Danielle Marra

Curious to know what we do at Appro-Rx?? Watch this short video to find out!

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To discuss how to de-risk your viral vector prior to clinical trials, please reach out to our team at #ISCT2024 in Vancouver. Farzin Farzaneh, PhD Traci Kyes Makis Sigalas

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Absolutely great meeting and productive exchange around trends and needs in today's Pharma world. Glad to hear points from Biotech, Mid-Pharma and Large Pharma attendees on novel program and portfolio successful approaches as well as challenges. Superb organization and focus. #Pharmaceutical development #Large Pharma #Biotech #project #program #portfolio #strategy

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Deciphera Pharmaceuticals to be Acquired by ONO Pharmaceutical For $2.4 Billion WALTHAM, Mass.--(BUSINESS WIRE)--Apr. 29, 2024-- Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, today announced that it has entered into a definitive merger agreement with ONO Pharmaceutical Co., Ltd. (ONO), under which ONO will acquire all outstanding shares of Deciphera common stock for $25.60 per share in cash through a tender offer followed by a merger of Deciphera with a wholly-owned subsidiary of ONO (the “Acquisition”), for a total equity value of $2.4 billion. The boards of directors of both companies have unanimously approved the transaction. Together, ONO and Deciphera will accelerate their shared vision to deliver innovative new drugs and serve patients around the world. Deciphera brings specialized research and development capabilities in kinase drug discovery, well-established commercial and sales platforms in the United States and Europe, and global clinical development capabilities. https://lnkd.in/dY_hDwFT

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As NAMs are becoming more relevant and accessible in today’s chemical product development, Labcorp is innovating in vitro alternatives so clients around the globe can leverage these new assays. From standard techniques to novel solutions, we can identify an approach that fits your product’s specific regulatory requirements. Discover your NAM options today. #CropProtection #ChemicalDevelopment #InVitroStudyAlternatives NewApproachMethodologies #NAMs

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🦠 Bristol Myers Squibb (BMS) and Cellares announced a worldwide capacity reservation and supply agreement for the manufacture of CAR T cell therapies in a transaction valued up to $380M. Cellares will optimize, automate, and tech-transfer select BMS CAR T cell therapies onto its automated and high-throughput manufacturing platform, the Cell Shuttle™. Cellares will dedicate multiple Cell Shuttle and Cell Q™ systems with fully automated, high-throughput quality control for BMS’ exclusive use. This strengthens BMS’ existing global network of cell therapy manufacturing facilities and provides increased agility, improved scalability and potential to improve turnaround time to support the company’s CAR T inline and pipeline assets 🦠 Legend Biotech announced that the European Commission has granted approval of CARVYKTI® for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent, have demonstrated disease progression on the last therapy and are refractory to lenalidomide. 🦠 Vertex Pharmaceuticals and TreeFrog Therapeutics announced that Vertex has obtained an exclusive license to TreeFrog's proprietary cell manufacturing technology, C-StemTM, to optimize production of Vertex's cell therapies for type 1 diabetes (T1D). C-Stem™, is designed to mimic the natural microenvironment, allowing cells to grow exponentially in 3D. The technology will enhance Vertex's ability to generate large amounts of fully differentiated cells for its portfolio of T1D cell therapies 🦠 TScan Therapeutics announced closing of upsized public offering. The gross proceeds to TScan from this offering were approximately $150.1 million, prior to any exercise of the underwriters’ option to purchase additional shares of voting common stock and before deducting underwriting discounts and commissions and other estimated offering expenses payable by TScan. The company intends to use the net proceeds from the offering for general corporate purposes. TScan expects its existing cash, cash equivalents and marketable securities will enable it to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2026. 🦠 SCG Cell Therapy and the A*STAR - Agency for Science, Technology and Research in Singapore launched joint laboratories to advance the development of iPSC technology to produce novel cell therapies that meet GMP standards. The collaboration is supported by $22 million under Singapore’s Research, Innovation and Enterprise 2025 Plan. The joint laboratories leverage SCG’s and A*STAR’s proprietary technologies to develop scalable GMP-grade iPSC and therapeutic products

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In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

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(Benefit-Risk Management / Gene Therapy / FDA Town Hall CMC Mtg) As gene Rx FDA guidance and precedent regulatory decisions are being actively shared or made publicly accessible, possibly worthwhile if you or your colleagues need to understand the "language" of gene therapy and stay with the conversation (listen to the successes and the challenges). Otherwise, for those that are already in this space, you know this is another one of those essential touchpoints to see what FDA is considering. Enjoy, happy Saturday! #fda #biologics #genomics #genetherapy #cmc #cber #drugmanufacturing #biologicmanufacturing (CMC = chemistry, manufacturing, and controls)

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Encourage people to attend the inaugural SITC Immuno-Oncology Drug Development Summit September 30-October 1, 2024 in Boston, MA. Co-organized by Amy McKee, Pedro Romero, Luisa Salter-Cid, Alexandra Snyder, Jon Wigginton. We now have over 25 IO drugs that have been approved, and this number will only continue to grow as science accelerates. From this experience, we as a field have learned that there are important distinctions in developing IO agents compared to conventional modalities such as targeted and cytotoxic therapeutics. There has been a significant need for some time for a program like this, to reflect on our learnings to date and discuss/debate the future. This program will include key stakeholders from the academic, pharma/biotech, regulatory and financial sectors, and is intended to promote dialogue between these key contributors to IO drug development as we all work together to make things better for patients. Terrific support from SITC leadership, including Leisha Emens (President), James Gulley (Vice-President), SITC board, SITC biotech committee led by Kristen Hege and Zhen Su, and of course SITC staff team led by Mary Dean. #cancer #oncology #immunooncology #cancerimmunotherapy #immunotherapy #drugdevelopment #bispecific #cytokine #celltherapy

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An IRB can review a "minor" study change via expedited review - but the regulations don't really define what "minor" means. Find out how IRBs navigate this grey area in our blog:

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❔ How do we position regional and community sites for success and mitigate risks for sponsors? ❔ In our new Oncology report, Parexel’s Karen McIntyre and Angela Hirst weigh in on why the traditional approach to site selection in oncology has created bottlenecks at major centers, potentially limiting patient access to life-changing therapies. 🚧 That’s why Parexel is bridging the gap by partnering with regional and community sites while still ensuring the high-quality standards of academic centers. Find out how: #ResearchSites #Oncology #NovelInsights

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❔ How do we position regional and community sites for success and mitigate risks for sponsors? ❔ In our new Oncology report, Parexel’s Karen McIntyre and Angela Hirst weigh in on why the traditional approach to site selection in oncology has created bottlenecks at major centers, potentially limiting patient access to life-changing therapies. 🚧 That’s why Parexel is bridging the gap by partnering with regional and community sites while still ensuring the high-quality standards of academic centers. Find out how: https://lnkd.in/ewxTEJXm #ResearchSites #Oncology #NovelInsights

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We're pleased to share new #GeneTherapy research that provides critical knowledge to support adeno-associated virus (AAV)-mediated gene therapies. In this recent publication, Christos J. Petropoulos, vice president for Labcorp’s research and development, and Terri Wrin, technical director for Labcorp’s virology research and development, uncover the global seroprevalence of neutralizing antibodies against AAV serotypes crucial for human gene therapies. With data from 10 countries, this study provides valuable insights into the prevalence of neutralizing antibodies across different demographics. Explore the full research article to learn how these findings are shaping the future of clinical gene therapies: https://lnkd.in/gnsBPS_W #AAVGeneTherapy #NAb

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In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

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Bispecific ADCs have the potential to deliver enhanced efficacy over canonical ADC approaches for the same payload dose, and this is exactly what we see with ALM-401. Great to see this novel engineered Bispecific ADC progressing forward.

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While cell and gene therapies (#CGT) are undeniably expensive, their financial impact can mean something different for differently sized plans. During episode 19 of Astonishing Healthcare, Casey Stockman, PharmD (VP, PBA Services) broke down what the financial impact could be for a national plan with only a few eligible members and a smaller regional plan with one eligible member. Listen to the full podcast here: https://okt.to/NmycuS

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In 2023, there were +50 FDA approvals. How many were successful in launch? Interesting article with thoughts on navigating the complexities. “If you’re rushing to market to hit clinical thresholds and not being strategic about how that asset is going to differentiate, you’re going to end up with a lackluster launch, even if the science has potential. That’s the category we want to zero in on the most — how do you take pretty good science, or even great science, and develop it in a way that the commercial market will accept it?”

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Read Parexel's latest article on differentiating new drugs in crowded markets and the anticipatory steps to take, ensuring optimal go-to-market outcomes! In this piece, my colleagues, Aleksandra (Ola) Lubojemska and Andreas Lysandropoulos MD, PhD, provide valuable insights and expert advice on navigating the evolving neuroscience landscape and maximizing market success. Read more about the anticipatory steps and innovative approaches accelerating patient access to life-saving treatments. #Neuroscience #MarketAccess #Regulatory #ParexelBiotech

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