Journal of Chromatography A’s Post

𝑭𝑫𝑨 𝑮𝒓𝒂𝒏𝒕𝒔 𝑭𝒂𝒔𝒕 𝑻𝒓𝒂𝒄𝒌 𝑫𝒆𝒔𝒊𝒈𝒏𝒂𝒕𝒊𝒐𝒏 𝒕𝒐 𝑩𝒂𝒚𝒆𝒓 𝒂𝒏𝒅 𝑨𝒔𝒌𝑩𝒊𝒐'𝒔 𝑮𝒆𝒏𝒆 𝑻𝒉𝒆𝒓𝒂𝒑𝒚 𝒇𝒐𝒓 𝑷𝒂𝒓𝒌𝒊𝒏𝒔𝒐𝒏’𝒔 𝑫𝒊𝒔𝒆𝒂𝒔𝒆  Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio) announceD that the FDA has granted Fast Track Designation for AB-1005, a promising gene therapy for moderate Parkinson’s disease. AB-1005 also received the Innovation Passport from the UK MHRA. 🌍 AB-1005, an investigational AAV2-GDNF neurorestorative gene therapy, is making strides in treating moderate Parkinson’s disease. Earlier this year, AskBio shared positive 18-month Phase Ib clinical trial results, achieving the primary goal of evaluating the safety of a one-time bilateral delivery directly to the putamen.💡 https://lnkd.in/dvya45vz AskBio is hiring for a Clinical Trial Manager (Remote) https://lnkd.in/d74TtC8D #GeneTherapy #ParkinsonsDisease #Innovation

Regeneron Pharmaceuticals signs Gene Editing Deal with Mammoth Biosciences: 🧬Regeneron Pharmaceuticals is collaborating with Mammoth Biosciences to develop in vivo CRISPR-based gene editing therapies, enabling therapeutic interventions directly inside the body that could potentially offer single-injection cures for various diseases 🧬The partnership builds on Regeneron's existing work in gene therapies, including their ongoing collaboration with Intellia Therapeutics, Inc. on CRISPR/Cas9 gene editing therapies 🧬This marks Mammoth's third major pharmaceutical industry partnership following collaborations with Bayer and Vertex Pharmaceuticals 🧬The agreement stipulates an upfront payment of $100 million from Regeneron, with potential for up to $370 million in development, regulatory, and commercial milestones per target, plus sales royalties. Additionally, Mammoth has the option to co-fund and share profits in most programs, rather than solely receiving milestones and royalties. 🧬Regeneron will contribute its expertise in using specialized viruses to precisely deliver gene therapies, while Mammoth provides its advanced, compact gene-editing tools. 🧬 Mammoth, initially focused on CRISPR-based diagnostics during the Covid-19 pandemic, is shifting more towards therapeutics, with its most advanced internal program targeting two liver diseases. 👇Link to articles in comments below #DigitalHealth #CRISPR 

AskBio presents 18-month phase Ib trial results of AB-1005 gene therapy for patients with Parkinson’s disease

Check out the latest article we created for our partner BAP Pharma on our Pharmaceutical-Technology website - Medicines access: Unlocking the immense potential in cell and gene therapy  Despite several ground-breaking developments, pharmaceutical and biotech companies are often reluctant or unable to invest in cell and gene therapy. Here, we discuss the key challenges to access and how the potential of these innovative treatments can be unlocked. - https://lnkd.in/djQmDiqb If you would like more information about our award winning content and account based marketing solution, please request a demo here: https://lnkd.in/e8wNB3cj #pharma #marketaccess #medicinesaccess #healthcaremarketing #digitalmarketing #contentmarketing #abm #accountbasedmarketing

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🌟 Exploring the Future of #RareDisease Treatment: Cell and Gene Therapies Market 🌟 DelveInsight Business Research LLP's latest report dives into the transformative landscape of Cell and Gene Therapies in Rare Disorders Market. With groundbreaking innovations in gene editing, cell-based treatments, and personalized #medicine, this report sheds light on emerging therapies aimed at revolutionizing rare disease management. The leading Cell and Gene Therapies in Rare Disorders Companies such as Pfizer, Sangamo Therapeutics, Inc., Roche (Spark Therapeutics, Inc.), Freeline Therapeutics, 4D Molecular Therapeutics, Astellas Pharma Gene Therapies, Actus Therapeutics (Acquired by Bayer), GenSight Biologics, Coave Therapeutics, Johnson & Johnson/MeiraGTx, Applied Genetic Technologies Corporation, Nanoscope Therapeutics Inc. jCyte, Inc., ReNeuron Group Plc, REGENXBIO, Amicus Therapeutics, Sarepta Therapeutics, Capricor Therapeutics, Inc./Nippon Shinyaku, BrainStorm Inc. Cell Therapeutics, CRISPR Therapeutics/Vertex Pharmaceuticals, Editas Medicine, Krystal Biotech, Inc., Abeona Therapeutics, Castle Creek Biosciences, Inc., Holostem Terapie Avanzate S.r.l., RHEACELL GmbH & Co. KG, Ishin Pharma, Anterogen, Ultragenyx Pharmaceutical, and others. 🔬 Uncover how novel therapies are opening doors to improved outcomes and hope for patients worldwide @ https://lnkd.in/djeg783R #CellAndGeneTherapy #RareDisorders #GeneTherapy #MarketResearch #HealthcareInnovation #DelveInsight

Metagenomi Announces Preclinical Data for Lead Hemophilia A Program Demonstrating Durable Factor VIII (FVIII) Activity Levels through Twelve Months Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, recently announced data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001. The twelve-month durability data from Metagenomi’s ongoing nonhuman primate (NHP) study in hemophilia A remains generally consistent with data previously released at 4.5 months. The NHPs remain healthy and exhibit normal weight gain. The treatment is generally well tolerated and the program is on track for IND filing in 2026. https://lnkd.in/g3XAnmgQ

Nucleus Biologics Launches NB-KUL™ DF: A DMSO-Free, Chemically-Defined Cryomedia Revolutionizing Cell and Gene Therapy Cryopreservation - https://lnkd.in/g5DU7MGg  Nucleus Biologics, a leading provider of cell culture and bioprocessing solutions for the cell and gene therapy (CGT) industry, has launched NB-KUL DF, a DMSO-free, chemically-defined cryomedia set to redefine cryopreservation standards. Designed for CGT manufacturers, NB-KUL DF matches DMSO-based media performance and outperforms other DMSO-free options in cell viability, recovery, and expansion. While DMSO is an effective cryoprotectant, its toxicity compromises cell viability, can cause patient issues, and requires complex wash steps, adding cost and variability. NB-KUL DF eliminates these issues, offering a DMSO-free solution that preserves cell integrity, eliminates the potential for patient side effects, and simplifies cryopreservation making it ideal for T cells, MSCs, and other sensitive cell types used in CGT. Key Benefits of NB-KUL™ DF: - Supports Viability and Recovery in Multiple Cell Types: NB-KUL DF ensures higher post-thaw viability and recovery than competitive cryomedia, optimizing therapeutic cell preservation for better clinical outcomes. - Enhanced Expansion: Supports superior cell expansion when compared to competitive medias, meeting the demands of large-scale CGT manufacturing. - DMSO-Free: Eliminating DMSO reduces cytotoxicity, protecting cells and patients, especially in high-dose or immunocompromised therapies. - Streamlined Workflow: Simplifies processes by removing the need for DMSO washing steps, reducing cell loss, variability, time, and costs. #celltherapy #genetherapy #research #ryopreservation David De Leonardis Brad Taylor

Thoughts on this? >> Philadelphia Cell and Gene Therapy Conference hosts FDA officials - The Philadelphia Inquirer >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #biotech #pharma #competitivemarketing #healthcare #pharmaceutical

Are you designing an early-phase clinical trial for your cell & gene therapy (CGT) product? Unsure of how to approach the trial design? Early phase trial designs for CGT products depend on the product's distinctive features, previous clinical experience and manufacturing logistics. Inevitably, these are significantly different (and may be more complex） than those of small molecules. It is important for sponsors to consider clinical safety issues, preclinical issues, and CMC related issues for CGT products. Read our blog that covers the key considerations when designing early-phase clinical trials for CGT products https://lnkd.in/dRzXEzcx Discuss your regulatory requirements for designing CGT clinical trials 🤝 Meet us at BIO International 2024: https://lnkd.in/gJt7Vxwv 🤝 Meet us at DIA Global 2024: https://lnkd.in/grrwWGU6 #cell #gene #therapy #clinical #trial #regulatory #BIO2024 #DIAGlobal #pharma #biologic #CMC #preclinical #safety