Precision Medicine Online’s Post

sharing this post about our recent paper and why we thought it was important to disclose lessons learned in ADC development (and not just the successes).

2023 4Q and YE results Verve Therapeutics announced today 2023: year of great progress with human proof of concept for in vivo base editing PCSK9 2024: year of clinical execution with 3 products in clinical trials 2023 1. in vivo base editing works in humans 2. regulatory path in U.S. with IND clearance 3. gained @EliLillyandCo as partner  4. strong balance sheet, ended with $624M in cash, runway to late 2026 5. got 2 more programs ready for clinic 2024 1. finish Heart-1 Ph1 trial (PCSK9) including dosing U.S. patients, data update 2H 2024 2. start Heart 2 Ph1 trial (VERVE-102, PCSK9 target, with GalNAC-LNP delivery) in 1H 2024 3. Start VERVE-201 Ph1 trial (ANGPTL3 target) in 2H 2024 4. Continue progress on research programs (LPA, undisclosed ASCVD target, liver disease target collaboration with Vertex) Should be an amazing year! Kudos and thanks to team. https://lnkd.in/gXDvZEBP

Multiple Myeloma Trial Enrollment Discontinued for KarMMa-9 Due to Advances in Treatment Options Bristol Myers Squibb and 2seventy bio announced the discontinuation of patient enrollment in the Phase 3 KarMMa-9 trial, which was investigating Abecma® (idecabtagene vicleucel) with lenalidomide maintenance in newly diagnosed multiple myeloma patients. Despite this, Bristol Myers Squibb and 2seventy bio remain committed to advancing the science of multiple myeloma treatment and expanding the global reach of Abecma. Both companies continue their efforts in multiple myeloma research through ongoing trials and commercial availability of Abecma, while expressing gratitude to the patients and staff involved in the KarMMa-9 trial. For more details please click the link! https://lnkd.in/dU9t34gW #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

We are excited to announce Qu Biologics Inc. as a Tier 3 Sponsor of the 11th Aging Research & Drug Discovery Meeting. Qu Biologics’ first-in-class innate immunomodulation platform is designed to transform the fields of health restoration and healthy longevity by restoring the cells, pathways, and roles of innate immunity to downregulate the mTOR pathway, clear inflammation, and reverse underlying etiologies of aging, metabolic disease, chronic inflammatory disease, and cancer. With four completed Phase 2 studies demonstrating proof of principle and excellent safety profile, Qu’s Site Specific Immunomodulators (SSIs) are poised to transform the way we think about the treatment and prevention of disease, the optimization of health, and the extension of healthy longevity. #ARDD2024 will feature a dynamic mix of academic institutions, clinicians, industry leaders, and pharmaceutical companies, fostering rich dialogues and unveiling new research in the field of aging. Mark your calendars for August 26-30, 2024 and register now at agingpharma.org https://lnkd.in/giHKd6F6 #ARDD2024 #DrugDiscovery #AgingResearch

In case you missed all the posts, emails, and press releases about it, here's a friendly reminder about this week's B2i Digital Featured Conference: The 3rd Annual ROTH Healthcare Opportunities Conference, which will be held on Wednesday, October 9, 2024, at the Metropolitan Club in New York, NY. This invitation-only event connects executives from 30 public companies in the healthcare sector with institutional investors. The conference features one-on-one and small group meetings, providing in-depth insights into each company’s strategies and potential. Highlights include: - 25-minute in-person meetings between investors and executives. - Themed industry panels focusing on Brain Disorders, Solid Tumors, Hematologic Malignancies, and Oncology. - Breakfast and lunch panels featuring industry experts. - Cocktail reception for networking opportunities. Confirmed public company participants include industry leaders such as - Baird Medical (BDMD) - Beyond Air®, Inc. (XAIR) - Biofrontera Inc. (BFRI) - BioRestorative Therapies, Inc. (BRTX) - Candel Therapeutics, Inc. (CADL) - Cardiol Therapeutics Inc. (CRDL) - Cellectar Biosciences, Inc. (CLRB) - CervoMed Inc. (CRVO) - Chemomab Therapeutics Ltd. (CMMB) - Cingulate Inc. (CING) - Cognition Therapeutics, Inc. (CGTX) - Fortress Biotech, Inc. (FBIO) - Gain Therapeutics, Inc. (GANX) - GeoVax Labs, Inc. (GOVX) - GT Biopharma, Inc. (GTBP) - IRadimed Corporation. (IRMD) - Journey Medical Corporation. (DERM) - Longeveron Inc (LGVN) - Moleculin Biotech, Inc. (MBRX) - Nuvectis Pharma, Inc. (NVCT) - Nuwellis, Inc. (NUWE) - Orthofix Medical Inc. (OFIX) - Ovid Therapeutics Inc. (OVID) - Replimune Group, Inc. (REPL) - Reviva Pharmaceuticals Holdings, Inc. (RVPH) - Sensus Healthcare, Inc. (SRTS) For profiles of participating companies, please visit https://lnkd.in/eNXsQ7Cc Just so you know, pre-registration is required and subject to approval by ROTH. For more information and registration details, visit: https://lnkd.in/esxwj854 I look forward to seeing the ROTH team on Wednesday: Sagar Sheth, Byron Roth, Isabel Mattson-Pain, Aaron Gurewitz, Nazan Akdeniz, Amanda McAdams, Otilia "Charo" Montano, Benjamin Bowen, James Antonopoulos, Jason Wittes, Jonathan Aschoff, Ph.D., Boobalan Pachaiyappan, Ph.D., Keith Wellen and many others. ROTH Capital Partners is a relationship-driven investment bank headquartered in Newport Beach, CA. For more information on ROTH, please visit www.roth.com.

We're thrilled to share our latest article, "A Microvascularized In Vitro Liver Model for Disease Modeling and Drug Discovery"! This innovative 3D human liver model integrates hepatocytes, stellate cells, and endothelial cells in a fully vascularized structure, mimicking complex liver functions like steatosis and fibrosis. With high scalability for drug testing and disease modeling, this system offers unparalleled accuracy in pharmaceutical research, reducing reliance on animal models and providing more human-relevant insights. 🔬 Why this matters: This liver model gives researchers and pharmaceutical companies a robust platform to improve drug efficacy predictions, paving the way for more successful treatments for liver-related diseases. We are proud of this milestone and the incredible potential it holds for advancing personalized medicine and enhancing drug development pipelines. 🔗 Read the news article and paper: https://lnkd.in/eie74Xvj #liverfibrosis #livermodel #drugdiscovery #pharma #diseasemodeling

Significant milestones achieved by AbbVie | A global biopharmaceutical innovator company addressing several unmet medical needs, and focusing in the fields of immunology, oncology, neuroscience, virology, and women's health. Firstly, in the ADC world (since we love it so much) the U.S. Food and Drug Administration has granted full approval for ELAHERE™ (mirvetuximab soravtansine-gynx), expanding treatment options for certain patients with ovarian cancer. ELAHERE™ is an antibody-drug conjugate that targets the folate receptor alpha, commonly found on ovarian cancer cells. This approval follows positive results from clinical trials demonstrating its efficacy and safety in advanced ovarian cancer patients who have undergone multiple lines of therapy. AbbVie's commitment to developing novel treatments for cancer patients is underscored by this significant achievement today. In another strategic move, AbbVie has announced its acquisition of Landos Biopharma, further strenghtening its portfolio in inflammatory and autoimmune diseases. This acquisition aligns with AbbVie's ongoing efforts to address unmet medical needs in the therapeutic areas mentioned above. Landos Biopharma, Inc. brings innovative drug candidates and a proprietary platform for the development of oral therapeutics targeting autoimmune diseases. As the company continues to expand its capabilities, portfolio and offerings, patients can look forward to advanced treatment options and improved outcomes (precision medicine) across a range of medical conditions. Source: https://lnkd.in/d2H76KZi https://lnkd.in/dxEV5KiY #abbvie #ADC #antibodydrugconjugates #bioconjugates #biopharma #biologics #biotech

Diagnostic Platform SOPHiA DDM™ Certified Under IVDR, Enhancing Genomic Analysis for Patient Care Across the European Union SOPHiA GENETICS has achieved a significant milestone with the certification of its SOPHiA DDM™ Platform under the European Union’s In Vitro Diagnostic Regulation. This certification reinforces the platform’s role in genomic analysis and enables its use across the European Union and other regions that recognize this regulation. With over 1.7 million genomic profiles analyzed globally, SOPHiA DDM™ is crucial for healthcare providers, particularly in oncology, offering precise, data-driven diagnostics. This achievement highlights SOPHiA GENETICS' commitment to maintaining the highest regulatory standards while continuing to innovate in healthcare technology, positioning the platform as a critical tool in the future of precision medicine. For more details please click the link! https://lnkd.in/d8pfjHPP #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical 

Collaboration on Flow Cytometry-Based Companion Diagnostics to Advance Personalized Health Care.  www.bd.com BD and Quest Diagnostics, a leader in diagnostic information services, announced a global collaboration agreement to develop, manufacture and commercialize flow cytometry-based companion diagnostics (CDx) intended to help select the best treatment for patients with cancer and other diseases. Together, BD and Quest will aim to provide the pharmaceutical industry with an end-to-end solution for CDx development. The joint offerings will range from exploratory panel development to the manufacturing and distribution of FDA-approved diagnostic kits. Using companion diagnostics to help select a first-line therapy for cancer patients can be critical to ensuring the best outcomes and cost savings. Today's companion diagnostic tests commonly involve technologies such as immunohistochemistry (IHC), fluorescence in situ hybridization (FISH), polymerase chain reaction (PCR), next-genera