Nicholas Giokas

Exciting times ahead in gene therapy! 🧬✨ Regeneron has just announced a colossal partnership with Mammoth Biosciences, spotlighting a promising future for CRISPR-based in vivo gene editing treatments. This dynamic duo is bringing together Regeneron's state-of-the-art viral vectors and Mammoth's cutting-edge compact nucleases to forge treatments with precision targeting. The deal includes a whopping $100 million upfront with an additional $370 million for milestones, plus royalties. That's a serious investment in next-gen healthcare. Dr. Christos Kyratsous from Regeneron cites years of groundwork laying the foundation for this collaboration, set to align the delivery systems with specific diseases for optimal outcomes. With equity involved and the option for Mammoth to co-fund and profit-share, the deal embodies a true symbiotic spirit. As the second major gene editing stride for Regeneron, after its ongoing work with Intellia Therapeutics, we're witnessing an exhilarating era where the potential for breakthrough therapies is skyrocketing. This is game-changing science with patient lives ready to be profoundly impacted! #GeneTherapy #CRISPR #Innovation #Healthcare

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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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Biopharma investment trends continue to shift, with venture funding jumping to $7.4 billion across 188 deals for the first quarter of the year. This compares to $6.3 billion across 248 deals the previous quarter and demonstrates an increase in deal value despite declining deal count, according to PitchBook. Despite investment trends changing, funding levels quarter-over-quarter have stayed relatively stable over the past two years, defining a new normal for the sector until federal interest rates change, according to PitchBook’s quarterly biopharma report. The cautiously optimistic climate remains focused on advanced clinical assets, with platform bets mostly out of the picture. #biopharmainvestment #biopharmafunding #biotech #biopharma #funding #investment #pitchbook

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Do you know when companion diagnostics (CDx) are needed for your cell and gene therapy programs? Or what kind of testing is required and how to choose the right lab partner for a CDx assay? In our blog, you’ll learn from Labcorp subject matter experts as they answer key questions you may have about developing CDx for your cell and gene therapy programs. Learn more: https://lnkd.in/eiJH-Y7h #CellandGeneTherapy #CompanionDiagnostics

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“Dual-Target” cell therapy appears to shrink brain tumors, Penn Medicine research finds Early trial results from six patients with recurrent glioblastoma may be a promising step towards developing cell therapies for solid tumors PHILADELPHIA— Targeting two brain tumor-associated proteins—rather than one—with CAR T cell therapy shows promise as a strategy for reducing solid tumor growth in patients with recurrent glioblastoma (GBM), an aggressive form of brain cancer, according to early results from the first six patients treated in an ongoing Phase I clinical trial led by researchers from the University of Pennsylvania Perelman School of Medicine and Penn Medicine’s Abramson Cancer Center. The findings, published today in Nature Medicine, suggest that this “dual-target” approach is an encouraging step toward developing effective, long-lasting therapies for solid tumors like GBM. “This is the first time CAR T cell therapy with two targets, rather than just one, has been administered to patients with glioblastoma,” said Stephen Bagley, MD, MSCE, an assistant professor of Hematology-Oncology, and Neurosurgery, and principal investigator in the clinical trial.

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FDA's final LDT rule will be official on Monday. Who were the winners and the losers? That's a good question. It's pretty undeniable that big labs that only run IVDs or have NYS/CLEP approved tests were handed a massive victory. AMCs made out pretty well too (with a few caveats). But thousands of small regional labs and at-home testing companies landed in a giant pile of ish. In this weekend's Premium issue ($5/month) of Omic․ly, I dig into the 528 pages of FDA's final rule on LDTs. Don't worry, I'll wear gloves! But, if industry drama isn't your thing, the free issue includes: 1) No DNA mutations needed. Epigenetic changes can cause cancer. 2) Pathobionts: The opportunists hiding in our microbiomes. 3) Barbara McClintock discovered a little thing called the transposable element in 1950 Supporters ($1/month) and above also get access to the Omic․ly Weekly Reading List! --- Not already a subscriber? Visit my website ⬆️ (link under my name)

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#BiomarkerBrilliance When is the optimal time to introduce clinical biomarkers in drug discovery? Integrating biomarkers during target selection is essential for a robust clinical trial design. Here are some critical questions to ponder: 🔹 Are treated patients responding to the compound? 🔹 Have proliferation or cell death markers been identified, and does morphology change with target modulation? 🔹 What clinical effects are apparent? 🔹 Can we forecast the impact on the target? 🔹 Which biomarker is most prevalent for patient response? Looking to enhance your Biomarker Strategy? Contact me for further details. Contact: laura.allbuary@md.catapult.org.uk @Gayle Marshall #proofofprinciple #proofofconcept #predictivebiomarkers #patientselection #pharmacodynamicmarkers #letussupportyou #clinicalbiomarkers #medicinesdiscoverycatapult #MDC Read more: [Link to article](https://lnkd.in/gzWF4aXg)

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🚀 Immunovant's strategic shift in autoimmune treatment focus! 🚀 Immunovant, a spinout of Roivant Sciences, has announced a pivotal shift in its research priorities. The company will now focus on IMVT-1402, a promising FcRn inhibitor, targeting a range of autoimmune disorders including Graves’ disease. 🧬💡 🔬 Key Highlights: - IMVT-1402: Designated as the lead asset due to positive in-class data. Planned for 4-5 registrational studies in endocrinology, neurology, and more within FY 2025. - Batoclimab: While still important, its prioritization for myasthenia gravis (MG) is being reassessed. Topline results expected within the fiscal year. - Vyvgart: With this shift, argenx’s Vyvgart, FDA-approved for MG, maintains its leading position for now. 🗣️ CEO Pete Salzmann: “We see tremendous opportunity for IMVT-1402, which we believe has a combination of potentially best-in-class features not seen with any other FcRn inhibitor.” 📈 Analyst Insights: William Blair analysts commend the decision, noting the prudence of addressing batoclimab’s LDL lowering concerns and the strategic move to bolster Immunovant’s future in autoimmune treatment. #Biotech #Pharma #AutoimmuneDisease #Research #ClinicalTrials #Innovation #Healthcare #Immunovant #RoivantSciences #FcRnInhibitor #MyastheniaGravis #GravesDisease #MedicalAdvancements #DrugDevelopment

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🚀 Another 2 mL autoinjector to hit the market! LEO Pharma's Adbry® (tralokinumab-ldrm) just received FDA approval for its new 2 mL autoinjector, offering a more convenient option for adult patients with moderate-to-severe atopic dermatitis (AD). This new device halves the number of required injections compared to the pre-filled syringe, making it easier for patients to manage their condition. Adbry, a highly responsive human monoclonal antibody, specifically targets and inhibits the IL-13 cytokine, a primary driver of AD symptoms. With this new delivery method, LEO Pharma continues to innovate and improve treatment options for patients. For more details, check out the full article here: https://lnkd.in/gzj5bm7S

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I've been hearing the reason for the slow uptake of cell and gene therapies isn't insurance coverage. It's finding a location that will administer the therapy. --- When you see #CellAndGeneTherapy (CGT) drugs that cost hundreds of thousands to millions of dollars, you would assume the major barriers to treatment are #HealthInsurance coverage=paying for therapy. However, most insurance covers CGTs. There have still been very few patients receiving these drugs. --- The major barrier (anecdotally) seems to be finding a location that administers CGTs since only a few locations feel comfortable offering these costly CGTs with detailed administration processes. As providers start to notice the gap in this niche, the number of CGT claims is guaranteed to increase. --- Who's ready to start an outpatient #infusion center with me where we only administer a subset of CGTs??

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If your oncology trial needs IBC review, consider this: In a case study of 52 consecutive IBC submissions for NCI-designated Cancer Centers, Advarra completed reviews in an average of 6.5 business days. (Our standard review is 10 days or less.) 

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H1 has a new industry report out on #MSL / #medicalaffairs impacts on #drugadoption and #healthcareoutcomes. "We discovered that HCPs engaged by MSLs were 7.64 times more inclined to treat their patients with a specific medication." Check it out below 👇

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Targeted Radiopharmaceuticals are creating remarkable advancements in the life sciences sector! These targeted therapies represent a paradigm shift in the battle against cancer within the United States, enabling unprecedented levels of personalized care. By zeroing in on cancerous cells and minimizing damage to surrounding healthy tissues, patients are witnessing a new era of medical optimism. The momentum we're seeing is, in part, propelled by recent FDA approvals, which underscore the growing vital role of precision medicine in oncology. As these treatments continue to demonstrate their potential, we're not just looking at breakthroughs, but at what could very well become the cornerstone of future cancer care protocols. It's moments like these that highlight the critical need for relentless innovation in the sector. I hope to see innovative and pioneering companies continuing to push the boundaries of what's possible in the quest to defeat cancer. #Radiopharmaceuticals #PrecisionMedicine #LifeSciences #CancerTreatment #InnovativeTherapies #FDAApprovals #HealthcareInnovation #Oncology #FutureOfMedicine

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Exciting times to keep it going and learn for the IVD, laboratory medicine and genetic testing industries! A cash bid of $239 million has been made for the acquisition of substantially all of Inviae's assets. This move ensures business continuity for customers, partners, and employees. There is market constraints and corrections to happen for sure with future FDA final rule on laboratory developed tests (LDT). #laboratorymedicine #genetictesting #businessacquisition

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FDA approved Yesafili (aflibercept-jbvf) and Opuviz (aflibercept-yszy) as interchangeable biosimilars to Eylea (aflibercept) to treat: o Neovascular (wet) age-related macular degeneration o Macular edema following retinal vein occlusion o Diabetic macular edema o Diabetic retinopathy The risks included as contraindication or under the warnings and precautions include: o Hypersensitivity o Endophthalmitis (inflammation of the vitreous humor and surrounding tissues), retinal detachments, and retinal vasculitis with or without occlusion o Increase in intraocular pressure o Thromboembolic events (blood clots)

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The FDA has announced the release of a draft guidance for industry titled “Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment.” Developed by the Division of Gastroenterology within the Center for Drug Evaluation and Research, this guidance aims to assist sponsors in the clinical development of treatments for pediatric ulcerative colitis and Crohn’s disease. It outlines key considerations for study population, design, efficacy, and safety assessments necessary for developing effective treatments for these conditions in children. This initiative underscores FDA’s commitment to improving therapeutic options and outcomes for pediatric patients with inflammatory bowel disease. Know more - https://lnkd.in/e45GyWEj #Pharmacovigilance #ClinicalResearch #FDA #Pediatrics #InflammatoryBowelDisease #DrugDevelopment

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