Kevin Hrusovsky

PTC was excited to learn, share and collaborate with other leading RNA experts at the recent 7th Annual RNA-Targeted Drug Discovery & Development Summit in Boston, MA, and the RNA at the Bench and Bedside IV conference in La Jolla, CA. Anu Bhattacharyya, Kerstin Effenberger, and Marla Weetall from our research team presented on ways PTC is leveraging its leading splicing platform to identify promising therapies for rare diseases with high unmet need. Learn more about our splicing platform here: https://bit.ly/4fi4w6L

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#BigPharma's growing interest in the #cellandgenetherapy space has the potential to reshape the industry's future. At this year's Cell & Gene #MeetingontheMesa, industry leaders highlighted that large pharma companies' significant resources and expertise are uniquely positioned to address the complex challenges of developing and commercializing these therapies. From high #manufacturing costs to navigating #regulatory hurdles, Big Pharma is playing an essential role in scaling innovations that smaller biotechs often struggle to bring to market. Their partnerships will prove critical to bringing life saving treatments to patients who need them most. As the sector continues to evolve, collaboration between Big Pharma and biotech can help shape the future of cell and gene therapies.

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New Patent Alert! We’re proud to announce that GenNext Technologies has received its fourth and fifth United States Patent! One protects an exciting and proprietary means by which Fox® Protein Footprinting is combined with in-line molecular separation, such as liquid chromatography, to separate and individually label heterogeneous samples, comprising mixed conformers, multi-unit protein complexes, or other diverse protein populations. The other protects the company’s exclusive means and methods by which robust and reproducible in-cell radical protein footprinting studies are performed. These two new patents further establish GenNext as the pre-eminent company in high-resolution protein footprinting, enabling researchers to develop safer, more effective, and more affordable therapeutics. A heartfelt thank you to our dedicated GenNext Team for your relentless effort and innovation in advancing the field of structural biology research! #GenNext #Research #Science #ScientificResearch #Biotherapeutics #ProteinCharacterization #ResearchInnovation #HRPF #ProteinResearch #ScientificInnovation #Teamwork #ProteinStructure #StructuralBiology #Biochemistry #Proteomics #Biotechnology #Antibody #mAb

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"The 12-month results from the PARADIGM Phase IIb study are encouraging, showing slowing disease progression and improved survival outcomes. This new analysis highlights PrimeC's ability to regulate the iron panel in people living with ALS, underscoring the drug's target engagement. These findings strongly support the proceeding to phase 3 testing of PrimeC in ALS." #NeuroSenseTherapeutics #ALSTreatment #ClinicalTrial #BiomarkerData #HealthcareInnovation #ProudMoment NeuroSense Therapeutics https://lnkd.in/dzVT6QWJ

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Good afternoon everyone! Today's blog dives into the power of a new AI-based predictive model (published in June in Nature Methods) that can effectively extract valuable drug discovery insight - but only after being trained on tons of single-cell RNA-seq datasets (more than 50 million data sets in fact). This exciting new predictive model, called as scFoundation, is designed to predict cellular responses to changes, gene interactions, and potential drug targets based on single-cell RNA sequencing data. It also improves the prediction of cancer drug responses and cellular responses to perturbations. In essence, it uses patterns in the data to make predictions about various aspects of cellular behavior. This makes it a valuable tool in fields like precision medicine, targeted cancer therapy, and understanding of complex cellular behaviors. Hope you enjoy! #bioinformatics #scRNA-seq #AI #predictivemodels

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Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP) Announces $2.5 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules   Acurx Pharmaceuticals, Inc. (#NASDAQ: #ACXP), a late-stage #biopharmaceutical company developing a new class of #antibiotics for difficult-to-treat #bacterial #infections, announced that it has entered into a definitive agreement for the purchase and sale of an aggregate of 2,463,058 shares of its common stock at a purchase price of $1.015 per share in a registered direct offering priced at-the-market under Nasdaq rules. In addition, in a concurrent private placement, the Company will issue unregistered warrants to purchase up to 2,463,058 shares of common stock. The warrants will have an exercise price of $0.90 per share, will be exercisable upon issuance and expire five years following the date of issuance. The closing of the offering is expected to occur on or about January 7, 2025, subject to the satisfaction of customary closing conditions.   https://lnkd.in/eAKy4iSM #nasdaq #acxp #antibiotics #acurx

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Using 'representation vectors' instead of prompt engineering to modulate #LLM responses enables tuneable behavior: https://lnkd.in/eUwSUvtg

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Kevin King, BioIVT’s Senior Director of #Donor Center Strategy and Growth will be providing his industry insight and expertise by moderating a roundtable session at the 2nd Annual Donor Selection & Source Summit today entitled “Mitigating Risks in #CellandGeneTherapy: Re-evaluating Donor Selection and Characterizing Cellular Starting Material". The discussion will include topics like: • Establishing industry standard criteria for donor eligibility and implementation of advanced screening technologies. • Characterization of cellular properties and standardized assays to assess donor derived cell quality and function. As experts in providing RUO & GMP cellular starting and ancillary materials for the manufacture of #AdvancedTherapies, our aim is to foster collaboration between stakeholders to share needs as well as provide input on implementing risk mitigation strategies and foresight for sourcing donor-derived cellular starting material. Make sure to connect with Kevin and Lisa Kretzschmar, MBA if you will be at the event. If you are not attending the event, you can contact our experts here: https://hubs.ly/Q02Ywbkk0 Or view a recording of one of our previous expert roundtables here: https://hubs.ly/Q02Yw96w0 And access other insightful reference materials here: https://hubs.ly/Q02Ywf510) #CellTherapy #GeneTherapy #ATMPs

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The #biosecure act is bad legislation and anti-competitive and will force US #biotech companies to abandon strong and honest #CDMO partners without making us more secure. Longstanding, productive drug-discovery collaborations will inevitably be compromised by an ill-conceived initiative — all for short-term political gain. Patients in need of innovative medicines are the ones that will lose out; I urge leaders in the #biotech industry to stand up and be counted in opposition to this proposed legislation.

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🚨 Webinar Announcement: Improving Outcomes and Access to Precision Medicine🚨- https://lnkd.in/eSvkPZPG Despite groundbreaking advances in precision medicine, a critical gap remains: the lack of diverse representation in genomic data, leading to unequal healthcare outcomes—especially for people of color. 🔬 What is the impact? Genomic testing is a cornerstone of modern medicine, yet current databases are largely based on data from individuals of European descent. This leaves underrepresented populations at a disadvantage, with less effective treatments and limited access to life-saving innovations. 💡What you’ll learn: 1️⃣ Understanding the genomic testing gap and the structural barriers in healthcare 2️⃣ How to advance precision medicine for all, ensuring inclusivity 3️⃣ Practical strategies for improving access, including policy changes, community engagement, and innovative healthcare models 📅 Date: Thursday, 21st November, 2024 ⏰ Time: 12:00pm - 1:00pm ET 📍 Location: Zoom (Register Here https://lnkd.in/eSvkPZPG) Let’s work together to create a healthcare system that serves everyone. #PrecisionMedicine #GenomicTesting #HealthEquity

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MassBio’s recently released Vision 2030 plan focuses on strengthening the #LifeScience ecosystem in Massachusetts by creating and retaining jobs, supporting #Biotech startups, and expanding the industry’s capacity for #Biomanufacturing. Download the full report to learn more! https://lnkd.in/eczzKP2i

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Don’t miss the latest episode of The Bio Report podcast, where our CBO Hilary Eaton, PhD joined Daniel Levine for a wide-ranging conversation about the role of #GenerativeAI in drug development, our OpenCRISPR-1 molecule and why we made it open-source. 👉 https://lnkd.in/gkD4iAEt #AI #CRISPR #OpenCRISPR #ProteinDesign #Podcast

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We've been speaking to Genetic Engineering & Biotechnology News about the importance of having a unified multimodal registration system when developing advanced therapeutics. Read the article to learn how a multimodal approach to entity registration can help accelerate the development of multimodal drugs. https://lnkd.in/eZV9HVG4 #ELN #LIMS #multimodal #ADC #CMB #PROTACs

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Jonathan Montagu, CEO of MassBio member HotSpot Therapeutics, Inc., writes on the #LifeSCiVC blog about hopes around an immuno-oncology spring this year, aligning with the emergence of #biotech from its own long winter. Jonathan points out that after the #lifesciences industry dove headfirst into I-O “to address the substantial need that still persists for many cancer patients with a range of tumor types,” much of the second-generation agents fell short. He writes about why he’s optimistic that new approaches have started to turn the tide. What are your thoughts on the I-O space right now? https://lnkd.in/eMh62Z8j #immunooncology #oncology #patientdriven

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Add more quantifiable data revvvvvv your AI engine!!!! Not just binding and sequences… add color to your data to tremendously increase your algo’s prediction power! Only on the #Beacon #optofluidic platform

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Lake N. Paul, PhD. is in San Diego at the Biotechnology Innovation Organization #BIO2024 conference meeting with colleagues & future clients. Connect with him in person to learn about how, as a Collaborative Research Organization, we are best positioned to support your analytical program. #aav #genetherapy #rarediseases #biophysics #STEM #STEMPhilly #biochemistry #celltherapy #bioanalysisllc #PhillyBusiness #phillyscience

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INPUT NEEDED: Help us prioritize topics for future #RegenerativeMedicine standards coordination efforts. Take a short survey on needed #standards. https://bit.ly/35ErlDk #CellTherapy #GeneTherapy #TissueEngineering

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ViroCell Biologics, a leading CDMO specializing in GMP viral vector manufacturing, successfully closed an oversubscribed convertible note offering to support its growth. New investor First Light Asset Management LLC led the funding round, joined by existing investors Sartorius Stedim Biotech S.A. and Dorset Opportunity Fund LP. The proceeds will fuel ViroCell's growth, meeting the rising demand for its viral vector design and manufacturing expertise. The company provides pre-clinical and clinical viral vector services, supporting the development of innovative cell and gene therapies. With regulatory approval from the UK's MHRA, ViroCell's services are in high demand, resulting in a substantial Master Services Agreement with a prominent US cancer center. CEO John W. Hadden II expressed gratitude to investors, especially First Light Asset Management LLC, for their confidence in the company's market position and growth potential. Matt Arens, CEO and Founder of First Light Asset Management LLC, emphasized the team's dedication to supporting ViroCell's ambitious growth journey. This investment will enhance the CDMO's services for cutting-edge CGT research organizations globally. ViroCell Biologics aims to be the preferred partner for entities developing CGT therapies, leveraging its expertise to streamline the clinical development process. Read more: https://lnkd.in/dSkMGaQn Farzin Farzaneh, PhD John W. Hadden II Todd Davis

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This standardization is crucial for the reproducibility of experiments, which is a cornerstone of scientific research. #BDBiosciences #LabAutomation #BDemployee https://bit.ly/3UCMxQL

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