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📢 #HighlyCitedPapers 1. #Efficient Gene Transfection by Electroporation—In Vitro and In Silico Study of Pulse Parameters 🔗 https://lnkd.in/gCBnzbg4 2. Polymeric #Nanoparticles—Tools in a Drug Delivery System in Selected Cancer Therapies 🔗 https://lnkd.in/gKZbrgG8 MDPI Encyclopedia MDPI

New Guidances Say Gene Therapies Require Strong Preclinical Testing . . Strong nonclinical testing should underpin development programs for both gene editing (GE) products and chimeric antigen receptor (CAR-T) cellular products, according to two new final guidances issued by the FDA. Since both of these therapeutics involve genetic alterations of living human cells and potentially, the introduction of viral vectors, sponsors need a firm foundation of in vitro and in vivo testing showing that they are safe and exert limited off-target activity, both guidances say. CAR-T for non-oncology indications is not covered. The guidance “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products” focuses on the manufacturing and characteristics of CAR-T cells for oncology indications, including hematologic malignancies and solid tumors. Gene editing products are complex products that can include the gene editor, DNA targeting elements and a donor DNA template, according to “Human Gene Therapy Products Incorporating Human Genome Editing. #newguidance #genetherapy #preclinicaltesting #regulation #regulationupdate #regulatorycompliance

What's next for CRISPR in 2024, and what are its main challenges coming into the New Year? Jasper Morley discusses below: “Challenges in this space include how to develop a fair pricing strategy, the logistics of offering broad access to the most needy populations in underserved markets (such as sickle cell patients in Africa), and potential unfamiliar adverse events associated with this novel modality of therapy. Vertex and CRISPR’s Casgevy will run $2.2 million for a one-time treatment for both SCD and TDT. That sticker price is in line with other novel gene therapies, such as bluebird bio’s Lyfgenia, which was also approved by the FDA for SCD and will cost $3.1 million. Yet one study of SCD patients found the total cost of disease over a patient’s lifetime was over $5 million." Read more below:

xcellbio just announced a collaboration with ElevateBio to explore novel approaches towards improving therapeutic potency of cell and gene therapies. As part of this collaboration, ElevateBio becomes the first member of Xcellbio’s beta program for its new AVATAR Foundry™ device. As part of this collaboration, ElevateBio BaseCamp scientists will have access to Xcellbio’s AVATAR™ incubator system for cell therapy research and development as well as two new platforms: the AVATAR Ai™ system for measuring the potency of cell therapies and the AVATAR Foundry system for cGMP cell therapy manufacturing. The AVATAR and AVATAR Foundry systems are used to metabolically reprogram therapeutic cells to improve their potency and persistence in the tumor microenvironment (TME), while the AVATAR Ai provides real-time analysis of tumor cytotoxicity under TME conditions. These collaborative efforts should streamline manufacturing protocols for cell and gene therapies. https://lnkd.in/epz98hXB #cellandgenetherapy

𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐓𝐨𝐝𝐚𝐲: 𝐊𝐞𝐲 𝐔𝐩𝐝𝐚𝐭𝐞𝐬 𝐢𝐧 𝐂𝐞𝐥𝐥 𝐚𝐧𝐝 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 – April 26, 2024 👉In recent biotechnology developments, the FDA has granted approval to Lifileucel, marking it as the first cellular therapy for cancer using tumor-infiltrating lymphocytes (TILs). This therapy, initially targeting melanoma, demonstrates significant potential in treating solid tumours, although its use is currently limited by the robust pre- and post-treatment care required. 👉Meanwhile, gene therapy continues to make strides with notable approvals and ongoing developments. The FDA has recently approved Casgevy, a CRISPR-based gene editing therapy for sickle cell disease and beta-thalassemia. This therapy, which alters DNA in a patient’s blood cells, carries a hefty price tag of $2.2 million but offers a permanent solution for patients suffering from these genetic disorders. 👉The sector anticipates further advancements with the possibility of up to 17 new approvals in the U.S. and EU by the end of 2024, driven by rapid advancements and high financial stakes in gene and cell therapies. These therapies are among the fastest growing sectors in biomedicine, with substantial investments flowing into their development. 🌐 Follow me to stay updated with the latest in biotech innovations

Read 📖 about Quantum Media Group, LLCs client Genprex, Inc. and their innovative research 🔬 in this TechBullion article by Angela Scott-Briggs: 👉 https://lnkd.in/gzsWMjAb 🧬 Genprex is committed to and focused on patient welfare; which it places at the core of its mission. Genprex’s overall objective is greater than providing effective treatments for diseases, it’s about the patient’s quality of life. 👉🏻 “Genprex, a dedicated biopharmaceutical firm, is at the forefront of innovating cancer treatment, including treatments for lung cancer. Central to its approach is gene therapy, aimed at replacing the missing protein encoded by tumor suppressor genes that are missing in many lung cancers. This technique aspires to elevate treatment efficacy while minimizing adverse effects.” 💬 What are your thoughts on gene therapy? Share them in the comments! #biotech #innovation #genetherapy #healthcare

Roche is collaborating with Ascidian Therapeutics to develop #genetherapies for challenging #neurologicaldiseases. This move helps Roche diversify as it faces declining #oncology sales. Key Points: ▶ Innovation - Utilizing Ascidian's RNA exon editing technology. ▶ Financials - $42M initial payment, up to $1.8B in milestone payments, plus royalties. ▶ Impact - Advancing treatments with reduced risks compared to traditional gene therapy.

The recent announcement by Vertex Pharmaceuticals that they have halted the development of their CRISPR-based gene-editing therapy for Duchenne Muscular Dystrophy (DMD) is indeed a significant disappointment. Families have been looking for this and other CRISPR therapy to cure this devistating disease.. The suspension not only underscores the complexities and challenges associated with developing CRISPR-based treatments but also marks another setback in the fight against DMD. It reinforces the urgent need for the medical research community to continue investing in a diverse portfolio of treatment strategies, including non-CRISPR approaches, to ensure that progress in combating this disease does not stall. Our collective commitment to innovation and the exploration of all potential pathways is essential to one day providing effective treatments to all those affected by DMD. Excerpt from Vertex Press Release "Vertex’s plans to enter the notoriously tricky Duchenne muscular dystrophy (DMD) space also seem to be stalled for the time being. A week after Sarepta Therapeutics saw its stock tank when its approved DMD gene therapy Elevidys failed to hit the primary goal of a pivotal study, Vertex said it won’t be rushing into the clinic in this indication anytime soon. Vertex has been investigating whether the muscle degenerative condition can be treated by delivering CRISPR/Cas9 gene editing technology to the muscle cells with a virus, AAV9. The aim is to change the targeted DNA sequence to restore dystrophin protein expression."

CARGO Therapeutics seeks $218 million IPO (when 130 biotechs are still stuck in IPO backlog) The IPO market is frozen over. Investors are looking for surer bets, investing in biotechs with clinical data. And Cargo Therapeutics may be the last #IPO of 2023. With less than 12 months of cash in the bank, this IPO will help extend the company’s runway into 2025 and fund its clinical activities. But even then, the challenge is far from over. Newly public companies like Adlai Nortye Biopharma Co., Ltd and Neumora are struggling, and trading below IPO price. The #biotech market took a huge hit last month when the XBI dropped to a 52-week low. And #investors are hoping next year will bring a fresh wave of listings.

The global #Biotech Industry was above average in terms of performance last year. It was full of innovations, new technologies entered the market, new approvals such as first CRISPR gene therapy. 2024 alone is projected to see the launch of up to 21 cell therapies and 31 gene therapies. Personalized mRNA vaccines in cancer (such as melanoma, pancreas, and others) will be an emerging therapy this year. New trends like microbiome research, AI integration & personalized medicine are driving the #biotechnology industry growth and innovation going forward. Through responsible navigation of the obstacles and harnessing of the enormous potential of these developments, the industry can build a more promising future in which biotechnology-driven solutions enhance patient outcomes globally.