Daniel DeVido

🗞 Breaking news 🤩 Genezen acquire uniQure's commercial GT manufacturing ops in Lexington!! This strategic acquisition will enable Genezen to deliver late-phase and commercial gene therapy development and manufacturing services to customers around the world. Check out full article below👇 #CDMO #Genezen #GeneTherapy

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#SABCS24 Following the FDA approval of Inavolisib, SABCS24 will showcase data on various PI3Ka inhibitors. After initial clinical data for RLY-2608 was presented in the September conference call and for STX-478 at ESMO24, updated Phase 1 data for both drugs will be shared at SABCS24. Although the clinical development of LOXO-783 was discontinued, SABCS24 will provide the first opportunity to review its clinical data. Additionally, the preclinical characterization of LY4045004, a new PI3Ka inhibitor being developed based on the lessons learned from the LOXO-783 program, will also be presented. Unlike the previously mentioned mutant-selective PI3Ka inhibitors, preclinical data will also be presented for BBO-10203, a PI3Ka:RAS interaction inhibitor. Recently, BBO-10203 was administered to the first patient in the Phase 1 BREAKER-101 study.

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The roll out of MoCRA continues. Mandatory facility registration and product listings under the Modernization of Cosmetics Regulations Act went into effect today, July 1. Also noted that while the FDA hasn't yet provided detailed guidance related to MoCRA's safety substantiation requirements, the essence of this requirement is already in effect and fully enforceable by the FDA. Affected cosmetic companies are advised to be able to provide safety data for their products "derived from scientifically robust methods." Being able to demonstrate that your cosmetic-related facility is in a state of control has always made good business sense; now it's a regulatory requirement.

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For anyone building diagnostics: Very cool approach to gain valuable insights into FDA decision summaries. Brendan O'Leary scraped the FDA’s decision summaries and provided some simple R code for anyone to get up and running in no time to prod and probe those summaries. Very nice work!

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Dear Friends, while we are all looking deeply and more deeply into the Revision 2 of the FDA’s Guidance for Industry, “Control of Nitrosamine Impurities in Human Drugs”, a significant change in the content of the website (https://lnkd.in/eYXSx-Gu) in the section, “Recommended Safety Testing Methods for Nitrosamine Impurities” of the website, an update that may merit looking with new eyes. This section says, “In addition to the enhanced Ames test, the Agency is currently requesting a second in vitro mammalian cell mutation assay and in vitro metabolism data (including human hepatocyte or microsome) to support an AI limit of 1500 ng/day." Scary, isn't it!! Chuck Lambert of Intrinsik and I put some thought into this and came up with some ideas of what may be needed in the form of in vitro testing beyond the modified Ames Test to derisk a nitrosamine. Please visit the BLOG at my Raaha LLC website. Just to let you know, this is "food for thought" as we wait for more clarification from FDA and should be taken only as a possibility. https://lnkd.in/eh5Y6kjW #nitrosamines, #pharmaceuticals, #carcinogenic, #genotoxic, #impurities, #FDA, #FDAguidances, #API, #drug

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oops! Gene editing needs to be undone, pronto...You can't make this up. DARPA on X: "We are soliciting innovative proposals to develop and demonstrate rapid methods to identify and optimize #novelmolecules that counteract gene editing technologies. To learn more, visit https://t.co/3L2HAUQgFy #BiotechInnovation #InnovationInScience https://t.co/or9N5S5aD6" / X

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Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP) to Discuss Second Quarter 2024 Financial Results on August 9, 2024 Conference Call and Provide Business Update   Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP), a clinical stage #biopharmaceutical company focused on developing new #antibiotics for difficult-to-treat #bacterial infections, has announced a conference call to discuss its second quarter 2024 financial results. The call is scheduled for August 9, 2024, at 8:00 am ET, before the U.S. financial markets open.   The company's President and CEO, David Luci, along with CFO Robert Shawah, will host the call to present the #financial results and provide a business update. #Investors and interested parties can join the call using the provided toll-free number for U.S. participants or international access numbers.   https://lnkd.in/esMuCywW #nasdaq #acxp #antibiotics #acurx Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP) David Luci

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DC Chemicals developed high purity of LP-01 for mRNA delivery,purity>98% https://lnkd.in/gzVpaB9y LP-01 is an ionizable cationic amino lipid (pKa = ~6.1). It has been used in the generation of lipid nanoparticles (LNPs). LNPs containing LP-01 and encapsulating both Cas9 mRNA and modified single-guide RNA (sgRNA) for the transport protein transthyretin (Ttr) induce gene editing in liver cells in mice in a dose-dependent manner resulting in reduced serum Ttr levels for at least 12 months. References: Finn, J.D., Smith, A.R., Patel, M.C., et al. A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep. 22(9), 2227-2235 (2018).

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Regeneron confirms FDA won’t approve myeloma drug over manufacturing issues The company expects a reinspection in the ‘coming months’ for a facility run by third-party manufacturer. After warning investors earlier this month of a possible delay, Regeneron Pharmaceuticals on Tuesday confirmed that U.S. regulators will not approve its experimental multiple myeloma drug at this time. The Food and Drug Administration previously identified flaws at a third-party fill/finish manufacturing facility. And while the manufacturer believes the issues have been resolved, a new inspection must take place before Regeneron can win approval of its medicine. The inspection is likely to happen in “coming months,” the company said. Regeneron emphasized the manufacturing issue was the only one related to approvability of its drug, called linvoseltamab. Executives told investors during an Aug. 1 conference call that they have not been informed by the FDA of any safety or efficacy concerns. While not unexpected, the FDA’s complete response letter represents a delay for Regeneron, which is vying to enter an already competitive market for a type of cancer treatment known as a bispecific antibody. Johnson & Johnson won FDA approval to sell its option, Tecvayli, for multiple myeloma in October 2022. And Pfizer in August 2023 got clearance to enter the market with a treatment called Elrexfio. The drugs work by targeting both diseased cells and the T cells that spur an immune response. Regeneron is hoping to distinguish linvoseltamab from already approved medicines by showcasing an administration schedule that requires less time in the hospital. And while it’s difficult to compare clinical trials run by different companies, Regeneron may have a slightly stronger data set to show doctors, according to a company presentation. https://lnkd.in/e9icn4Nu

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🔍 Navigating the New FDA-LDT Rule: Winners and Losers in Diagnostic Laboratory Testing 🥇🥉 📖As the FDA introduces stricter regulations for Laboratory Developed Tests (LDTs), the implications for established and emerging diagnostic laboratories are starkly different. 🏆 Strategic Advantages for Established Labs: 📌 Grandfathering Provision & NYS CLEP carve out: The FDA’s grandfathering clause allows on market LDTs and those approved by NYS CLEP to operate under enforcement discretion. This distinctly benefits established labs that have a broad portfolio of on market tests, providing them stability and continuity that new labs lack. Myriad Genetics' latest earnings call, featuring insights from COO Sam Raha reveals how established laboratories may emerge as winners: https://lnkd.in/daMqhZGS 📢Favorable Regulation: "Our current view is that this finalized version of the rule is more favorable than the preliminary guidance," notes Raha. 📢Compliance and Experience: "All of our on-market tests meet CAP/CLIA, New York State requirements...we have a strong quality assurance regulatory affairs organization," Raha adds, highlighting Myriad's preparedness and long-standing FDA collaboration. 📈 Strategic Insight: The regulatory shift is likely to create a bifurcated market where established diagnostic laboratories can capitalize on their entrenched positions and regulatory expertise. 👉Established laboratories are likely to face fewer disruptions compared to startups, which must navigate complex regulatory frameworks from scratch. 👉Established laboratories generally have more extensive resources and infrastructure to manage regulatory changes, which can be a significant hurdle for startups with limited capital and smaller teams. 🤔 How will this affect innovation in the diagnostics industry?… #Diagnostics #FDALDTRule #BusinessStrategy

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Demonstrating the technical leadership of SK pharmteco! 🧬 Catch up with our team at ASGCT to discuss how SKPT can support your #cellandgene development!

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An important update on the #BiosecuresAct, which includes a grandfather clause on existing contracts thru 2032, but this new draft prohibits setting up new agreements or extending existing ones. While balancing national security, also to ponder is "the potential fallout could have negative impacts on the supply chains of several drugmakers." https://lnkd.in/ebcE6x4w #Policy #Biotech #Innovation #GovernmentalAffairs #Pharma #Healthcare

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DC Chemicals can provide the key lipid components :Acid-degradable Cationic Lipid (ADC) Lipid 132 and lipid III-45 which were used in the referenece https://lnkd.in/gDxNe6gH https://lnkd.in/gDa4bSCx https://lnkd.in/g5wz4jDt A study by Jennifer A. Doudna's team at UC Berkeley, published in Nature Biotechnology on October 16, 2024, introduces a method for gene editing in the lungs and liver using LNPs to deliver stable CRISPR-Cas9 RNPs, addressing limitations in delivery methods. Background: CRISPR-Cas9 faces delivery challenges, with LNPs showing promise but limited in delivering CRISPR components beyond the liver. GeoCas9's thermal stability makes it suitable for LNP delivery but has faced efficiency and PAM sequence issues. Results: GeoCas9 Evolution: Directed evolution produced iGeoCas9 variants with significantly higher editing efficiency and broader PAM compatibility. LNP Delivery: Optimized LNPs improved iGeoCas9 RNP delivery, enabling efficient in vitro gene editing and site-specific integration via HDR. Enhanced Efficiency: New LNP formulations, FX12 and FC8, showed high RNP delivery efficiency and low cytotoxicity, outperforming mRNA+sgRNA-LNP delivery at low doses. In Vivo Editing: Tissue-selective LNPs enabled efficient liver and lung editing in mice, with high efficiency in editing the PCSK9 and SFTPC genes. Discussion: The study presents iGeoCas9 and optimized LNPs as a new CRISPR gene therapy platform, offering reduced immunogenicity and cytotoxicity, and adjustable for organ-specific targeting. Methods: The paper outlines methods from plasmid construction to in vivo gene editing, including LNP and RNP preparation, and assessment of delivery and immunogenicity. Conclusion: The iGeoCas9 RNP-LNP complex is a promising, efficient, and low-toxicity delivery method for CRISPR gene therapy with broad applications.

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Looking for a reliable partner for your pharmaceutical development needs? Seven Star Pharmaceutical Services (Seven Star) has got you covered. Our team specializes in CMC/solid-state chemistry research, crystallization process development, pre-formulation studies and formulation development studies. We also offer comprehensive routine and focused analytical testing services, ensuring accurate and timely results without any compromise on quality. Contact us to learn more about our services. https://lnkd.in/etWhi36 #chemistry #research #pharmaceutical #CMC #drugdevelopment #pharmaceuticaldevelopment #SolidStateChemistry #SaltScreening #CrystallizationScreening #CocrystalScreening #PolymorphScreening #preformulations #formulationdevelopment #MedicinalChemistry #SingleCrystalXrayDiffraction #materialsscience #analyticalresearch #materialsresearch #cdmo #CRO #contractresearch #drugdiscovery #api #crystalform #pharmaceuticaldevelopment 

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Perhaps you should consider the lightning-fast, cost-effective, non-compartmental PK analysis and reporting offered by Aplos Analytics, Inc.. Aplos can have you up and running in less than a week (actually, in a day if you happen to be in a hurry). Once set up, you simply enter your parameters and with the click of a button, receive validated data and comprehensive reports in seconds. Since it's cloud-based, there is nothing to install, and training is minimal. If your organization is already doing NCA, Aplos allows you to free-up those resources. Expand your service offerings quickly and cost-effectively with Aplos Analytics. #pharmacokinetics #pk #bioanalysis #pharmacology #toxicokinetics #aplosanalytics

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Go non-viral, lipid nanoparticle (LNP) stay safe #GT #CRISPR ATTR, or Transthyretin Amyloidosis, is a rare but serious condition caused by the misfolding of the transthyretin (TTR) protein. This misfolding leads to the accumulation of amyloid deposits in various organs and tissues, most commonly affecting the heart and nervous system. There are two main types of ATTR: Hereditary ATTR (hATTR): Caused by genetic mutations in the TTR gene, passed down through families. Wild-type ATTR (wtATTR): Occurs due to age-related misfolding of the normal TTR protein, not associated with genetic mutations. NTLA-2001, is designed to target and deactivate the TTR gene in liver cells, the primary site of TTR protein production NTLA-2001 delivers sgRNA & Cas9 into the nucleus. This allows to edit the TTR gene thus inactivating it precisely. Once inactivated - it prevents the production of the TTR protein 0.1 mg/kg dose of NTLA-2001, which led to a 52% median reduction in serum TTR by day 28

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It's another Wednesday and we open the gift that keeps on giving...Warning Letter Wednesday! In today's list, we have a facility that produced a (contaminated) material, then kept retesting until it finally passed enumeration tests: "Your firm delayed the shipment of the failing batches and kept testing until the added preservative decreased the microbial count below the limits. This is in direct contrast to the United States Pharmacopeia (USP) Chapter <51> Antimicrobial Effectiveness Testing, which states, “Antimicrobial preservatives should not be used as a substitute for good manufacturing practices, solely to reduce the viable microbial population of a nonsterile product." And this comment on why your bioburden recoveries should be identified (regardless if the testing is within spec): "You should test all appropriate quality attributes including, but not limited to, identity and strength of active ingredients and microbiological quality (total counts and identification of bioburden to detect any objectionable microbes) of each batch." Yes, there were a ton of other significant problems as well as this group being a repeat offender, but these comments reflect on the FDA's requirements for performing IDs on microbial recoveries to find potential objectionable microorganisms. You can see the whole letter at this link: https://lnkd.in/eUUcBjwG #fda #warningletter #objectionableorganism #cGMP #perrittlabs

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"The U.S. House of Representatives on Monday overwhelmingly passed the bipartisan bill, which targets WuXi AppTec, WuXi Biologics and other Chinese biotech companies as potential national security risks." While the bill still needs to go through the Senate, it looks likely that it will get passed. If you are looking to re-evaluate your #API #GMP manufacturing for a US based company with almost 40 years of experience, look no further than Ascensus Specialties. From small molecule synthesis, organometallic compound synthesis, high purity metals for cell culture media, and water soluble phosphines, we have a great reputation in support of pharma and biotech in the US. https://lnkd.in/exkt_w8V

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