BioDataStudio’s Post

🔬 Orbital Therapeutics Raises Record-Breaking $270M Series A TL;DR: • Largest biotech Series A of the year • Focus: Next-gen RNA technology development • Timeline: IND filing planned within 2-4 years • Key partnership with Beam Therapeutics Despite current market headwinds, Orbital Therapeutics has secured massive investor confidence, demonstrating the enduring potential of innovative RNA therapeutics. The Cambridge-based company is targeting applications beyond traditional RNA limitations, including vaccines, protein replacement, and treatments for autoimmune conditions and cancer. Led by CEO Giuseppe Ciaramella and industry veteran John Maraganore (former Alnylam CEO), Orbital is positioned to expand the reach of RNA-based drugs beyond liver-targeted therapies - a significant limitation of current technologies. Strategic moves include: • CircBio acquisition for circular RNA IP • Stanford University delivery tech licensing • Dual presence in Cambridge and South SF • Partnership with Beam Therapeutics Funding led by Arch Venture Partners, with participation from a16z, Newpath Partners, and others. 💭 What are your thoughts on the future of RNA therapeutics? How might this funding reshape the biotech landscape? #Biotech #RNATherapeutics #VentureCapital #Innovation #BiotechNews #intelligencesimplified #pharma #biotech #biodatastudio

✨ Want to know more about how our technology can solve the challenges of complex targets? Look below to learn more about how our teams address RNA through cutting edge simulations.

🧬 Unlocking RNA Targets: Advancing Drug Discovery with Cutting-Edge Simulations 💫 At Qubit Pharmaceuticals, we’ve made a breakthrough in computational drug design, tackling one of the biggest challenges in modern medicine: RNA-targeted therapeutics. By combining cutting-edge technologies like: 🔸 Lambda-ABF for precision binding affinity prediction 🔸 Tinker-HP for enhanced molecular dynamics 🔸 Advanced sampling techniques in combination with machine learning to capture complex RNA conformational changes …we're paving the way for faster, more accurate drug discovery, with unprecedented insights into RNA-small molecule interactions. RNA-targeting therapeutics also expand the druggable target space in the human genome from 0.2% to ~70%, offering an attractive alternative to reach traditionally undruggable proteins! 📰Our latest blog explores how our advanced methods open new possibilities for designing drugs to treat the most difficult diseases. The blog is based on a scientific publication, currently available as a preprint, showcasing these innovations. A special thanks to our partners Bpifrance, European Innovation Council and SMEs Executive Agency (EISMEA) , #FRANCE 2030, GENCI , Secrétariat général pour l'investissement, Région Île-de-France, and Sorbonne Université, as well as our investors Quantonation, XAnge & Omnes. And, of course, 👏massive congratulations👏 to the authors, Narjes A. , Chengwen Liu, Florent Hédin, Jérôme Hénin, Jay Ponder, Pengyu Ren, Jean-Philip Piquemal, Louis Lagardère, and Krystel El Hage, PhD El Hage for their remarkable work! 👉 Read more and discover how we're revolutionizing RNA drug discovery: https://loom.ly/SdIjUxA 👉 Straight to the preprint? Sure! https://loom.ly/Al9PcUU Pharmaceuticals and biotech companies seeking to unlock new treatment possibilities, let’s collaborate and make the next breakthrough together - Reach out to us today! #ComputationalChemistry #QuantumAidedDrugDesign #RNAtherapeutics #DrugDiscovery

Radar Therapeutics Completes $13.4M Seed Financing To Advance Smart Programmable Medicines Learn more & get our take 👇 https://lnkd.in/g5DfBuay “With Radar’s technology, we can now precisely alter the biology of the cell, delete harmful cells, or potentially reprogram cells for autoimmune diseases. This has the potential to enable a new generation of safer, more durable, and effective mRNA therapeutics for applications beyond vaccines.” — Jim Collins, Ph.D., Co-Founder at Radar Therapeutics “Creating genetic expression-regulation systems that operate at the level of translation while being programmable to ensure compatibility with next-generation mRNA-based medicines has been a long-lived dream.” — Xiaojing Gao, Ph.D., Associate Professor of Chemical Engineering at Stanford University and Radar Co-Founder “Like a safety switch, our payload is always off, and only gets turned on in the right cell. We can selectively write a function into any cell type. Programmable mRNA-based therapies have the potential to be in vivo, scalable, and modular, to improve patient access.” — Sophia Lugo, CEO & Co-Founder at Radar Therapeutics #biotech #funding #SoHCNews

Reflecting on my time at the RNA Leaders Conference last week, here are my top takeaways: 1) Excellent talks and panels. The PolymerRAISE series was on another level of conference pitches. Especially loved the talks from Rabia T. K. at Serna Bio and Carter Palmer at Third Element Biosciences. Also really resonated with the corporate values reflected by execs at Johnson & Johnson and Novo Nordisk. 2) The RNA space is moving beyond the liver and CNS, where delivery was most straightforward in early days, and I’m excited to see where innovative targeting platforms take mRNA and saRNA therapeutics and vaccines, and how that effects dosing. 3) Storytelling is as important as ever in partnering in this space as in others. Platform companies need to make a compelling case for how the technology they’re using fits the indication and population they’ve chosen for their lead asset. This helps decision makers in pharma really see how a deal with you will fit into their portfolio and have impact on the market Excited to fold what I’ve learned into the work we do! 🧠 📓 #biotech #rna #therapeutics #computationalbiology #conferencereflections

🚀 Big News from Radar Therapeutics! 🚀 Radar Therapeutics has successfully raised $13.4 million in seed financing to advance its mission of developing smart programmable medicines using molecular RNA sensors! 🎉💉 The funding round was led by NfX Bio with participation from major investors including Eli Lilly and Company, Biovision Ventures, and KdT Ventures. Additional support came from PearVC, BEVC, and other esteemed investors. 🙌💰 What They're Working On: 🔬 Programmable Genetic and mRNA-Based Therapeutics: Radar Therapeutics is pioneering the use of RNA sensors—mRNAs that control gene expression based on cell-specific RNAs—to enable precise, targeted, and timed drug delivery. This approach aims to minimize systemic side-effects and enhance therapeutic efficacy. Key Highlights: 🌟 Innovative Technology: The RADAR platform allows for the rational design of precision therapeutics, capable of altering cell biology, deleting harmful cells, or reprogramming cells for autoimmune diseases. 🌟 Expert Leadership: Co-founded by synthetic biology pioneer Jim Collins, Ph.D., and Xiaojing Gao, Ph.D., with industry-leading insights from CEO Sophia Lugo and CSO Eerik Kaseniit, Ph.D.. 🌟 Impactful Publications: Featured in Nature Biotechnology, showcasing highly specific, compact sensor sequences for targeted gene expression. 🌟 Industry Recognition: Honored with the AbbVie Golden Ticket, Johnson & Johnson West Coast Cell and Gene Therapy Symposium "Judge's Choice" award, and the Amgen Diversity, Inclusion, and Belonging Award. “We’re leveraging the explosion in single-cell transcriptomic data and advances in RNA-editing enzymes to create smart mRNA therapies,” said Eerik Kaseniit, Ph.D., Chief Scientific Officer & Co-Founder. “By leveraging a broad dataset offered by single-cell transcriptomics, Radar can precisely identify cellular signatures and engineer programmable therapies accordingly, offering unparalleled specificity to avoid off-target effects,” said Omri Amirav-Drory, PhD, Partner, NfX Ventures. With the support of these top-tier investors, Radar Therapeutics is set to advance its preclinical programs and continue expanding its team. 🚀 #Biotech #mRNA #RNAEditing #PrecisionMedicine #GeneticTherapeutics #HealthcareInnovation #Investment #SeedFunding #SmartMedicine #BiotechInnovation #SingleCellTranscriptomics #DrugDevelopment #RadarTherapeutics

🧬 𝐖𝐢𝐥𝐥 𝐦𝐢𝐜𝐫𝐨𝐑𝐍𝐀 𝐦𝐚𝐤𝐞 𝐢𝐭 𝐭𝐨 𝐭𝐡𝐞 𝐦𝐚𝐫𝐤𝐞𝐭? Last week, Victor Ambros and Gary Ruvkun received the Nobel Prize in Physiology or Medicine for identifying microRNA, tiny RNA molecules that regulate gene expression. Personally, I had an '𝒂𝒘𝒆' moment, as I have worked with these molecules for over five years developing delivery systems. More recently, I came across an article published in Nature summarising the commercialisation potential of microRNAs. It's true that early clinical trials with microRNAs have faced several setbacks, such as immune responses and poor delivery. However, the recent acquisition of Cardior Pharmaceuticals (a company developing microRNA therapies for heart failure) by Novo Nordisk for US$1.12 billion highlights that the pharma industry still believes in these therapeutics. After mRNA vaccines developed by Pfizer and Moderna, or RNAi therapeutics developed by Alnylam Pharmaceuticals, should we expect novel RNA modality to make it to the market? 𝘞𝘩𝘢𝘵 𝘢𝘳𝘦 𝘺𝘰𝘶𝘳 𝘵𝘩𝘰𝘶𝘨𝘩𝘵𝘴? #microrna #nobelprize #pharma #biotech #drjojo Source to the article from Nature in comments. ---------- Hi! I am Joanna, and my friends call me Dr Jojo 🌸 𝐈 𝐭𝐚𝐥𝐤 𝐚𝐛𝐨𝐮𝐭 𝐢𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐚𝐧𝐝 𝐛𝐮𝐬𝐢𝐧𝐞𝐬𝐬 𝐢𝐧 𝐩𝐡𝐚𝐫𝐦𝐚 𝐚𝐧𝐝 𝐛𝐢𝐨𝐭𝐞𝐜𝐡 ♻️ Share this if you find it interesting 🔔 Follow for insights you won't want to miss

The building blocks of proteins are L-amino acids which are chiral molecules. Boston-based startup Abiologics (apt name by the way... 😀) uses generative AI and high-throughput chemical synthesis to synthesise #Synteins which are composed of D-amino acids! D-amino acids do exist in nature, including humans. Interestingly, L-to-D isomerization is associated with aging and age-related disorders including neurodegeneration. I found out that there is a decent body of research on this, especially D-peptides for therapy. But what would be some of the considerations when designing and working with therapeutic D-proteins? (1) Folding - If L-proteins are delivered to cells, can they stay folded in the cellular milieu? Can they utilise L-chaperones to remain stable once inside? A perfect D-complement of an engineered L-antibody can be expected to stably fold. But what if we are to design novel moieties? We can apply the rules of folding from our existing knowledge. I think this is where the ML part comes in. (2) Therapeutic Activity - Can D-proteins bind and "work" with L-proteins or other biomolecules like DNA/RNA/lipids which are themselves chiral. For instance, can a D-antibody bind the L-counterpart's antigen? The answer again perhaps lies in AI/ML-based computational design. Upon target-binding, can D's have desired downstream effects, like phosphorylation, localization/targeting ability (say to proteosomes), catalytic activity, nuclease activity? Do these evade the immune system? Likely, because the proteolytic machineries which are L-proteins themselves are likely to recognize only the L-forms. (3) Structure - D-proteins will have interesting spectroscopic/biophysical properties for sure! NMR, X-ray, cryo-EM, MD-simulations? Sure. Maybe some obstacles to building atomic models, but unlikely to be difficult. (4) Degradation - Can D-proteins be inactivated, denatured or actively degraded? What would be their half-life? Can these be excreted out by passive exocytosis? Low pH and high temperature can still inactivate/denature the protein. If they can be degraded, will there be side effects from the build-up of D-amino acids? (5) Delivery - Cannot be genetically encoded, hence must be delivered directly for therapeutics. There are ways to deliver proteins directly and this is an already explored area. Ideally, a mix of L- and D-amino acids will have the best of both worlds. For e.g., an L-localization sequence (mitochondria, nucleus etc.), a D-segment for target-binding, and an effector with both L- and D-sequences. Wonder how this would influence secondary/tertiary structure of proteins? Eager to know more about Synteins and what they can deliver. Wishing Abiologics the best. #DrugDiscovery #ProteinTherapeutics #ProteinFolding #GenerativeAI #MachineLearning #Proteins #ProteinEngineering #StructuralBiology

New biotech startup emerges to introduce new class of biologic drugs Flagship Pioneering has unveiled Abiologics, a new company pioneering supranatural biologics. With an initial $50 million commitment, Abiologics aims to develop Synteins™, a novel class of programmable medicines that could advance patient treatment across various diseases, initially focusing on oncology and immunology. Synteins™ represent a paradigm shift in biologic design. Unlike traditional biologics limited to 20 natural amino acids, Synteins are computationally designed using a broader set of artificial building blocks, including D-amino acids. This approach leverages cutting-edge generative AI for design and innovative chemical synthesis techniques for production. The result is a class of biotherapeutics with extraordinary properties: ✔ Immune evasion: By using non-natural building blocks, Synteins can potentially avoid recognition by the immune system. ✔ Enhanced stability: This could lead to less frequent dosing and possibly oral delivery options. ✔ Improved tissue penetration: Synteins may access previously unreachable parts of the body. ✔ Programmability: These molecules can be designed to interact with virtually any therapeutic target. This news appeared today on BioPharmaTrend.com. Follow Where Tech Meets Bio (Substack Newsletter) for daily biotech insights with a focus on platforms and technological advances. #biopharmatrend #drugdiscovery #biotech Image source: Distinctive Roles of D-Amino Acids in the Homochiral World: Chirality of Amino Acids Modulates Mammalian Physiology and Pathology, Jumpei Sasabe and Masataka Suzuki, The Keio Journal of Medicine 68(1)