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🔊 The FDA has granted approval to Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy as the first-ever CRISPR-edited therapy for beta-thalassemia. 🔊 This follows the earlier approval in December for treating severe sickle cell disease. Casgevy, co-developed by Vertex and CRISPR, is priced at $2.2 million for the one-time ex vivo treatment. The approval is for patients aged 12 and older who require regular blood transfusions to survive. Casgevy will compete with bluebird bio's Zynteglo in the beta-thalassemia market. The FDA's decision came earlier than the expected March 30 target date, marking a significant milestone in CRISPR gene editing technology. The EMA also recommended Casgevy for transfusion-dependent beta-thalassemia in mid-December. Vertex CEO Reshma Kewalramani, MD FASN expressed excitement about the approval and the potential benefits for patients with TDT. Full story: https://lnkd.in/g6mDDi-V

Gene&Cell Therapy >> AstraZeneca expands stake in cell and gene therapy partner Cellectis: UK pharma giant AstraZeneca on Monday unveiled another $140 million equity investment in cell and gene therapy partner Cellectis. AstraZeneca now owns 44% of Cellectis’ share capital and has 30% of the partner’s voting rights. Two AstraZeneca leaders, Alexion CEO Marc Dunoyer and corporate development executive director Tyrell Rivers, are also now on Cellectis’ board. AstraZeneca’s investment in its Paris-based partner was previously planned, the companies said Monday morning. In November 2023, AstraZeneca paid $25 million upfront and made an $80 million equity investment in the French and US biotech for a 22% stake. A month later, Cellectis took its CAR-T manufacturing in-house. Cellectis is using its gene editing technology to find up to 25 targets for AstraZeneca. The UK pharma can snag worldwide exclusive licenses on up to 10 of those for cell and gene therapies in oncology, immunology and rare diseases. Each of those comes with biobucks in the range of $70 million to $220 million. The higher end is roughly equal to Cellectis’ current market cap of $215 million. #lucidquest #genetherapy #celltherapy

Big News! Pfizer Inc. has announced that the US Food and Drug Administration (FDA) has granted approval to Beqvez™ (fidanacogene elaparvovec-dzkt), its gene therapy for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. According to Pfizer representatives, Beqvez has a price of $3.5 million, the same as Hemgenix® (etranacogene dezaparvovec) from CSL Behring, which was approved in 2022 for adults with hemophilia B. Emerging Therapy Solutions (ETS) can help payers and other risk-bearing entities with cost containment related to the pipeline of cell and gene therapies, and has contracted rates at Hemophilia Treatment Centers (HTCs). Reach out to ETS to learn more at 877.445.4822. https://bit.ly/4bcnutP

Lyophilization addresses stability challenges associated with complex APIs, biologics, microbiomes, gene therapies, viral vectors, liposomes and nanoparticles. This process is advantageous for parenteral drug developers. Learn how to overcome these challenges in our new lyophilization eBook. #lyophilization #formulationdevelopment #qualitybydesign #drugdelivery

Residual host cell genomic DNA (gDNA) is a critical consideration when developing and testing biopharmaceuticals made and purified from living (host) cells. Host cell gDNA must be removed to extremely low levels and manufacturers must demonstrate this clearance with highly sensitive methods such as quantitative PCR (qPCR). Because these tests are so sensitive the smallest changes in kits lot-to-lot or vendor-to-vendor can have a big impact on results. For reliable and consistent results, it's important to validate and run the methods with high-quality controls that are authenticated. USP and ATCC have collaborated to deliver high-quality, precisely quantitated gDNA controls for residual DNA detection of six cell substrates commonly used to manufacture cell and gene therapies, vaccines and proteins. To learn more, go to➡️ https://ow.ly/KLC050Q2Kxz #hostcell #gdna #genomic #innovation #quality #qpcr

Residual host cell genomic DNA (gDNA) is a critical consideration when developing and testing biopharmaceuticals made and purified from living (host) cells. Host cell gDNA must be removed to extremely low levels and manufacturers must demonstrate this clearance with highly sensitive methods such as quantitative PCR (qPCR). Because these tests are so sensitive the smallest changes in kits lot-to-lot or vendor-to-vendor can have a big impact on results. For reliable and consistent results, it's important to validate and run the methods with high-quality controls that are authenticated. USP and ATCC have collaborated to deliver high-quality, precisely quantitated gDNA controls for residual DNA detection of six cell substrates commonly used to manufacture cell and gene therapies, vaccines and proteins. To learn more, go to https://ow.ly/KLC050Q2Kxz #hostcell #gdna #genomic #innovation #quality #qpcr

Residual host cell genomic DNA (gDNA) is a critical consideration when developing and testing biopharmaceuticals made and purified from living (host) cells. Host cell gDNA must be removed to extremely low levels and manufacturers must demonstrate this clearance with highly sensitive methods such as quantitative PCR (qPCR). Because these tests are so sensitive the smallest changes in kits lot-to-lot or vendor-to-vendor can have a big impact on results. For reliable and consistent results, it's important to validate and run the methods with high-quality controls that are authenticated. USP and ATCC have collaborated to deliver high-quality, precisely quantitated gDNA controls for residual DNA detection of six cell substrates commonly used to manufacture cell and gene therapies, vaccines and proteins. To learn more, go to https://ow.ly/KLC050Q2Kxz #hostcell #gdna #genomic #innovation #quality #qpcr

Marked by two groundbreaking FDA approvals for cell-based gene therapies and high price tags, the recent strides in treating sickle cell disease present a paradoxical challenge. Authoring the article, "Battling Exorbitance: High Costs in Sickle Cell Gene Therapies and the Imperative of Global Patient Registries for Equity" at American Pharmaceutical Review, our Founder & CEO, Dr. Harsha K Rajasimha, Ph.D. emphasizes the indispensable role of global patient registries in enhancing inclusivity and accessibility in gene therapies. Modern advancements in clinical trial technology and the importance of government support are also explored to address vital affordability considerations. For more, visit: https://hubs.la/Q02spg5Z0 #GeneTherapies #SickleCellDisease #InclusiveRegistries #AccessibleHealthcare

✴️ Gene therapies based on viral vectors hold immense promise for treating a multitude of diseases. However, inefficient manufacturing methods and the absence of established standardized processes result in high costs and limited accessibility for patients. 💡 Global life sciences leader, Cytiva, is bringing solutions to these challenges with its new ELEVECTA cell lines. 🎯 Tapping into its deep expertise, the company has created three cell lines for adeno-associated virus (AAV) production. Together, the three lines comprehensively meet the distinct objectives of various therapeutic programs with the ability to transition between cell lines as needs evolve and can be paired with Cytiva’s established cell culture media offering. 👉 Find out more about the ELEVECTA cell lines at https://buff.ly/3Vc4Ega #GeneTherapy #gene #therapy #cell #patients #viralvector #manufacturing #biopharma #biotech #diseases #therapeutic

🧬mRNA Insider - Week 20   This week's mRNA insider discusses the potential of mRNA vaccines in preventing and managing the PRRS epidemic, offers a promising strategy to enhance the thermostability and storage longevity of mRNA/LNP systems, and demonstrates the impact of LNP formulation on mRNA transfection and gene editing, enhancing treatment safety while maintaining efficacy.   If you have any questions about mRNA or mRNA formulations in LNPs, feel free to contact us: https://lnkd.in/dyfmHhVg.   References 1. Zhou et al. 2024 https://lnkd.in/e8KcMYwD 2. Eygeris et al. 2024 https://lnkd.in/e4rF8cfk 3. Hashiba et al. 2024 https://lnkd.in/eF2eb4qA   #mRNAvaccine #genedelivery #lipiddesign #LNP #thermostability