Tracy Nicholson

Tomorrow at MassBio's Align Summit, I'll be representing CytoReason on a panel with distinguished representatives from Genentech, BigHat Biosciences, BioNTech SE and Locust Walk. In the session "AI Partnerships: Integrating for Strategic Value" we'll bring up best practices for integrating AI/ML into pharma and biotech companies. We'll also discuss the the competitive advantages that these capabilities can unlock, and when to build, buy, or rent these capabilities. Thanks MassBio for organizing this important event, in partnership with Locust Walk and McKinsey & Company. For more info, see the link in comment below.

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When investors think your gene therapy isn't good enough... That's what Lexeo is feeling right now on LX2006 for cardiomyopathy associated with Friedreich's Ataxia. Their Ph1/2 trial saw 50% of patients (8 total in the trial) experience a reduction of 10% in left ventricular mass index. There are only a handful of companies developing therapies for Friedreich's Ataxia, let alone the heart-related components of this rare disease. While Lexeo was optimistic about the data and taking it to discuss a future pivotal study with regulators, the market was not, knocking the stock down at present ~25%. The public markets are brutal with earlier stage companies. Depending on capital needs, it can be a bit easier to not be public when you're early in development. Regardless, another piece of positive news for AAV as it slowly comes out of its winter. Lines up with our beginning of the year predictions below! https://lnkd.in/drRreSCd

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Appreciate John Glasspool’s op/ed in the Boston Business Journal. He brings the perspective of a CEO of a Cambridge-based #biotech, MassBio member Anthos Therapeutics, to the debate over the White House’s plans to use Bayh-Dole as a blunt instrument to force price controls. The reality is that any effort to use “march in” rights like this would hurt innovation, chill the collaborative relationship between research institutes and small biotechs (which are the lion’s share of Massachusetts’ industry) when it comes to advancing early science, and do nothing to address the price of drugs. You can’t argue with Bayh-Dole’s success: 6 million jobs, $2 trillion in economic output, thousands of startups, and 200+ drugs and vaccines. As John points out, we can’t go back to a time when science sat on shelves instead of getting into medicine cabinets. https://lnkd.in/ezkNndKA cc: The Bayh-Dole Coalition

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For investors interested in our Q2 roadshow covering New York, Boston, Princeton, Chicago, San Diego, and San Francisco, BIORCHESTRA Co., Ltd.'s #RNAi update covers: ✨ In-vivo progress on our neurodegenerative diseases franchise and proprietary IV-formulated RNAi research in #Alzheimer's and #Parkinson's ✨ Status of our #ALS program FDA IND pathway  ✨ Licensing negotiations in #CNS, #INHBE/ #MAFLD, and #Glaucoma ✨ Our bridge round syndicate and NYSE / NASDAQ pathway timing To those requesting one-on-ones through LinkedIn, email davidoxley@biorchestra.com for slots. Bramalea Partners, PJT Partners, Perella Weinberg, William Blair Investment Banking, Jefferies, Leerink Partners, Nomura, Cain Brothers, CITIC Securities Company Limited, CITIC CLSA Forums, UBS, Oppenheimer & Co. Inc., Wells Fargo Corporate & Investment Banking, Mikael Dolsten Guggenheim Partners, Outcome Capital, Brian Doyle, Michael Bardi, Michael Zhang, Veet Misra, Cantor Fitzgerald, Noël Brown, Samuel Siu, CPA, RBC Capital Markets, John C. Reed, Novo Holdings, Lilly Asia Ventures 礼来亚洲基金, Richard A. Gonzalez, Sumitomo Mitsui Banking Corporation – SMBC Group, #pfizercolleague, Centerview Partners, Morgan Stanley USA, Citi, Bank of America Business, Greenhill & Co., Evercore, Rothschild & Co., Moelis & Company, New York Stock Exchange, Nasdaq Exchange

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🌡️ It's July and it's hot. And so is biotech: 💰 Two Massachusetts companies - #MassBioMember Scorpion Therapeutics and Cardurion Pharmaceuticals - just raised $410M in #oncology and #cardiovasculardisease respectively (Boston Business Journal: https://lnkd.in/eViMeruS). 📈 The $XBI is flirting on either side of $100 this week for the first time since March (Endpoints News: https://lnkd.in/ea4RXmy9). 💊 The drugs in these clinical trials (BioPharma Dive: https://lnkd.in/e_UpE-VM) and others could change lives. 📍And Worcester continues to make its strong case to be a #biomanufacturing hub (BBJ: https://lnkd.in/e-Z3hB6k). What's hot in your world?

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New blog alert 🚨 (summary of ASGCT) With a week to digest the annual meeting of the American Society of Gene & Cell Therapy, it's time for a recap! 💡 Exciting research developments The conference featured numerous posters that outlined groundbreaking research using iPSC technology. These studies demonstrated significant advancements in the application of iPSCs for the discovery of new gene and cell therapies.  Here are some of our favorite posters: 616: Phenotypic Rescue of STXBP1 In Vitro and In Vivo Models Using AAV-STXBP1 Gene Replacement Approach 1598: Disruption of Aberrant Splicing of STMN2 by Gene Editing with a Type V CRISPR-Cas Enzyme as a Potential Treatment for ALS 1801: iPSC-Derived Macrophages Engineered to Express IL-12 Enhance Cancer Cell Killing of a Multi-Cell Type Therapeutic Platform 📈 Trends in cell therapy One notable trend observed at the conference was the balanced focus on autologous and allogeneic cell therapies. Both approaches are being actively explored and developed, with each offering unique advantages. Autologous therapies, derived from a patient’s own cells, minimize the risk of immune rejection. Allogeneic therapies, which use donor cells, offer the potential for mass production and off-the-shelf availability. With the allogeneic approach, iPSCs are particularly popular as a starting material, thanks to their versatility and potential for differentiation into various cell types. 🚀 Growing industry focus The industry’s focus on cell and gene therapies is expanding rapidly. This trend is reflected in the increasing attendance at the ASGCT conference, which has nearly doubled over the past five years. This year, the conference attracted 8,000 attendees, highlighting the growing interest and investment in this sector.  🧡 Ncardia's distinctive presence Ncardia also had something that drew the attention of other attendees: Our striking orange outfits. Worn in honor of the Netherlands, our home country, they made us easy to find on the conference floor, and became a big hit with everyone passing by. This unique touch not only showcased our national pride but also facilitated numerous connections and conversations, enhancing our overall conference experience. Curious to check out the rest of the blog? Additional posters and future advancements in the full article! Link in the comments below ⬇ #genetherapy #celltherapy #conference Hi, I'm Jeff Bissen (pronounced BEE-sen) 👋 In my current role as Senior Director of Business Development at Ncardia, I help with applications of iPSCs in drug discovery - and also in cell therapy (see the sister company Cellistic). 🧬 Send a message to discuss your project! I also post almost every weekday about the science and business of biotech. 🔔 Follow my profile, and subscribe to my weekly newsletter: https://lnkd.in/e8r_WBP8 *** Views expressed on LinkedIn are entirely my own and not necessarily held by any past, present, or future employer***

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I am very excited to finally let the cat officially out of the bag! We are joining the growing field of adoptive cell therapies for the treatment of autoimmune disease. Our g-NK cell therapy is uniquely positioned to be effective through additional mechanisms than deep depletion of B cells, as g-NK cells are naturally controlling the development and progression in several autoimmune diseases via their innate capability to kill HLA-E expressing autoreactive B and T cells. This is best documented in the literature for Multiple Sclerosis and provides a strong scientific rationale for the treatment of patients with MS with Indapta's IPD-023 in combination with B cell targeting mAbs. https://lnkd.in/gjAGdCgF #https://indapta.com/

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𝐀𝐭𝐭𝐞𝐧𝐭𝐢𝐨𝐧 𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐂𝐄𝐎𝐬 𝐚𝐧𝐝 𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 𝐋𝐞𝐚𝐝𝐞𝐫𝐬: Check Out This Transformational VP of R&D Candidate (𝘗𝘳𝘦𝘴𝘦𝘯𝘵𝘭𝘺 𝘌𝘮𝘱𝘭𝘰𝘺𝘦𝘥 - 𝘕𝘰𝘵 𝘈𝘷𝘢𝘪𝘭𝘢𝘣𝘭𝘦 𝘖𝘯 𝘛𝘩𝘦 𝘖𝘱𝘦𝘯 𝘔𝘢𝘳𝘬𝘦𝘵)! Our executive search firm is 𝐞𝐱𝐜𝐥𝐮𝐬𝐢𝐯𝐞𝐥𝐲 representing a highly accomplished and mission-driven VP of Research & Development who has a stellar track record in leading R&D teams to develop FDA-approved products and drive market success. 𝐖𝐡𝐚𝐭 𝐓𝐡𝐢𝐬 𝐂𝐚𝐧𝐝𝐢𝐝𝐚𝐭𝐞 𝐁𝐫𝐢𝐧𝐠𝐬: - Proven Innovator: Spearheaded multi-disciplinary R&D teams to secure FDA approvals for groundbreaking therapies. - Patent Powerhouse: Holder of 20+ US patents and co-author of 20+ published papers. - Startup to Success: Successfully scaled both national and international R&D teams, transforming concepts into commercially viable products and expanding global market presence. 𝐈𝐝𝐞𝐚𝐥 𝐑𝐨𝐥𝐞𝐬: - Chief Scientific Officer (CSO) - Vice President of Research & Development (VP R&D) - Head of Regulatory Affairs - Director of Product Development 𝐖𝐡𝐲 𝐓𝐡𝐢𝐬 𝐂𝐚𝐧𝐝𝐢𝐝𝐚𝐭𝐞 𝐂𝐨𝐮𝐥𝐝 𝐃𝐫𝐢𝐯𝐞 𝐈𝐦𝐦𝐞𝐧𝐬𝐞 𝐈𝐦𝐩𝐚𝐜𝐭 𝐘𝐨𝐮𝐫 𝐎𝐫𝐠𝐚𝐧𝐢𝐳𝐚𝐭𝐢𝐨𝐧: - Significant Biotech Expertise: Mastery across chemistry, biophysics, pharmacology, materials science, and various engineering disciplines, ensuring a comprehensive approach to drug development. - Regulatory Savvy: Extensive experience with FDA interactions, having co-authored multiple IND filings and secured NDA approvals, ensuring regulatory compliance and streamlined approval processes. - Leadership Excellence: Demonstrated ability to build and mentor diverse, high-performing R&D teams, driving innovation and achieving strategic objectives in fast-paced environments. This candidate has shown significant employment tenure historically throughout their career and has impactful executives willing to speak on their behalf as professional references. They want to spend the last decade of their career contributing to one more company's mission that will positively impact people's lives. Due to life circumstances, this candidate is willing to relocate to many areas across the US if they find that ideal opportunity outside of California. Biotech Leaders - Your Next Innovator Awaits! If you’re ready to harness top-tier talent to drive your company’s growth and innovation, let’s connect! This innovative leader is ready to propel your biotech firm to new heights. 📩 Contact Me Today to learn more about how this VP of R&D can be the catalyst for your next breakthrough. #Biotech #Innovation #Leadership #RegulatoryAffairs #R&D #ExecutiveTalent #CareerOpportunities

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Mustang Bio Bio Announces Favorable Efficacy and Safety Data from Complete Waldenstrom Macroglobulinemia Cohort of Phase 1/2 Clinical Trial of MB-106, CD20-Targeted Autologous CAR-T Therapy WORCESTER, Mass., June 17, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell therapies into potential cures for difficult-to-treat cancers, today announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy, show a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia (“WM”), a rare form of blood cancer. MB-106 is being developed in a collaboration between Mustang and Fred Hutch Cancer Center (“Fred Hutch”) to treat patients with relapsed or refractory B-cell non-Hodgkin lymphomas (“B-NHLs”) and chronic lymphocytic leukemia (“CLL”). The updated results from the single-institution Phase 1/2 clinical trial were presented during a poster session at the European Hematology Association 2024 Hybrid Congress (“EHA2024”) by Brian Till, M.D., Associate Professor and physician at Fred Hutch and University of Washington. All ten patients in the study were previously treated with Bruton's tyrosine kinase inhibitors (“BTKi”), and their disease continued to progress while on BTKi. Overall, 90% (9/10) of the patients treated with MB-106 responded to treatment, including 3 complete responses, 2 very good partial responses and 4 partial responses. In addition, 1 patient experienced stable disease. One of the patients who achieved a complete response has remained in remission for 31 months, with an immunoglobulin M (IgM) level that decreased rapidly to the normal range after treatment with MB-106 and has remained normal since. Patients had a median of nine prior lines of therapy and only one patient has started additional anti-WM treatment after being treated with MB-106. https://lnkd.in/eRSu7Msk

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Time is money in drug discovery. So key question is: “How long until my iPSC’s are assay ready and showing a functional response/signal ?” See answers below!! #cellculture #microfluidics #organoids #organonchip #drugdiscovery #drugdevelopment #biotech #pharma #neuroscience #PNS #mea

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🎉 The BioHub Episode with Rob Etherington, CEO of Clene Nanomedicine, Inc. is Live! 🎉 Rob dives into Clene's groundbreaking work against neurodegenerative diseases, predicted by the WHO to be the second leading cause of death in the coming decades. In this episode, you'll hear about: 🧬 Clene’s groundbreaking nanotechnology 🌍 The critical importance of their mission 🧠 Why understanding the brain remains one of the biggest challenges in medicine Don’t miss out on this enlightening episode! Click the link in the comments to watch the full episode. Remember to like, share, and subscribe to The BioHub Podcast! 🚀 Let us know your thoughts in the comments below. #TheBioHub #Podcast #Biotech #Innovation #NeurodegenerativeDiseases #Nanomedicine #CleneNanomedicine #RobEtherington #Healthcare #FutureOfMedicine

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🚀 Big News from Radar Therapeutics! 🚀 Radar Therapeutics has successfully raised $13.4 million in seed financing to advance its mission of developing smart programmable medicines using molecular RNA sensors! 🎉💉 The funding round was led by NfX Bio with participation from major investors including Eli Lilly and Company, Biovision Ventures, and KdT Ventures. Additional support came from PearVC, BEVC, and other esteemed investors. 🙌💰 What They're Working On: 🔬 Programmable Genetic and mRNA-Based Therapeutics: Radar Therapeutics is pioneering the use of RNA sensors—mRNAs that control gene expression based on cell-specific RNAs—to enable precise, targeted, and timed drug delivery. This approach aims to minimize systemic side-effects and enhance therapeutic efficacy. Key Highlights: 🌟 Innovative Technology: The RADAR platform allows for the rational design of precision therapeutics, capable of altering cell biology, deleting harmful cells, or reprogramming cells for autoimmune diseases. 🌟 Expert Leadership: Co-founded by synthetic biology pioneer Jim Collins, Ph.D., and Xiaojing Gao, Ph.D., with industry-leading insights from CEO Sophia Lugo and CSO Eerik Kaseniit, Ph.D.. 🌟 Impactful Publications: Featured in Nature Biotechnology, showcasing highly specific, compact sensor sequences for targeted gene expression. 🌟 Industry Recognition: Honored with the AbbVie Golden Ticket, Johnson & Johnson West Coast Cell and Gene Therapy Symposium "Judge's Choice" award, and the Amgen Diversity, Inclusion, and Belonging Award. “We’re leveraging the explosion in single-cell transcriptomic data and advances in RNA-editing enzymes to create smart mRNA therapies,” said Eerik Kaseniit, Ph.D., Chief Scientific Officer & Co-Founder. “By leveraging a broad dataset offered by single-cell transcriptomics, Radar can precisely identify cellular signatures and engineer programmable therapies accordingly, offering unparalleled specificity to avoid off-target effects,” said Omri Amirav-Drory, PhD, Partner, NfX Ventures. With the support of these top-tier investors, Radar Therapeutics is set to advance its preclinical programs and continue expanding its team. 🚀 #Biotech #mRNA #RNAEditing #PrecisionMedicine #GeneticTherapeutics #HealthcareInnovation #Investment #SeedFunding #SmartMedicine #BiotechInnovation #SingleCellTranscriptomics #DrugDevelopment #RadarTherapeutics

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🔍 Join our facilitator Alexander Ivliev at his Roundtable Discussion at the AI in Drug Discovery Xchange 2024, Boston! 🧪 📚 Dive deep into the realm of target discovery with our esteemed facilitator, Alexander Ivliev, Director of Computational Biology at Ikena Oncology. In this interactive roundtable discussion, we will brainstorm the vital components essential for a robust target discovery engine and unravel the mystery of how to integrate them effectively. ⌚ Time: 15:40 – 16:40 📅 Date: 20th June 2024 📍 Location: Woburn Hilton Hotel, 2 Forbes Road, Woburn, US 🪧 Join us today & claim your COMPLIMENTARY pass today! 🪧 https://bit.ly/43qUJXC ✨ Session Highlights ✨ 🔵 The balance between in-house experimentation and leveraging public resources in target identification. 🔵 Exploring the most informative experimental approaches and data types. 🔵 Navigating approaches tailored for efficacy versus safety. 🔵 Strategies for managing the trade-off between target novelty and confidence level. 🔵 Piecing together the components into a cohesive framework. 🗣️ Alexander brings a wealth of expertise to the table, with a master’s and PhD in Bioinformatics and over a decade of experience in the field. Having spearheaded bioinformatics initiatives at Clarivate Analytics, supporting diverse clients in pharmaceutical and biotech sectors, Alexander now leads the computational biology domain at Ikena Oncology. His role is pivotal in accelerating the development of small-molecule inhibitors for cancer treatment. Only available for Senior Scientists and above, from Bio and Pharma companies with a drug pipeline. 💊 * Agenda subject to change #TargetDiscovery #DrugDiscovery #Bioinformatics #AIinHealthcare #AIinDrugDiscovery #InnovationInMedicine #RoundtableDiscussion #Biotech #Pharmaceuticals #DataDrivenDiscovery #ExperimentalApproaches #EfficacyVsSafety #NovelTargets #ConfidenceInDiscovery #InHouseExperimentation #PublicResources #CollaborativeScience #ScientificAdvancements #BiomedicalResearch #PrecisionMedicine #FutureOfMedicine #StrategicThinking #BioinformaticsInsights #hubXchange #Roundtable #FreePass #RegisterNow #ComplimentaryPass

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💥 The BioHub is back with another episode! 💥 Tomorrow, we're dropping a fascinating episode featuring Rob Etherington, CEO of Clene Nanomedicine, Inc.! 🌟 Rob dives into the critical work Clene is doing to combat neurodegenerative diseases, which the World Health Organization predicts will be the second most prevalent cause of death in the coming decades. 🧠 The brain remains a mystery in many ways, and Clene's revolutionary nanotechnology is truly groundbreaking. 🧬 Don't miss out on tomorrow's release! Like, share, and subscribe to The BioHub Podcast! 🚀 #TheBioHub #Podcast #Biotech #Innovation #NeurodegenerativeDiseases #Nanomedicine #CleneNanomedicine #RobEtherington #Healthcare #FutureOfMedicine

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The Innovation Imperative: Pioneering New Modalities for Therapeutic Leadership -- great report from Revvity Signals, creators of the ChemDraw software I used to operate for many years during my research career. Grateful for featuring BioPharmaTrend.com's article "The Explosion of Therapeutic Modalities: Small Molecules, Biologics, and Everything in Between!" "New drug modalities encompass a broad spectrum of therapeutic strategies, each distinct in its action mechanism and target specificity. These span from monoclonal antibodies (mAbs) and RNA-based therapies to advanced gene editing techniques and cell therapies. The classification of these modalities is multifaceted, considering both their therapeutic mechanisms and stages of development. Established methods like mAbs coexist with emerging frontiers like CRISPR and CAR-T cell therapies, illustrating the breadth and depth of this field." A noteworthy trend within the small-moleculemodality is the reemergence of PROteolysis TArgeting Chimeras (PROTAC) although they were first described in 2001. This class within small molecule therapeutics Targeted Protein Degraders (TPDs), along with covalent inhibitors, and macrocycles represent an exciting frontier in small molecule therapy. TPDs, for instance, are designed to harness the body’s own protein disposal systems to eliminate disease-causing proteins, a strategy that could offer a new therapeutic angle for challenging targets. Covalent inhibitors, with their ability to form permanent bonds with target proteins, offer enhanced specificity and potency. Macrocycles, characterized by their large, ringshaped structures, present a unique balance of specificity and stability, enabling them to interact with complex protein surfaces that were previously inaccessible." Links to report and article in the comments 👇 #medicinalchemistry #drugdiscovery #biotech #biologics #biopharmatrend Image credit: Revvity Signals

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Exciting news to share today

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Whether you recover from #bio2024, or held down the fort in the #lab: If you are working in small molecule drug discovery, the #SanDiego event reemphasized the importance of #AI for your field. If you are planning to engage with an external partner for your drug design and chemistry, consider how the partner or CRO is integrating AI into their #DMTA cycles. Some of these players drive their AI integration via acquisitions or partnering with a third party AI outfit, often resulting in a patchworked landscape, which compromises the sought-after efficiency gains. The most efficient way to leverage this groundbreaking technology - a great overview is provided in the recent Chemical Engineering News article below - is by have natively grown AI/ML expertise work hand-in-hand with your synthetic and medicinal chemists. Something Evotec's Molecular Architects, headed by Christophe Boldron, embody in many of the integrated drug discovery programs we design and execute for our partners and clients. #bayarea #chemistry #researchneverstops #ML #medchem #drugdesign #drugdiscovery #idd

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Thank you to Steve Lohr and The New York Times for featuring Terray Therapeutics. We’re proud to be changing the way computation and experimentation work together to generate the kind of data needed to deliver on the promise of A.I. in drug discovery to solve the toughest therapeutic challenges. As our CEO, Jacob Berlin, PhD said: “Once you have the right kind of data, the A.I. can work and get really, really good.” That is our data, and it's all pointed at a “once-in-a-century opportunity" for the pharmaceutical industry to deliver for patients in need. Thank you to our extraordinary team, our partners at Bristol Myers Squibb and Calico Life Sciences, our collaborators at NVIDIA, and all of our amazing investors including Madrona, Digitalis Ventures, Two Sigma Ventures, and KdT Ventures.   The best is ahead. Read more: https://lnkd.in/eMtyFdNQ   #generativeAI #biotech #ML #drugdiscovery

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