Dennis Zaller

🎤 uBriGene Presents Exclusive Sessions at SAPA 2024 @Philly CGT Annual Conference 🎤 🚀 We're thrilled to announce our participation at 2024 @Philly Cell and Gene Therapy Annual Conference with two exclusive sessions aimed at revolutionizing the field: 1️⃣ An Innovative Technology for High Yield and More Stable #CRISPR Guide RNA: Discover how our innovative approach of using in vitro transcription (IVT) in producing guide RNA (#gRNA) that can simplify the process while being more cost-effective and free of organic chemicals. Join us to delve into the future of precision gene editing. 2️⃣ How Could CDMO and Technology Innovations Make CGT Affordable: Dive into the intersection #CDMO of and technological advancements with uBriGene. Learn about our strategies to drive down costs without compromising quality or efficacy. Let's explore how cutting-edge technology can make #CellandGeneTherapy more accessible to all. 2024 @Philly Cell and Gene Therapy Annual Conference brings together the innovators and business leaders to foster collaborations and advance cell and gene therapy in the #GreaterPhiladelphia area. 💡 Join #uBriGene to be at the forefront of innovation and collaboration in the dynamic world of cell and gene therapy and let's make cutting-edge cell and gene technology more accessible!

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Erich Bozenhardt, PE, Associate Director of Process Engineering and Therapeutics for United Therapeutics, an active ISPE member, and close collaborator on the ISPE Guide: Advanced Therapy Medicinal Products – Allogeneic Cell Therapy, has participated in an interview with Cell & Gene in which he shares insights on the new ISPE Guide. In the interview, Bozenhardt touches on the current state of manufacturing standards for allogeneic cell therapies, key differences between allogeneic advanced therapy medicinal products (ATMPs) and a typical aseptically manufactured biologic, and how the ISPE Guide approaches de-risking for more risk-heavy steps such as downstream during fill/finish. Read more: https://hubs.la/Q02CjS-80 #ISPE #ISPEguides #atmp #allogenic

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To further understand the genetic causes of certain types of lung cancer in families, GO2 for Lung Cancer is collaborating with the Dana-Farber Cancer Institute, and the Addario Lung Cancer Medical Institute (ALCMI), GO2's medical consortium, on INHERIT. INHERIT builds on a previous study that identified an inherited gene mutation for lung cancer. It will look more deeply into families' genetic makeup to identify other mutations that increase the risk for lung cancer to inform prevention and treatment. Learn more about the INHERIT trial and how you can participate at inheritstudy.org.

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16July: How does Orum Therapeutics plan to replace chemo with the targeted conditioning agents for gene editing therapies that it will work to develop with Vertex Pharmaceuticals? Read about the collaboration in my latest for Genetic Engineering & Biotechnology News: https://lnkd.in/epKWDUbZ

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I am looking forward to speaking tomorrow in Boston, MA at the Arena CLINICAL TRIALS IN ONCOLOGY EAST COAST. Niki Khoshkbary Affinity Patient Advocacy Christina DiArcangelo My topic is entitled “Strengthening clinical trial diversity to reduce inequities in cancer trials and delving into the humanitarian aspects of patient care”. The session outline is as follows: • Working with unrepresented communities to help patients globally and sharing best practice how to recruit and integrate these populations into oncology trials • What is your humanitarian responsibility as it relates to clinical trial recruitment? • Meeting FDA expectations: Guidance on the DEPICT Act compliance and how to write, implement and report on Diversity Plans • Working effectively with sites: What strategies CROs, patient advocacy firms and pharmaceutical companies can design and support to encourage sites to implement these for a diverse patient population • Educating patients to reduce fear and knowing what to ask for from your doctor regarding off label usage and black box warnings from the patient advocacy perspective https://lnkd.in/eTfB_Mp4

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Phase IIb data for Auxora in an acute pancreatitis subpopulation is expected to set up the CRAC channel inhibitor for Phase III in that indication while suggesting positive read-through for AKI. https://lnkd.in/eDzU4m8e?

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New trends in M&A deal breakdown, IO in this case... ... May shed lights on domestic and international BD licensing deal negotiation in 2024 too. Here is my scientific breakdown of the same deal 2 days ago...https://lnkd.in/exG464j8 Good to learn the business side of the deal. Michael Mischke-Reeds

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RNAi-based drug design: considerations and future directions More than 25 years after its discovery, the post-transcriptional gene regulation mechanism termed RNAi is now transforming pharmaceutical development, proved by the recent FDA approval of multiple small interfering RNA (siRNA) drugs that target the liver. Synthetic siRNAs that trigger RNAi have the potential to specifically silence virtually any therapeutic target with unprecedented potency and durability. Bringing this innovative class of medicines to patients, however, has been riddled with substantial challenges, with delivery issues at the forefront. Several classes of siRNA drug are under clinical evaluation, but their utility in treating extrahepatic diseases remains limited, demanding continued innovation. In this Review, we discuss principal considerations and future directions in the design of therapeutic siRNAs, with a particular emphasis on chemistry, the application of informatics, delivery strategies and the importance of careful target selection, which together influence therapeutic success. https://lnkd.in/ecB46Cfr

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IVIEW Therapeutics Inc. Announces Dosing of First Patient in Phase 1/2 Clinical Trial of IVW-1001 for Dry Eye Disease [Cranbury, New Jersey, June 25, 2024] – IVIEW Therapeutics Inc., a clinical stage biotechnology company advancing innovative treatments for ocular diseases, proudly announces the dosing of the first patient in the Phase 1/2 clinical trial of IVW-1001 Ophthalmic Eyelid Wipe. This milestone marks the initiation of the clinical evaluation of IVW-1001, a novel TRPM8 agonist designed to treat signs and symptoms of dry eye disease. The commencement of patient dosing in the Phase 1/2 trial represents a significant advancement in IVIEW Therapeutics' mission to develop innovative solutions for individuals suffering from dry eye disease, particularly those inadequately managed by artificial tears. This first-in-human (FIH) trial aims to rigorously assess the safety, tolerability, and preliminary efficacy of IVW-1001. Dry eye disease is a prevalent and burdensome condition affecting millions worldwide. IVW-1001, developed by IVIEW Therapeutics, offers promising potential as a therapeutic intervention through its TRPM8 agonist mechanism, targeting the underlying causes of dry eye. The innovative delivery route via an ophthalmic eyelid wipe further distinguishes IVW-1001 as a potentially groundbreaking treatment. Houman Hemmati, MD, PhD, Chief Medical Advisor, leading the clinical development of the IVW-1001 program, stated, "Dosing the first patient in our Phase 1/2 clinical trial is an exciting and critical step forward. Our focus remains on thoroughly evaluating the safety and potential efficacy of IVW-1001. This investigational TRPM8 agonist offers an innovative approach to treating dry eye diseases, and we are committed to advancing this program to address the unmet needs of patients worldwide." Bo Liang, PhD, MBA, Co-founder, Chairman, and CEO of IVIEW Therapeutics Inc., added, "We are thrilled to initiate the clinical development of IVW-1001 with the dosing of our first patient. This milestone underscores our commitment to pioneering treatments for dry eye diseases and highlights the potential of our novel TRPM8 activation mechanism. We look forward to the progress of this trial and the insights it will provide."

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Happy share the most recent #Alentis article on #CLDN1 as target in #oncology

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While #ASCO24 is at an end, it was great following all of it on social media. If you were at ASCO and interested in potential collaborations with the journal Technology in Cancer Research & Treatment, please feel free to reach out. Thanks for sharing OncoDaily! #cancer #oncology #cancerresearch #oncodaily

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Evaluating Secondary Primary Malignancy Risk After CAR T-cell Therapy The FDA Adverse Event Reporting System (FEARS) reported secondary primary malignancies in 4.3% of patients who received CAR T-cell therapies, according to a recently published study in Blood. The study authors analyzed 12,394 unique CAR T adverse effect (AE) reports, and after exclusion criteria were applied, 536 (4.3%) identified secondary primary malignancies. The most common treatments that resulted in AEs included axicabtagene ciloleucel (axi-cel; Yescarta) in 51.7% of patients and tisagenlecleucel (tisa-cel; Kymriah) in 33.0%. The report also highlighted a number of trials conducted using CAR T-cell therapies, with 6 of 8 trials reporting secondary primary malignancies. Tisa-cel Phase 2 ELIANA NCT02435849) and phase 2 ENSIGN (NCT02228096): 2.2% for those with acute lymphoblastic leukemia Phase 2 JULIET (NCT02445248): no secondary primary malignancies Axi-cel Phase 1/2 ZUMA-1 (NCT02348216) and phase 3 ZUMA-7 (NCT03391466): secondary primary malignancies were reported in less than 1% and 4.7%, respectively Brexucabtagene autoleucel (Brexu-cel; Tecartus) Phase 1/2 ZUMA-3 (NCT02614066): no secondary primary malignancies Liso-cel Phase 1 TRANSCEND NHL-001 (NCT02631044) and phase 3 TRANSFORM (NCT03575351): secondary primary malignancy rates of 8.1% and 3.3% Cilta-cel Phase 1/2 CARTITUDE-1 (NCT03548207): secondary primary malignancies in 25.8% Ide-cel Phase 2 KarMMa-1 (NCT03361748): no secondary primary malignancies

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Take a sneak peek inside our recent white paper on considerations for commercialization of cell and gene therapies outside of non-orphan indications: https://lnkd.in/es86YMsS #CGTs #advancedtherapies #drugdevelopment

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New podcast episode 👇 Get your headphones ready for another great biotech conversation!

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"GRAIL's Galleri™ test is the only commercially available multi-cancer early detection test on the market. (WRONG). It has enjoyed the fastest first-year revenue ramp in cancer screening history and has reached over 85,000 patients. (AND THE FASTEST LOSS ACCELERATION IN CANCER SCREENING HISTORY). The clinically validated technology (REALLY?!) unlocks an underpenetrated multi-cancer screening market worth more than $44 billion. (SAYS WHO? NOT CMS....) GRAIL already has strong market traction, with $90 million to $110 million of expected annual 2023 revenue (AND $700mm LOSSES) and 60-90% expected compounded annual revenue (AND LOSS) growth over the next five years. Importantly, GRAIL will save lives (WILL IT?) , and Illumina will accelerate that mission: Galleri™ is predicted (...NOT BY ME OR ANY PHYSICIAN OR SCREENING EXPERT I KNOW) to avert one in three cancer deaths within a five-year timeframe." I am not sure this statement is covered by D&O insurance 😥 ...

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This is our second FDA IND clearance for the use of SNK01 in neurodegenerative disease. Given the ability to address amyloid, tau, and alpha-synuclein proteins as well as reduce neuroinflammation, we believe its potential utility extends to any and all neurodegenerative diseases associated with any of these proteins. #nkcells #neuroinflammation #neurodegeneration #tau #amyloid #alphasynuclein #neuroimmunology

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I read with great interest the characterization of Novartis's pan YAP/TAZ/TEAD inhibitor, IAG933. I am particularly intrigued by Figure 8b - a profile of in vivo anti-neoplastic activity in a series of PDX. https://lnkd.in/gSNuZUcN It would appear that, YTP-75, a IAG933 analog, stimulates tumor growth (red vs black bars) as a monotherapy in select PDX.

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Are you working on the next generation of kinase inhibitors? Join experts and peers at the 15th Annual Strategies for Targeting Kinases symposium on September 30th in Boston to explore novel chemistries, techniques, and emerging drug modalities for targeting kinases in various diseases. Featured Topics: ▪ Targeting diverse kinase pathways for cancer, inflammation, and more. ▪ Covalent and non-covalent strategies for kinase inhibition. ▪ Utilizing machine learning for kinase inhibitor discovery. ▪ Exploring the "dark kinase" with novel approaches. View the preliminary agenda and register by June 7 for Early Savings! https://lnkd.in/epqYsiQm #kinase #drugdiscovery #targetedtherapy #neurodegeneration #BostonDOT24

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