Alex Sim

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

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A novel case of BiTE (EGFRxCD28) plus anti-PD-1: Targeting the 'right' patients counts! Colon cancer with microsatellite stability (MSS), 85% of patients¹, is non-responsive to anti-PD-1 therapy²⁻³. Recently, REGN7075, a EGFRxCD28 BiTE, plus anti-PD-1 showed encouraging efficacy, 6% (3/51) objective response rate (ORR); moreover, all responders were non-metastatic patients. Among the subset of non-metastatic patients (15), ORR became 20% (3/15)³. This validated our proposal that BiTE or anti-PD-1 or duo is more applicable to early-stage non-metastatic, instead of late-stage metastatic, solid-tumour cancer patients (see recent POSTs). The wild-type (WT) EGFRs are expressed on normal and cancer cells⁴, and colon cancer expresses WT EGFR⁵. A novel EGFR mAB targeted the epitope in Domain II (CR1) of extracellular domains⁵. The Domain II is masked in quiescent EGFRs of normal tissues, yet is exposed in EGFRs of cancer cells⁵. We deduced that REGN7075 also targeted the epitopes in Domain II, resulting in minimal on-target off-tumour toxicity of normal cells. TGN1412, a CD28 mAB, induced severe cytokine release syndrome⁶ (CRS); however, a novel CD28 mAB had shown lower CD28 affinity by targeting the epitope near the apex, instead of TGN1412's targeting the lateral epitope near cell membrane⁷. We deduced that REGN7075 also targeted the epitope near the apex of CD28, resulting in low CRS rate. By targeting novel epitopes of EGFR & CD28 presumably, REGN7075 incurred 35% of patients with grade 3/4 adverse events³ vs typical BiTE's 62-87%⁸⁻⁹. The CD3-based BiTE mimics signal 1 for T-cell activation by binding CD3. The CD28-based BiTE mimics signal 2 by binding the costimulatory CD28 receptor (ligands: CD80/86). The 'functional' DCs have become rare in tumour milieu and tumour itself does NOT emit danger signals to activate these rare DCs for upregulating CD80/86. Thus, there is the scarcity of CD80/86 (signal 2) in tumour milieu for properly 'reactivating' subprimed PD-1⁺ tumour-infiltrating T cells, which were initially 'subprimed' in lymph nodes by only signal 1 (see recent POSTs). By using anti-PD-1 (low dose) for T-cell 'reactivation'¹⁰ as signal 1 and CD28-based BiTE for direct CD28 binding as signal 2, the sub-primed PD-1ˡᵒʷ⁻ᶦⁿᵗ CD8⁺ T cells can resume cytotoxic effector function (granzymes and perforin) to kill tumours. Thus, Regeneron's novel duo of CD28-based BiTE plus anti-PD-1 is an encouraging therapy for early-stage non-metastatic colon cancer patients with MSS. 1. Brenner, Lancet, 2014 2. Lin, Cancer Immunol Immunother, 2023 3. https://lnkd.in/gH876tXN 4. S Khaznadar, Oncotarget 5. Gan, Cancer Res, 2012 6. S Suntharalingam, New Engl J Med 7. A Elsayed, MABS 8. Dickinson, New Engl J Med, 2022 9. Kantarjian, New Engl J Med, 2017 10. T Elliot, Immunity #Immunotherapy #ImmunoOncology #BiTE #PD1 #Costimulation #Pharmaceutical

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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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💊🔬 #MondaysMajorMoves - Pharma Industry Updates Positive News: 🎉 World Health Organization's Milestone: Egypt eradicates malaria after nearly a century. 🤝 Strategic Partnership: Samsung Biologics signs a $1.24B production deal. 🚀 Clinical Success: Monopar Therapeutics Inc. Therapeutics' shares soar after Phase 3 success for Wilson disease drug. 🔥 Pipeline Development: Lundbeck plans to have four NMEs in Phase 3 trials in 2026, including an epilepsy drug from the Longboard Pharma acquisition. 💰 Septerna raises $288M in IPO to fund Phase 1 hypoparathyroidism drug targeting GPCRs. 🏭 GSK to invest up to $800M to expand Marietta, PA plant, doubling capacity and adding 200 jobs, including R&D facilities. 👁️ TEVOGEN BIO joins Microsoft's startup program to advance AI algorithms for drug development; shares rise 54%. 📊 Ipsen raises 2024 revenue growth forecast to 8% and operating margin over 31% after strong quarterly results. 📊 Sanofi reports Q3 revenue up 15.7% to €13.44B, boosted by 25.5% growth in vaccine sales. 🟢 FDA approves Pfizer's RSV vaccine Abrysvo for adults aged 18-59, expanding eligibility for vaccination. 🤝 M&A Activity: Merck acquires Modifi Bio for $30M upfront to expand its cancer drug pipeline. 🟢 BeiGene's Tevimbra receives positive EU opinion for extended use in gastric and esophageal cancers after improved survival in trials. Challenges and Setbacks: 📉 Clinical Trial Trends: Industry-sponsored trials decline in Europe. Dropping to 12% of global share; Spain bucks trend with increased studies. ❌ Job Cuts: Bayer to cut 400 jobs in France. ❌ Marinus Pharma's ganaxolone fails Phase 3 trial in tuberous sclerosis seizures; shares plummet 83%. 😢 Broad Institute of MIT and Harvard lays off 87 employees claiming to aim to stay at forefront of technological changes as key partnerships end. 🔴 Store Closures: Walgreens to close 1,200 pharmacies over 3 years as a cost cutting exercise amid rising expenses and lower prescription reimbursements ❌ Alto Neuroscience fails Phase 2b trial in depression; stock plunges 70% after missing primary endpoint. Interesting: 👁️ U.S. pharma CEOs shift political donations toward Democrats, with most supporting Democratic candidates over Republicans. 💼 Roche CEO urges authorities to block Novo Holdings' acquisition of Catalent Pharma Solutions, citing competition concerns. 🔄 Catalent Pharma Solutions assures clients company will continue as CDMO post-acquisition by Novo Holdings. What are your thoughts on these developments? #pharma #biotech #healthcare #drugdevelopment #clinicaltrials #biopharma #innovation #healthcarenews #pharmaindustry

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It's increasingly difficult for biotech companies to secure funding. As a result, biotechs are looking for new ways to communicate their value to investors. Development pipelines are traditionally seen as tools to gain regulatory approval for novel drugs, but this isn't the only role they can play. Pipelines can also be a valuable tool for securing investment. This is because a solid pipeline highlights your understanding of the development process and demonstrates how you aim to overcome certain challenges along the way. In an industry where 90% of drug candidates fail, this is an excellent way to provide investors with some confidence and peace of mind. #Funding #Biotech #VentureCapital #BusinessStrategies

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[JUST RELEASED] Global CAR-T Cell Therapy Market – Market Size, Forecasts, Trials & Trends, 2024 - https://lnkd.in/eCkW5rn Between 2017 and 2024, nine CAR-T products reached commercialization, and this number is estimated to reach double-digits by 2032. Of these, six are U.S.FDA-approved CAR-T cell therapies, each of which have received approvals in other major healthcare markets as well. The six U.S. FDA approved products include: Kymriah (tisagenlecleucel) Yescarta (axicabtagene ciloleucel) Tecartus (brexucabtagene autoleucel) Breyanzi (lisocabtagene maraleucel) Abecma (idecabtagene vicleucel) Carvykti (Ciltacabtagene autoleucel) In addition, two CAR-T therapies have received approval from the Chinese National Medical Products Administration (NMPA), Relma-cel and Yuanruida, and one CAR-T cell therapy has received approval from the Indian Central Drugs Standard Control Organisation (CDSCO), NexCAR19. The billion dollar CAR-T cell therapy market would not have been possible without the remarkable efficacy of the early CAR-T therapies in treating several types of blood cancers. The next frontier for CAR-T cell therapies will be to apply them in the fight against solid tumors. All of the nine marketed CAR-T cell therapy products and nearly three-quarters (75%) of the ongoing clinical trials utilize an autologous treatment approach. Thus, the development of allogeneic CAR-T cell therapies will open critical market opportunities. Another key issue is the “vein-to-vein” time or the time that elapses between apheresis and product delivery. For this reason, CAR-T therapies are usually recommended for the end-stage patients who have exhausted all other treatment options. Another challenge encountered by CAR-T therapies is the reimbursement issues across the U.S. and Europe. For the continued progress of CAR-T cell therapies, the industry is trying to mitigate these challenges. Several CAR-T players have started to use efficient gene-transfer tools to impregnate T cells with CARs, and there are numerous examples of partnerships to develop CRISPR and electroporation technologies to modify T cells. Some companies also use “on-off” switches that can turn off CAR-T cells to prevent toxicity. To date, the goal of achieving success with solid tumors remains elusive with clinical trials producing a low response rate. Thus, ongoing efforts within the CAR-T sector are focused on discovering effective solid tumor-specific antigens. The purpose of this report is to describe the current state of CAR-T cell therapies, as well as the future of the CAR-T industry landscape at large. Importantly, it describes details of the CAR-T cell therapy products approved to date, as well as late-stage CAR-T clinical trials that could lead to near-term market approvals. Ranging from small start-ups to billion-dollar companies, CAR-T companies are now proliferating in all healthcare markets worldwide. #cartcelltherapy #advancedtherapies #celltherapy

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Global CAR-T Cell Therapy Market – Market Size, Forecasts, Trials & Trends, 2024 - https://lnkd.in/eCkW5rn Between 2017 and 2024, nine CAR-T products reached commercialization, and this number is estimated to reach double-digits by 2032. Of these, six are U.S.FDA-approved CAR-T cell therapies, each of which have received approvals in other major healthcare markets as well. The six U.S. FDA approved products include: Kymriah (tisagenlecleucel) Yescarta (axicabtagene ciloleucel) Tecartus (brexucabtagene autoleucel) Breyanzi (lisocabtagene maraleucel) Abecma (idecabtagene vicleucel) Carvykti (Ciltacabtagene autoleucel) In addition, two CAR-T therapies have received approval from the Chinese National Medical Products Administration (NMPA), Relma-cel and Yuanruida, and one CAR-T cell therapy has received approval from the Indian Central Drugs Standard Control Organisation (CDSCO), NexCAR19. The billion dollar CAR-T cell therapy market would not have been possible without the remarkable efficacy of the early CAR-T therapies in treating several types of blood cancers. The next frontier for CAR-T cell therapies will be to apply them in the fight against solid tumors. All of the nine marketed CAR-T cell therapy products and nearly three-quarters (75%) of the ongoing clinical trials utilize an autologous treatment approach. Thus, the development of allogeneic CAR-T cell therapies will open critical market opportunities. Another key issue is the “vein-to-vein” time or the time that elapses between apheresis and product delivery. For this reason, CAR-T therapies are usually recommended for the end-stage patients who have exhausted all other treatment options. Another challenge encountered by CAR-T therapies is the reimbursement issues across the U.S. and Europe. For the continued progress of CAR-T cell therapies, the industry is trying to mitigate these challenges. Several CAR-T players have started to use efficient gene-transfer tools to impregnate T cells with CARs, and there are numerous examples of partnerships to develop CRISPR and electroporation technologies to modify T cells. Some companies also use “on-off” switches that can turn off CAR-T cells to prevent toxicity. To date, the goal of achieving success with solid tumors remains elusive with clinical trials producing a low response rate. Thus, ongoing efforts within the CAR-T sector are focused on discovering effective solid tumor-specific antigens. The purpose of this report is to describe the current state of CAR-T cell therapies, as well as the future of the CAR-T industry landscape at large. Importantly, it describes details of the CAR-T cell therapy products approved to date, as well as late-stage CAR-T clinical trials that could lead to near-term market approvals. Ranging from small start-ups to billion-dollar companies, CAR-T companies are now proliferating in all healthcare markets worldwide. #cartcelltherapy #advancedtherapies #celltherapy

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[JUST RELEASED] Global CAR-T Cell Therapy Market – Market Size, Forecasts, Trials & Trends, 2024 - https://lnkd.in/eCkW5rn Between 2017 and 2024, nine CAR-T products reached commercialization, and this number is estimated to reach double-digits by 2032. Of these, six are U.S.FDA-approved CAR-T cell therapies, each of which have received approvals in other major healthcare markets as well. The six U.S. FDA approved products include: Kymriah (tisagenlecleucel) Yescarta (axicabtagene ciloleucel) Tecartus (brexucabtagene autoleucel) Breyanzi (lisocabtagene maraleucel) Abecma (idecabtagene vicleucel) Carvykti (Ciltacabtagene autoleucel) In addition, two CAR-T therapies have received approval from the Chinese National Medical Products Administration (NMPA), Relma-cel and Yuanruida, and one CAR-T cell therapy has received approval from the Indian Central Drugs Standard Control Organisation (CDSCO), NexCAR19. The billion dollar CAR-T cell therapy market would not have been possible without the remarkable efficacy of the early CAR-T therapies in treating several types of blood cancers. The next frontier for CAR-T cell therapies will be to apply them in the fight against solid tumors. All of the nine marketed CAR-T cell therapy products and nearly three-quarters (75%) of the ongoing clinical trials utilize an autologous treatment approach. Thus, the development of allogeneic CAR-T cell therapies will open critical market opportunities. Another key issue is the “vein-to-vein” time or the time that elapses between apheresis and product delivery. For this reason, CAR-T therapies are usually recommended for the end-stage patients who have exhausted all other treatment options. Another challenge encountered by CAR-T therapies is the reimbursement issues across the U.S. and Europe. For the continued progress of CAR-T cell therapies, the industry is trying to mitigate these challenges. Several CAR-T players have started to use efficient gene-transfer tools to impregnate T cells with CARs, and there are numerous examples of partnerships to develop CRISPR and electroporation technologies to modify T cells. Some companies also use “on-off” switches that can turn off CAR-T cells to prevent toxicity. To date, the goal of achieving success with solid tumors remains elusive with clinical trials producing a low response rate. Thus, ongoing efforts within the CAR-T sector are focused on discovering effective solid tumor-specific antigens. The purpose of this report is to describe the current state of CAR-T cell therapies, as well as the future of the CAR-T industry landscape at large. Importantly, it describes details of the CAR-T cell therapy products approved to date, as well as late-stage CAR-T clinical trials that could lead to near-term market approvals. Ranging from small start-ups to billion-dollar companies, CAR-T companies are now proliferating in all healthcare markets worldwide. #cartcelltherapy #advancedtherapies #celltherapy

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Global Regenerative Medicine (RM) Industry Database - Featuring 1,550+ Companies [Updated for August 2024] - https://lnkd.in/eYxEsiw The regenerative medicine industry has experienced remarkable growth propelled by advancements in cell therapies, gene therapies, stem cell research, tissue engineering, exosome therapeutics, 3D printing, cultured meat production, and beyond. This burgeoning field offers promising solutions for treating a myriad of diseases and injuries by harnessing the body's natural ability to heal and regenerate. This growth is both revolutionizing healthcare by offering new treatment modalities and addressing unmet medical needs on a global scale. The number of RM market competitors keeps growing every month. If you work in this market area, it can be hard to keep up. Thankfully, you can now access the world’s only searchable, sortable database of regenerative medicine companies that is publicly available. This week only, it is available for 50% off.

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Acurx Pharmaceuticals Announces Presentation of Ibezapolstat Phase 2 Clinical Trial Results for CDI at Scientific Conference   • Additional analyses of clinical and microbiological data from the Phase 2b segment show favorable gut microbiome changes including increased relative proportion of Actinobacteria in ibezapolstat-treated patients with C. difficile Infection (CDI) • Results consistent with those shown in earlier human volunteer studies and Phase 2a studies • Preservation and increased concentrations of beneficial Firmicute (Bacilotta) phylum known to be involved in bile acid homeostasis and short chain fatty acid metabolism was demonstrated  • Actinobacteria and Bacteroides preservation also confirmed using quantitative analysis   https://lnkd.in/eseFt8GZ   #nasdaq #acxp #antibiotics #acurx Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP) David Luci

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Join BIORCHESTRA Co., Ltd. on-site for the Fierce Biotech Summit in Boston from September 30 to October 1, where we will share detailed updates on our IV- and SC-formulated RNAi therapeutics, including: 🔬 IV-Formulated RNAi #CNS Delivery: Showcasing our lead Amyotrophic Lateral Sclerosis (ALS) program, #MAPT program in Alzheimer's, and #SNCA program in Parkinson's disease. 🧬 SC-Formulated RNAi #MALFD Delivery: This highlights exciting news in our obesity program, which generated significant outcomes targeting the #INHBE gene. These results compare head-to-head against commercially available GLP1 programs. ⏳ 18-Week Durability Outcomes: Revolutionary results in epilepsy management through our novel IV-formulated RNAi delivery platform. Explore the intricacies of our RNAi delivery platform, designed to enhance stability, safety, and targeted delivery efficacy of diverse RNAi cargos (e.g., ASO, siRNA, and mRNA). Join us to delve into cutting-edge CNS and metabolic disease therapeutics and discuss the transformative potential of RNAi technology. 📧 Contact: [email protected] AbbVie, Christian R. Schubert, Avidity Biosciences, Inc., Alnylam Pharmaceuticals, Arbor Biotechnologies, Arrowhead Pharmaceuticals, AstraZeneca, BioMarin Pharmaceutical Inc., Fanny Cavalie, Dicerna Pharmaceuticals, Inc., Deep Genomics, Eli Lilly and Company, Christine Donahue, GSK, Horizon, Ionis Pharmaceuticals, Inc., Bayer | Pharmaceuticals, Christian Rommel, The Janssen Pharmaceutical Companies of Johnson & Johnson, Novartis Gene Therapies, Novartis, Novo Nordisk, Jacob Petersen, ONO PHARMACEUTICAL CO., LTD., Otsuka Pharmaceutical Co., Ltd., Pedro Morais, Pfizer, #pfizercolleague, Benjamin Yaden, Regeneron, RNA Therapeutics and Oligonucleotides, Roche, Sanofi, Silence Therapeutics plc, Spark Therapeutics, Inc., Wave Life Sciences, NYSE. Nomura Securities International, Inc., Leerink Swann & Company #RNAi #CNS #ALS #Parkinsons #Alzheimers #Obesity #Epilepsy.  Learn more: https://lnkd.in/gD5rKNHq Fierce Life Sciences Events

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Global Regenerative Medicine (RM) Industry Database - Featuring 1,550+ Companies [Updated for September 2024] - https://lnkd.in/eYxEsiw The regenerative medicine industry has experienced remarkable growth propelled by advancements in cell therapies, gene therapies, stem cell research, tissue engineering, exosome therapeutics, 3D printing, cultured meat production, and beyond. This burgeoning field offers promising solutions for treating a myriad of diseases and injuries by harnessing the body's natural ability to heal and regenerate. This growth is both revolutionizing healthcare by offering new treatment modalities and addressing unmet medical needs on a global scale. The number of RM market competitors keeps growing every month. If you work in this market area, it can be hard to keep up. Thankfully, you can now access the world’s only searchable, sortable database of regenerative medicine companies that is publicly available. This week only, it is available for $150 off.

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Interested in the latest developments at the FDA's Office of Therapeutic Products (#OTP)? Join a free, public webinar on June 14th featuring Dr. Nicole Verdun, OTP's Super Office Director, and Rachael Anatol, Ph.D., Deputy Super Office Director. What you'll learn: The new "Super Office" structure of the OTP Progress on hiring new staff An overview of the OTP's organization Brought to you by Alliance for Regenerative Medicine. Please use the link in the post below. #celltherapy #genetherapy #cellandgenetherapy #advancedtherapies #lifescience #lifesciences #regulatory #structure #biotech #biotechnology #webinar #productupdate #regulatorystructure

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As we approach a potential vote in the United States Senate, now is the time for #biotech leaders to stand up for reputable #CDMOs in China like WuXi AppTec. The #biosecure act is misguided and politically motivated in an election year. Trying to discredit companies that have become trusted members of the US #biopharma ecosystem and key contributors to our R&D programs based on unsubstantiated and unvalidated allegations is disgraceful. What is a “biotechnology company of concern?” Biotechnology Innovation Organization John F. Crowley

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Another important step.

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December 13 and 16, 2024 headlines from the Collabrity Report, a daily summary of financings for therapeutic biotechnology company executives and investors - www.collabrityreport.com New! Collabrity Report (CR) Analytics NewAmsterdam’s $479 Million Public Offering AbbVie’s $200 Million Acquisition of Nimble Redesign’s $175 Million Fund Raise Pharming’s $66 Million Acquisition of Abliva Emergent’s $50 Million Grant Inventiva’s $22 Million Equity Investment

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