Cell & Gene at ZS’ Post

Novartis has acquired Kate Therapeutics, a San Diego-based #genetherapy company, in a deal valued at up to $1.1 billion. Kate Therapeutics specializes in #preclinical programs targeting Duchenne muscular dystrophy (#DMD), facioscapulohumeral dystrophy (#FSHD), and myotonic dystrophy type 1 (#DM1). The company employs its proprietary DELIVER platform, which utilizes directed evolution of adeno-associated virus (#AAV) capsids to create #liver-#detargeted, muscle-tropic #vectors. Acknowledging recent challenges in muscular dystrophy treatments, such as Pfizer's investigational gene therapy for DMD failing to demonstrate efficacy in a Phase 3 trial, Novartis CEO Vas Narasimhan emphasized the company's commitment to learning from past experiences in the #musculardystrophy domain. By acquiring Kate Therapeutics, Novartis aims to leverage innovative #delivery #technologies to overcome previous obstacles in gene therapy for #neuromuscular #diseases. Muscular Dystrophy Association Muscular Dystrophy UK Muscular Dystrophy Canada Facioscapulohumeral Muscular Dystrophy Duchenne UK Duchenne Heroes #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation #geneediting https://lnkd.in/giRnS_6G

Gene & Cell Therapy Approvals Are Heating Up 2024 has seen a wave of new approvals in the cell and gene therapy space, with Rocket Pharmaceuticals, Adaptimmune, and Autolus Therapeutics pushing the envelope. Rocket's Kresladi, a gene therapy for a rare genetic disorder called leukocyte adhesion deficiency-I (LAD-I), is expected to be approved soon, offering a critical alternative to bone marrow transplants. Autolus Therapeutics' CAR-T therapy for B-cell acute lymphoblastic leukemia (obe-cel) is also poised for approval by the end of the year, showcasing how advanced gene therapies are progressing in oncology and rare diseases Why this matters to the industry? These advancements not only bring new treatment options for patients but also signify a growing confidence in gene therapies, creating more opportunities for biotechs working on similar treatments. The success of these therapies will likely lead to increased investment and development in the gene-editing space, particularly with

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

After years of dedicated research, an incredible milestone has been achieved in the field of gene therapies. Sarepta Therapeutics recently received accelerated clearance from the FDA for its groundbreaking treatment targeting Duchenne muscular dystrophy (DMD). Being involved in some DMD studies at Ncardia, this is exciting news and helps to substantiate that including human biology in the early stage of drug discovery helps to bring better therapies to patients faster 👏 The success of gene therapy depends on reliable models that can effectively predict the behavior of these therapies in human patients. Unfortunately, traditional animal models often fall short in replicating the intricate cellular and molecular mechanisms of human diseases. This discrepancy between preclinical and clinical studies poses a significant challenge. However, there is hope on the horizon 😊 Induced pluripotent stem cell (iPSC) technology is a promising solution that bridges the gap between preclinical and clinical studies in gene therapies. iPSC-based models not only provide valuable tools to investigate disease mechanisms but also offer a relevant platform for screening gene therapies. By leveraging iPSC technology, researchers and clinicians gain confidence in making complex decisions, such as selecting delivery systems and assessing safety and efficacy. This streamlined approach reduces the divide between preclinical and clinical studies, saving valuable time and resources ⏳ Collaborative efforts between organizations like Ncardia and gene therapy developers are crucial in driving therapy development forward. These partnerships facilitate the creation of disease models suitable for high-throughput screenings, accelerating the research process. For example, Ncardia employs human iPSC-derived cardiomyocytes and a multitude of molecular and functional readouts, such as multi-electrode arrays, high-content imaging, and qPCR, to optimize transduction efficiencies and validate the effectiveness of various AAV vectors for their clients. By harnessing these advanced tools and techniques, Ncardia plays a significant role in accelerating the go-to-market process of cutting-edge gene therapies, contributing to their development and transformative potential 🚀 Want to read more? Check out the full article on Ncardia's site - link in the comments ⬇ #genetherapy #drugdevelopment #ipsc

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

📊 Gene & Cell Therapy Landscape Report for Q3 2024 1️. New FDA Approval Marks Progress in Cell Therapy -Adaptimmune’s Tecelra (afamitresgene autoleucel) received FDA approval for synovial sarcoma. This MAGE-A4-directed, genetically modified autologous T-cell therapy highlights progress in personalized immunotherapy. 2️. Gene Therapy Trials Shift Towards Non-Oncology Indications -Non-oncology trials are on the rise, with 51% of gene therapy trials in Q3 2024 targeting non-cancer indications, up from 39% in Q4 2023. -Oncology remains prominent, as 52% of all gene therapy pipeline assets and 53% of rare disease gene therapies focus on cancer indications. 3️. Dealmaking Flat, but Start-up Financing Sees a Rebound -Q3 saw 101 deals in the advanced molecular therapy space, similar to Q2, with four notable acquisitions, including Agilent’s $925M acquisition of Biovectra in the CDMO sector. -Seed and Series A funding tripled to 19 deals, raising $484M in Q3—a nearly 2x increase over Q2. 📈 Data Source: Pharmaprojects | Citeline, October 2024 #GeneTherapy #CellTherapy #Biotech #FDAApproval #ClinicalTrials #Pharma

💎💎💎Whatsapp!! 💎💎💎Xcell Biosciences Expands AmplifyBio Gene Therapy Manufacturing Collaboration Xcell Biosciences expanded its collaboration with AmplifyBio, a CDMO, by installing a new AVATAR™ Foundry system as part of Xcellbio’s beta access program. The new instrument will enable AmplifyBio’s team to advance from small-scale workflows on the previously installed AVATAR™ Odyssey platform to achieve automated cell therapy manufacturing workflows suitable for clinical use, according to an Xcellbio spokesperson. Xcellbio and AmplifyBio have an ongoing collaboration aimed at streamlining and improving the manufacturing process for engineered #T_cell #receptor (TCR) #therapies #targeting #solid #tumors, which have proven difficult to treat with cell #therapies due to the #immunosuppressive #tumor #microenvironment. Through this collaboration, scientists are working to identify key elements for successful manufacture of TCR therapies, such as the relationship between various T cell conditioning #matrices and product characteristics such as #phenotype, #metabolic #profile, and #potency. Experiments already have progressed from in vitro to in vivo, and an IND filing is expected next year to target human #papillomavirus-positive tumors using a conditioned, engineered #TCR #therapy, continued the Xcellbio official. Xcellbio developed the AVATAR #platform for cell and gene therapy R&D with the AVATAR Odyssey system designed for small-scale research and process development needs. Its latest platform, the AVATAR Foundry system, is a cGMP manufacturing platform that delivers novel capabilities for improving the potency of cell and gene therapies, said J. Kelly Ganjei, president, and CEO of AmplifyBio. “Building on our experience with the AVATAR Odyssey system that is providing us with the tools to #optimize various T cell conditioning #protocols, we are excited to get access to the AVATAR Foundry system so we can progress our client’s research efforts toward automated, clinical-scale manufacturing of cell products,” continued Ganjei. ➡ 💎 You can find more pieces of work by clicking here. https://lnkd.in/eSG67K5G #T_cell #receptor

💥 Another Huge Week for Cell & Gene Therapy: Roche Drops $1.5B, Following Novartis’ $1.1B Move ❗ Big deals are making waves in the biotech world! Roche has just agreed to acquire Poseida Therapeutics, Inc. for $1.5 billion, doubling down on its mission to bring off-the-shelf CAR-T therapies to the masses. But that’s not all—this comes hot on the heels of Novartis’ $1.1 billion deal to acquire Kate Therapeutics just last week. Together, these deals signal a surge of confidence in the Cell & Gene Therapy space. Let’s break down why this matters: 👉 Roche & Poseida: Roche is betting big on Poseida’s platform to scale CAR-T therapy. Their off-the-shelf approach, powered by donor-derived T stem cell memory cells, could make advanced treatments faster, safer, and more accessible to the 80% of eligible patients currently missing out. 👉 Novartis & Kate: Novartis’ acquisition adds muscle to its CGT pipeline, bolstering its position in the fight against rare diseases and building on its proven expertise in delivering groundbreaking therapies like Kymriah. Both deals highlight a shared goal: democratising advanced therapies. CGT is no longer just about niche breakthroughs—it’s about making life-changing treatments available to broader populations. 🤑 With $2.6 billion in CGT deals announced in a single week, it’s clear the industry is heading into 2025 with momentum. The question now is whether these innovations will live up to the hype. What do you think—is this the start of a new golden age for CGT?

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