David Saussy

🎤 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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CDMOs eye industrywide growth in 2025 as many spurn BIOSECURE Act, CPHI research shows - Fierce Pharma After experiencing a series of ups and downs in recent years, the biopharma contracting business is gearing up for a rebound in 2025. That's according to new research from CPHI Milan ahead of the group's manufacturing conference next month. As it stands, 49% of the CDMO industry is “highly positive” on contract services growth in the next 18 months, CPHI said in a release, citing new research pooling insights from 280 pharma companies. The optimistic mood comes despite an uneven recovery in the biopharma industry after the pandemic. While funding has flowed to later-stage projects, early-stage assets continue to face a funding deficit, Gil Roth, president of the Pharma & Biopharma Outsourcing Association said in a statement. “This means that the lifeblood of CDMOs—projects moving through the development pipeline and smaller customers sticking with key service providers—remains hobbled,” Roth said. https://lnkd.in/eegVNZKf

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Want glycan analysis method expertise? In this video, our Navin Rauniyar discusses his recently published study. Check it out! #proteinsciences #protein #groundbreaking #biotech #CDMO #analyticaldevelopment #glycananalysis

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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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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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Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP) to Discuss Second Quarter 2024 Financial Results on August 9, 2024 Conference Call and Provide Business Update   Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP), a clinical stage #biopharmaceutical company focused on developing new #antibiotics for difficult-to-treat #bacterial infections, has announced a conference call to discuss its second quarter 2024 financial results. The call is scheduled for August 9, 2024, at 8:00 am ET, before the U.S. financial markets open.   The company's President and CEO, David Luci, along with CFO Robert Shawah, will host the call to present the #financial results and provide a business update. #Investors and interested parties can join the call using the provided toll-free number for U.S. participants or international access numbers.   https://lnkd.in/esMuCywW #nasdaq #acxp #antibiotics #acurx Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP) David Luci

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RAPT Therapeutics Therapeutics and Shanghai Jemincare Pharmaceutical Announce Exclusive License Agreement for Novel Long-Acting anti-IgE Antibody SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing novel therapies for patients with significant unmet needs in inflammatory diseases, and Shanghai Jemincare Pharmaceutical Co., Ltd (“Jemincare”), a subsidiary of Jiangxi Jemincare Group, a leading pharmaceutical company in China, today announced they have entered into an exclusive license agreement for JYB1904 (RAPT designation RPT904), a clinical-stage, half-life extended anti-immunoglobulin E (IgE) monoclonal antibody. Under the terms of the license agreement, RAPT is granted worldwide rights excluding mainland China, Hong Kong, Macau and Taiwan (together, the “Jemincare Territory”) to develop and commercialize RPT904. As consideration for the license, Jemincare receives a $35 million upfront license fee, up to $672.5 million in additional payments upon the achievement of various regulatory and commercial milestones, and royalties on future sales of RPT904 outside the Jemincare Territory. RPT904 is being developed to offer patients a potentially improved therapeutic option compared to omalizumab (marketed as Xolair®), an anti-IgE monoclonal antibody approved for several allergic disorders, including asthma, chronic spontaneous urticaria (CSU), chronic rhinosinusitis with nasal polyps and, most recently, food allergy. RAPT plans to pursue development of RPT904 initially in food allergy. Separately, Jemincare is conducting Phase 2 clinical trials of JYB1904 in China in asthma and CSU. “We’re delighted to partner with Jemincare and excited by RPT904 and its potential to be a novel best-in-class treatment option for patients with food allergy. Omalizumab’s rapid uptake in food allergy since its approval earlier this year confirms the high unmet need and large opportunity in this growing market,” commented Brian Wong M.D., Ph.D., President and CEO of RAPT. “RPT904 targets the same clinically validated epitope as omalizumab and combined with extended half-life, gives this molecule a best-in-class profile. We plan to initiate a Phase 2b clinical trial of RPT904 in food allergy in the second half of 2025.” https://lnkd.in/e92YkAmT

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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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For anyone building diagnostics: Very cool approach to gain valuable insights into FDA decision summaries. Brendan O'Leary scraped the FDA’s decision summaries and provided some simple R code for anyone to get up and running in no time to prod and probe those summaries. Very nice work!

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Elevation Oncology Partners with Synaffix BV in $368M Deal to Advance HER3 ADC Development Synaffix announces that it has entered into a licensing agreement with Elevation Oncology to develop EO-1022, a HER3-targeting Antibody-Drug Conjugate (ADC) leveraging Synaffix’s advanced platform technologies. This collaboration aims to create a highly effective and safer therapy for HER3-positive solid tumors. With up to $368 million in milestone payments, this partnership highlights the transformative potential of ADCs in precision oncology and patient care. Cutting-Edge Technology Integration Synaffix provides Elevation Oncology with global access to its clinical-stage, site-specific ADC technology platform, including GlycoConnect® antibody conjugation, HydraSpace® polar spacer, and the toxSYN® linker-payload SYNstatin E™. These advanced technologies enable the creation of a differentiated HER3 ADC candidate with enhanced efficacy and tolerability, holding the potential to set a new benchmark in oncology therapeutics. Strategic and Financial Framework Under the agreement terms, Synaffix will receive up to $368 million, contingent on achieving specific clinical, regulatory, and commercial milestones. Additionally, Elevation Oncology will handle research, development, and commercialization, while Synaffix will manage manufacturing components tied to its proprietary platform. https://lnkd.in/em89DTzB

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Astellas Pharma's subsidiary, Xyphos Biosciences, is partnering with Poseida Therapeutics, Inc. Therapeutics to develop two "convertible" CAR-T therapy candidates for solid tumors. The goal of this collaboration is to create a safer option compared to traditional cell therapies. Xyphos' platform and Poseida's genetic editing technology will be combined to make more effective solid tumor cell therapies. Astellas will pay Poseida $50 million upfront and another $550 million in potential milestone payments, with low double-digit royalties if a therapy is commercialized. #advancedtherapies #cart #therapies #celltherapy #cellandgenetherapy #regenerativemedicine #solidtumors #cancer #oncology #milestones #royalties #collaboration #biotech #biotechnology #pharma #pharmaceuticals #lifesciences #therapeutics Kristin Yarema, Ph.D. Gary Starling

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🎉 Huge congratulations to Orum Therapeutics for their deal yesterday! Vertex bolts on Orum's Degrader-Antibody Conjugate(DAC) conditioning agents to ImmunoGen's ADC conditioner, acquired in March 2023. But what drives Vertex to explore novel conditioner candidates?   Traditional conditioning agents are poorly tolerated and have posed significant challenges for Hematopoietic Stem Cell (#HSC) Cell and Gene Therapies (CGT), such as Vertex's Casgevy. These hurdles are now being addressed by novel, less aggressive conditioners, as above. For instance, companies like Actinium Pharmaceuticals are also advancing novel conditioning agents in this space. Thus, improved conditioning agents will play a crucial role in expanding the application of HSC CGT.   Another critical challenge in HSC CGT is effective #mobilization. Emerging CGTs often require a higher yield of HSCs than conventional HSC transplantation in cancer. When it comes to in vivo approaches, having more HSCs available in peripheral blood will be critical since ex vivo collection and engineering are not feasible. Thus, there is a growing demand for enhanced HSC mobilizers in this evolving field.   Here comes #GPC201, a potential best/fastest-in-class HSC mobilizer currently in phase 2 clinical trials in the US. It is demonstrating superior features for HSC transplantation in multiple myeloma. As the universe of various CGT pipelines expands rapidly, GPC201 is poised to showcase its full potential.   I eagerly anticipate the transformative impact of pairing GPC201 with novel conditioning agents to enhance patients' HSC transplantation experiences.   Curious about GPC201? 👉👉👉 GPCR Therapeutics

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NY Illumina/GRAIL lawsuit Final Part: Summation "Illumina’s directors and officers, foreseeing their non-exculpated liability in connection with their decision to close the Acquisition in spite of the EC standstill order, determined to shift that liability to Illumina at a cost of up to $100 million, made that shifting of liability a requirement before they would approve the Acquisition, and at the same time abandoned $150 million in insurance for the Company that could have funded Illumina’s financial losses associated with the Acquisition. At no time was this approved by a disinterested vote of disinterested directors or stockholders, and it was not fair to Illumina. By stripping away the Company’s insurance down to $0, while at the same doubling their D&O insurance to $300 million, Illumina’s directors and officers abandoned their fiduciary duties to the Company and left Illumina unprotected while protecting themselves at Illumina’s expense." "Prior to acquiring GRAIL, Illumina’s financials were strong, and its share price reflected that as it traded at its closing peak of $524.24 on August 16,2021. However, after the Acquisition, GRAIL acted like a millstone around Illumina’s neck, costing billions of dollars to maintain while providing no benefit to Illumina." "The Acquisition ultimately dropped Illumina’s cash balance to zero. On March 8, 2021, Illumina entered into a credit agreement with Bank of America for $750 million to help pay for the Acquisition and to keep the lights on at Illumina. When that proved insufficient to carry Illumina’s rising costs, on March 16, 2021, Illumina issued another $1 billion in notes. Less than two years later, on January 5, 2023, Illumina deemed it necessary to sign another $750 million credit agreement to “finance the working capital needs, and for general corporate or other lawful purposes, of Illumina and its subsidiaries.” Illumina has been unable to seek any other potential acquisition opportunities as it spent the bulk of its cash balances on the Acquisition." "By reason of the Director Defendants’ positions with the Company as directors, they owed the Company and its stockholders a duty of loyalty. deSouza and Dadswell owed the same duty of loyalty as Illumina officers. By closing the Acquisition, Defendants breached that duty in three ways: causing a knowing violation of the law, consciously disregarding their fiduciary duties and engaging in self-dealing."

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Vertex Pharmaceuticals Expands in vivo Gene Editing Delivery Horizon with Orna Therapeutics Partnership Strategic Collaboration: Vertex has entered a three-year agreement with Orna Therapeutics to leverage Orna’s lipid nanoparticles (LNPs) for in vivo delivery of gene editors, targeting advancements in treatments for sickle cell disease and beta thalassemia. Unlike existing therapies like Casgevy, this approach aims to eliminate the need for ex vivo cell editing and complex chemotherapy conditioning. Financial Details: The deal includes an upfront payment of $65 million (partially as a convertible note), with Orna eligible for up to $635 million in milestones for the sickle cell and thalassemia programs and an additional $365 million for up to 10 other indications. Tiered royalties on potential products are also part of the agreement. Long-Term Impact: This in vivo gene editing strategy could simplify manufacturing, increase accessibility, and potentially provide a more patient-friendly alternative to existing therapies, which currently reach only a fraction of those in need. #65mupfront #635milestones #365madditional #funding #financing #cellandgenetherapy #invivo #geneediting #sicklecell #thalassemia #raredisease #biotech #biotechnology #lifesciences #patientaccess #LNP

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Dr. Nina E. Weisser, Director of Preclinical, Multispecific Antibody Therapeutics, recently authored an article published in Drug Target Review discussing the development of trispecific T cell engagers (TriTCEs) with integrated CD28 costimulation. TriTCEs are designed to address the limitations of conventional bispecific TCEs in treating solid tumors by providing balanced T cell activation, leading to potentially more sustainable anti-tumor responses. This innovative approach could significantly advance immunotherapy for patients with difficult-to-treat solid tumors. Access the article: https://lnkd.in/ewRJvPA9

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Why Gilead Sciences and Arcellx Anito-cel's data shows Promise in Multiple Myeloma Therapy Innovative Safety Profile: Early clinical data suggest anito-cel offers a reassuring safety advantage, with no reported cases of Parkinsonism, cranial nerve palsies, or Guillain-Barré syndrome—potentially addressing critical quality-of-life concerns. Encouraging Efficacy: With a 97% response rate and a median progression-free survival of 30.2 months in Phase 1, anito-cel demonstrates strong potential as an effective treatment option in heavily pretreated multiple myeloma patients. Cutting-Edge Technology: Anito-cel’s unique D-Domain binding technology may contribute to its promising safety and efficacy, reflecting innovation in CAR-T therapy design. #CellTherapy #MultipleMyeloma #InnovationInHealthcare #CAR_T #advancedtherapies #biotech #biotechnology #lifesciences #Data #ASH2024 https://lnkd.in/eCmJ_vP3

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