to establish a robust preclinical proof-of-concept, laying a strong foundation for its advancement into clinic trials in the near future. Read our press release: https://lnkd.in/euTD95Vt More about the ALS Barnett Drug Development Program: https://lnkd.in/ebWw8D9G #ALS #genetherapy #autophagy #clinicaltrials

Comings and Goings: Denali Therapeutics Announces Anticipated Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases. - Expect enrollment to be completed for late-stage clinical trials for MPS II (Hunter's) and ALS (elF2B Agonist) in 2024 - Expect initiation of clinical development for 2nd Enzyme Transport Vehicle (TV)-enabled program, DNL126 in MPS IIIA, and 1st two Oligonucleotide TV-enabled programs in IND-enabling studies targeting tau in Alzheimer’s disease (OTV:MAPT) & alpha-synuclein in Parkinson’s disease (OTV:SNCA) - Will prioritize advancing additional TV programs for common neurodegenerative diseases see https://lnkd.in/eCNnedcZ see also JP Morgan Healthcare Conference Presentation https://lnkd.in/eas9pytb

This week is exciting! Two companies announced positive interim results from their respective clinical trials in two rare kidney diseases that have an exceptionally high unmet need for new treatment options.   📣 Dimerix Limited annouced positive interim results for DMX-200 based on the proteinuria efficacy endpoint in its Phase 3 ACTION3 trial in #FSGS.   📣 Regulus Therapeutics announced positive topline data for RGLS8429 based on imaging-based biomarkers in its Phase 1b MAD trial in autosomal dominant polycystic kidney disease (#ADPKD).   Through Spherix's tracking of these markets over the years, it is evident that nephrologists are frustrated by the challenges they face in managing these diseases and welcome new therapies to help fulfill the unmet medical needs of their patients. #marketdynamics #marketresearch #nephrology

Pick of the week from J Med Chem: IRAK4 degrader for autoimmune diseases from Kymera Therapeutics (Xiaozhang Zheng). The  discovery story of clinical compound KT-474 highlights CRBN binder and linker optimization towards a heterobifuncional degrader with meaningful systemic exposure. A great high-level overview of how to get an in vivo efficacious PROTAC #medchem https://lnkd.in/eW-Zg2C9

𝐓𝐡𝐞 𝐠𝐥𝐨𝐛𝐚𝐥 𝐨𝐥𝐢𝐠𝐨𝐧𝐮𝐜𝐥𝐞𝐨𝐭𝐢𝐝𝐞 𝐬𝐲𝐧𝐭𝐡𝐞𝐬𝐢𝐬 𝐦𝐚𝐫𝐤𝐞𝐭 𝐬𝐢𝐳𝐞 𝐢𝐬 𝐞𝐱𝐩𝐞𝐜𝐭𝐞𝐝 𝐭𝐨 𝐫𝐞𝐚𝐜𝐡 𝐔𝐒𝐃 𝟏𝟎.𝟏𝟕 𝐛𝐢𝐥𝐥𝐢𝐨𝐧 𝐛𝐲 𝟐𝟎𝟑𝟐, 𝐞𝐱𝐡𝐢𝐛𝐢𝐭𝐢𝐧𝐠 𝐭𝐡𝐞 𝐂𝐀𝐆𝐑 𝐨𝐟 𝟏𝟐.𝟓𝟓% 𝐝𝐮𝐫𝐢𝐧𝐠 𝐭𝐡𝐞 𝐟𝐨𝐫𝐞𝐜𝐚𝐬𝐭 𝐩𝐞𝐫𝐢𝐨𝐝. 𝙁𝙤𝙧 𝙈𝙤𝙧𝙚 𝙍𝙚𝙥𝙤𝙧𝙩 𝘿𝙚𝙩𝙖𝙞𝙡𝙨, 𝙑𝙞𝙨𝙞𝙩: https://lnkd.in/diVgT8px Oligonucleotide therapeutics exhibit substantial promise in addressing chronic conditions such as retinal disorders, cancers, and cardiovascular, among others. The continuous evolution and newfound implementation of oligo sequences across various biotechnological domains are anticipated to propel market expansion. A significant upsurge in demand for molecular diagnostics, especially in precision care, stands out as a crucial emerging application for oligonucleotide synthesis. #oligonucleotidesynthesis #biotechnology #genomics #dnaresearch #molecularbiology #pharmaceuticalresearch #genetherapy #biotechinnovation #customsynthesis #geneticengineering

Scientific American recently published a piece highlighting the potential of next-generation psychedelics as medicines, especially non-hallucinogenic compounds like Delix Therapeutics's #neuroplastogens. The article features our CSO Kurt Rasmussen and our CMO Aaron Koenig MD discussing our groundbreaking work with our lead #neuroplastogen, DLX-001, and how Delix is pioneering innovative new treatments for #neuropsychiatric conditions that deliver therapeutic benefits without causing hallucinogenic or cardiotoxic effects. Check out the full article: https://lnkd.in/eaWzGF7h.