BioPioneers’ Post

Novartis has announced a $1.1 billion deal with Kate Therapeutics, a San Diego-based biotech focused on developing innovative gene therapies for challenging conditions like Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1). The deal is the combination of upfront cash and milestone payments. This deal comes as the muscular dystrophy field faces setbacks, with high-profile failures reminding us of the hurdles in tackling these diseases. However, Novartis’ CEO, Vas Narasimhan, emphasized how Kate’s platform—designed to target muscle and cardiac tissue while avoiding the liver—could help address these challenges and advance treatments for neuromuscular conditions. Building on its prior experience with Zolgensma, Novartis aims to learn from past industry struggles while pushing the boundaries of gene therapy for complex diseases. Read more in the following article in Fierce Biotech: https://lnkd.in/erYpX2Vn #musculardystrophy #aav #deliver #miRNA #novartis

Huge Acquisition 🚨 Novartis Takes Bold Step in Muscular Dystrophy with $1.1B Kate Therapeutics Deal Novartis is expanding its gene therapy efforts by acquiring Kate Therapeutics, focusing on treatments for Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1). 𝗞𝗲𝘆 𝗣𝗼𝗶𝗻𝘁𝘀: ➡️ Kate Therapeutics cutting-edge platform targets muscle and cardiac tissues while avoiding the liver, solving key delivery challenges. ➡️ Novartis aims to learn from past industry setbacks to advance its neuromuscular pipeline. ➡️ This move strengthens Novartis gene therapy portfolio beyond its spinal muscular atrophy treatment, Zolgensma. What do you think about Novartis push into this challenging space? 𝗙𝘂𝗹𝗹 𝗔𝗿𝘁𝗶𝗰𝗹𝗲 𝗕𝗲𝗹𝗼𝘄: ⬇️ #biotech #genetherapy #novartis #katetherapeutics

🚀 Exciting Developments in Cell & Gene Therapy! 🚀   The cell & gene therapy landscape is buzzing with two major acquisitions in the last week: 1. Roche's acquisition of Poseida Therapeutics, Inc. Therapeutics for up to $1.5B. This strategic move will bolster Roche's capabilities in off-the-shelf CAR-T therapies, aiming to democratize access to these life-saving treatments. Poseida's innovative non-viral technology platform, rich in T stem cell memory cells, will hopefully enhance the safety and efficacy of CAR-T therapies, potentially benefiting a broader patient population. 2. Meanwhile, Novartis has acquired Kate Therapeutics in a deal valued at up to $1.1B. This acquisition aligns with Novartis' commitment to advancing gene therapies for neuromuscular diseases. Kate Therapeutics' cutting-edge AAV-based gene therapy platform and promising preclinical candidates for conditions like Duchenne muscular dystrophy and myotonic dystrophy type 1 will strengthen Novartis' pipeline and innovation in gene therapy.   These acquisitions underscore the relentless pursuit of innovation in the biotech industry, aiming to bring transformative therapies to patients worldwide. #GeneTherapy #HealthcareInnovation #CART  

As more #clinicaltrials are initiated and safety issues are addressed, #iPSC-based #genetherapy is likely to play an increasingly important role in #personalizedmedicine. In his most recent article for Cell & Gene, CSTEAM biotechnology's Jack (Jie) Huang MD, PhD discusses the current progress, applications, challenges, and future prospects of iPSC-based gene therapy, focusing on its potential in treating #geneticdiseases, #cancer, and age-related disorders. https://lnkd.in/eYziRt-H

Odylia Therapeutics, a nonprofit biotechnology organization based in Atlanta, has received a $1.5 million grant from the Foundation Fighting Blindness to fast-track their gene therapy, OT-004, targeting vision loss caused by RPGRIP1 gene mutations. This grant will accelerate the development of their gene therapy aimed at treating rare vision loss by targeting specific gene mutations that lead to rapid vision decline. Mutations in the gene lead to rare forms of vision loss like Leber congenital amaurosis (LCA) 6, cone-rod dystrophy 13 (CORD13), or juvenile retinitis pigmentosa. With backing from the FDA and this award, Odylia is advancing toward the late-stage development of OT-004. If you are interested in reading more, click the link below. https://loom.ly/r-RPP4I #FoundationFightingBlindness #FFB #LCA #LCA6 #HopeInFocus #HIF #GeneTherapy #OT004 #CORD13 #Odylia

On World Duchenne Muscular Dystrophy (DMD) Day, I want to shed light on the crucial advancements in therapeutic research highlighted in the paper published in Nature in 2023 by Roberts and his collegues comprehensive review delves into transformative approaches such as gene therapy, exon-skipping, and CRISPR-based technologies, aimed at restoring dystrophin function—the key protein absent in DMD patients. These innovations offer new hope in slowing disease progression and enhancing muscle function. Gene therapies aim to address faulty genes by either replacing or repairing them, while exon-skipping methods are designed to circumvent genetic mutations, enabling the body to produce a functional version of the dystrophin protein. Additionally, CRISPR technology offers the potential to correct these mutations directly within the DNA, providing a more permanent solution. This review presents recent developments in therapeutic approaches for Duchenne Muscular Dystrophy (DMD), focusing on innovative techniques These efforts represent a significant leap forward in the treatment of DMD, a disorder that causes progressive muscle degeneration. Continued support for research and innovation is vital in our collective mission to better manage, or potentially cure, Duchenne Muscular Dystrophy. You can access the paper here: https://lnkd.in/dZ-C8HYe Roberts, T. C., Wood, M. J., & Davies, K. E. (2023). Therapeutic approaches for Duchenne muscular dystrophy. Nature Reviews Drug Discovery, 22(11), 917-934. DOI: 10.1038/s41573-023-00775-6 #WorldDMDday #DuchenneMuscularDystrophy #DMDResearch #GeneTherapy #ExonSkipping

𝗘𝘅𝗰𝗶𝘁𝗶𝗻𝗴 𝘀𝘁𝗿𝗶𝗱𝗲𝘀 𝗶𝗻 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆 𝗳𝗼𝗿 𝗣𝗮𝗿𝗸𝗶𝗻𝘀𝗼𝗻’𝘀 𝗗𝗶𝘀𝗲𝗮𝘀𝗲: https://lnkd.in/geViB34C At the Society for Neuroscience annual meeting, Capsida Biotherapeutics presented promising preclinical data on their CAP-003 gene therapy, showing potential for disease modification in Parkinson’s patients with GBA mutations. CAP-003 demonstrated a 200-fold increase in brainwide RNA expression over AAV9, with impressive improvements in GCase enzyme activity and reduced lysosomal dysfunction markers. With clinical trials anticipated in early 2025, this therapy could offer new hope for the 5-15% of PD patients impacted by GBA mutations. #IMAPAC #ParkinsonsDisease #GeneTherapy #Biotech #Innovation #Healthcare

#genetherapy Nonviral Ocular Gene Therapy for Retinal Disease Treatment A recent article published in Science Advances, from the researchers at The Johns Hopkins University School of Medicine, shows that nonviral gene therapy, utilizing biodegradable nanoparticles made from poly(β-amino ester)s (PBAEs), has successfully achieved widespread expression in both photoreceptors and retinal pigment epithelium (RPE) cells, offering therapeutic benefits in rodent models. PBAEs can condense expression plasmid DNA into nanoparticles (NPs) that are internalized by cells through endocytosis and are designed to be hydrolytically degradable. A significant portion of these NPs manage to escape from endosomes and decompose within a timeframe ranging from minutes to hours. This rapid degradation process allows the expression plasmids to enter the cell nucleus, initiating the expression of the introduced gene. When applied to larger eyes, similar to those of humans, in a study involving minipigs, an injection containing 50 μL of nanoparticles with 19.2 μg of a gene-carrying plasmid resulted in gene expression throughout the entire eye without causing harm. Although initial variations in the level of expression were observed, they became more uniform over time, thanks to refined injection techniques and plasmid adjustments. These results suggest that suprachoroidal gene delivery, without the use of viruses, is a viable strategy for targeting the entire retina in eyes comparable in size to human eyes, offering promising prospects for future retinal disease treatments. Link to the article: https://lnkd.in/gjGVMK2v Jordan Green Peter Campochiaro #retina #eyedisease #nanoparticles Novartis Gene Therapies GenSight Biologics Clearside Biomedical, Inc. Nanoscope Therapeutics Inc. CRISPR Therapeutics Locanabio, Inc. Boehringer Ingelheim AGTC Spark Therapeutics, Inc. Beacon Therapeutics Oxford Biomedica

"Gene therapy company Repair Biotechnologies has revealed promising preclinical results that demonstrate its technology rapidly reverses the progression of atherosclerosis in mouse models. The company says the development holds potential for treating both atherosclerosis and a rare genetic condition called familial hypercholesterolemia, in humans. Atherosclerosis is a condition characterized by the buildup of plaque in arteries, eventually blocking blood flow, and contributing significantly to heart disease, stroke, and death. In experiments, scientists at Repair Biotechnologies treated atherosclerotic mouse models with the LNP-mRNA therapy over a six-week period, with promising results." #genetherapies #hearthealth https://lnkd.in/eAUmrjz5

As we draw to the end of the year, the #lifescience, #biopharma, and #CGT industries are reviewing what happened in #2024, and looking forward to #2025. Here, Erin Harris, Chief Editor at Cell & Gene, the Life Science Connect publication, asks 3 KOLs about the outlook for 2025 in the #cellandgenetherapy sector. Miguel Forte, President, ISCT, International Society for Cell & Gene Therapy, and CEO, Kiji Therapeutics, and Vered Caplan, CEO, Orgenesis Inc. "As technology improves, and is doing so rather quickly, we will be better at accelerating the development and improving conditions of adoption and use, making the field delivering value to patients and stakeholders faster.” Miguel Forte, MD, Ph.D. Incorporating the advancements in automation, closed systems, quality control and IT solutions into a standard platform will make POC accessible for many institutes and enable a more efficient approach for the clinical development stage and will lay the basis for enhancing accessibility and reducing costs for commercial stage products.” Vered Caplan https://lnkd.in/eFWHqkpW