Rizwan Chaudhrey’s Post

A big step in realizing our strategy towards on integrated offering for viral vector manufacturing!

Intellia Therapeutics, Inc., a Cambridge-based leader in CRISPR-based gene editing therapies, and ReCode Therapeutics, a Menlo Park-based innovator in genetic medicines, have announced a strategic partnership to develop new gene editing treatments for cystic fibrosis (CF). This collaboration aims to leverage Intellia's CRISPR technology and DNA writing capabilities with ReCode's Selective Organ Targeting (SORT) lipid nanoparticle delivery system to target and correct CF-causing gene mutations. Initially focusing on patients with limited or no current treatment options, the alliance could expand to broader applications. Intellia will oversee the design of the editing strategy, while ReCode will handle preclinical and clinical development, and lead global commercialization for some programs, with Intellia retaining the option to lead U.S. commercialization for certain projects. This partnership underscores both companies' commitment to advancing gene therapy and offers hope for transformative treatments for those affected by CF. #intellia #recode #LNP #CRISPR #CF #cysticfibrosis

Recommendations for Method Development and Validation of qPCR and dPCR Assays in Support of Cell and Gene Therapy Drug Development The emerging use of qPCR and dPCR in regulated bioanalysis and absence of regulatory guidance on assay validations for these platforms has resulted in discussions on lack of harmonization on assay design and appropriate acceptance criteria for these assays. Both qPCR and dPCR are extensively used to answer bioanalytical questions for novel modalities such as cell and gene therapies. Following cross-industry conversations on the lack of information and guidelines for these assays, an American Association of Pharmaceutical Scientists working group was formed to address these gaps by bringing together 37 industry experts from 24 organizations to discuss best practices to gain a better understanding in the industry and facilitate filings to health authorities. Herein, this team provides considerations on assay design, development, and validation testing for PCR assays that are used in cell and gene therapies including (1) biodistribution; (2) transgene expression; (3) viral shedding; (4) and persistence or cellular kinetics of cell therapies. https://lnkd.in/eEnj2D4f

 🧬𝐌𝐢𝐥𝐥𝐢𝐩𝐨𝐫𝐞𝐒𝐢𝐠𝐦𝐚 𝐀𝐧𝐧𝐨𝐮𝐧𝐜𝐞𝐬 𝐂𝐥𝐨𝐬𝐮𝐫𝐞 𝐨𝐟 𝐌𝐢𝐫𝐮𝐬 𝐁𝐢𝐨 𝐀𝐜𝐪𝐮𝐢𝐬𝐢𝐭𝐢𝐨𝐧🔬 Exciting news coming from MilliporeSigma today, closing the acquisition of Mirus Bio for approximately $600 million after the completion of closing conditions. This move affirms their commitment to supporting their customers advancing cell and gene therapies throughout the life cycle, from preclinical through to commercial production. ► Sebastián Arana A., EVP & Global Head of Process Solutions stated "Mirus Bio’s advanced technology, combined with our bioprocessing expertise and broad portfolio, will enable us to deliver integrated solutions across the viral vector value chain and help meet the growing demand for these life-saving therapies.” Check out the full-press release below for more details 👇 https://lnkd.in/edjNu3JT #cellandgenetherapy #acquisition #viralvector #bioprocessing

Choosing the Right Delivery System: Viral vs. Non-Viral Whether you’re advancing therapeutics, modifying organisms for industrial applications, or diving into cutting-edge research, the choice between non-viral and viral delivery systems is crucial. While viral vectors offer high transduction efficiency, non-viral methods like electroporation have distinct advantages. As @James Brady, our Senior Vice President of Technical Applications & Customer Support, says, “Electroporationlowers production costs and improves scalability, making cell and gene therapy development more efficient and safer.” Learn more in this @Biocompare article: Viral vs. Non-viral Gene Delivery

Bayer Consumer Care AG and Asklepios BioPharmaceutical, Inc. (AskBio) have begun Phase II trials for AB-1002, a promising gene therapy for congestive heart failure. This marks a significant stride in addressing the unmet needs of heart failure patients, leveraging cutting-edge gene therapy technology. Quick Facts - 🔷 AB-1002 aims to treat non-ischemic cardiomyopathy and advanced heart failure symptoms. 🔷 The GenePHIT trial will rigorously test the safety and efficacy of this innovative treatment, potentially altering the landscape of heart failure management. Impact - Launching during Heart Failure Awareness Week, this trial spotlights the ongoing quest for groundbreaking treatments. It's a beacon of hope for millions battling this challenging condition. What's Next - As we await the outcomes, this development underscores the critical role of collaboration and innovation in advancing cardiac care. #HeartHealth #GeneTherapy #Innovation #ClinicalResearch

🧬 CRISPR Gene Therapy Casgevy Receives European Approval 🇪🇺 Casgevy's path to approval stands as a testament to the power of scientific exploration, daring risks, and unwavering determination. It all began with back-to-back green lights by the MHRA 🇬🇧 and the FDA 🇺🇸 in December 2023, heralding a transformative era in treating sickle cell disease and transfusion-dependent beta-thalassemia, ushering a new dawn for gene editing therapy and personalized medicine. Thus, it is no surprise that the EMA has granted a conditional marketing authorization to Vertex Pharmaceuticals and CRISPR Therapeutics this week, making Casgevy the first and only gene therapy approved in Europe for these conditions. 📣 Press release: https://lnkd.in/ekMbDRVi #Biotechnology #CRISPR #GeneTherapy #MedicalInnovation

📢 Flash BioSolutions expands its Gene Delivery RUO production capacity for C2 and C3 GMO-level viral particles!   To meet the growing demand for projects requiring containment levels 2 and 3 for GMOs , we are doubling our research-use-only (RUO) bioproduction capabilities! This expansion, operational by October 2024, positions us to better serve the rapidly advancing cell and gene therapy sectors across Europe and North America.   🎯 Our lentiviral products are essential for CAR and TCR research and the development of therapeutic vaccines, playing a key role in groundbreaking pharmaceutical developments in gene therapy and cancer immunotherapy. With over 11,000 lentiviral constructions completed in the past 20 years, we are equipped to support the next wave of breakthroughs in regenerative medicine and vaccination.   👉 Read the press release on our website: https://lnkd.in/ec6NPQTD   #CDMO #Biopharmaceuticals #GeneTherapy #CellTherapy #VaccineDevelopment #CARTCR

Choosing the Right Delivery System: Viral vs. Non-Viral Whether you’re advancing therapeutics, modifying organisms for industrial applications, or diving into cutting-edge research, the choice between non-viral and viral delivery systems is crucial. While viral vectors offer high transduction efficiency, non-viral methods like electroporation have distinct advantages. As @James Brady, our Senior Vice President of Technical Applications & Customer Support, says, “Electroporationlowers production costs and improves scalability, making cell and gene therapy development more efficient and safer.” Learn more in this @Biocompare article: Viral vs. Non-viral Gene Delivery

We're thrilled to announce the expansion of our Gene Delivery RUO production capabilities in France, doubling our capacity to meet the surging demand for C2 and C3 GMO-level viral particles. This milestone strengthens our commitment to supporting groundbreaking research in gene therapy, cancer immunotherapy, regenerative medicine, and beyond. At Flash BioSolutions, we produce over 1000 lentiviral constructs annually for research use, building on a legacy of 11,000+ constructions over two decades. Our lentiviral particles are critical for advancing: -CAR and TCR therapies that harness immune cells to target cancer. -Therapeutic vaccines aimed at both infectious diseases and cancer. -Regenerative medicine breakthroughs, where non-integrative lentiviruses play a key role in reprogramming and differentiation. This expansion positions us to serve the growing European and North American markets more efficiently, enhancing our ability to drive innovation globally. We're proud to support our clients on the frontlines of tomorrow's therapies. Together, we are pushing the boundaries of what's possible in gene and cell therapies! #France #Occitanie #Toulouse #CDMO #RUO #RD #GeneTherapy #CellTherapy #Lentivirus #Immunotherapy #RegenerativeMedicine #ViralVectors #FlashBioSolutions