Earlier this month at the Jefferies Global Healthcare Conference, Precision BioSciences, Inc. CFO Alex Kelly sat down with Precision AQ EVP & Managing Director, Hannah Deresiewicz, to discuss the company’s proprietary ARCUS® in vivo gene editing platform and pipeline with the aim of delivering lasting cures for patients with a range of genetic diseases where no adequate treatments exist. Discover the key features and differentiating characteristics that may enable ARCUS nucleases to drive durable therapeutic outcomes in chronic hepatitis B (PBGENE-HBV), m3243 primary mitochondrial myopathy (PBGENE-PMM), Duchenne muscular dystrophy (PBGENE-DMD) and beyond in Precision AQ's "Access to the Best and Brightest" series! Statements contained in this video by Precision Biosciences regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. A full description of risks and uncertainties can be found in Precision Biosciences filings with the Securities and Exchange Commission.
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Recruiter: Regulatory Affairs, Quality, Engineering & Commercial Top Talent in the Medical Device, IVD, Pharma and C> Industry
As #CGT continues to grow with significant investment and research, the FDA has created listening meetings for patients of rare diseases and their caregivers to allow them to provide feedback on issues related to approved cell & gene therapy products to support evaluations of short-term and long-term risks, improve industry decision making, and considerations for post-market studies. With the CRISPR Therapeutics approval for CASGEVY, there are now a total of 19 products on the market for 27 indications. With over 1500 clinical trials in progress last Feb and continued investment and focus, that number is expected to expand significantly in the next few years. The FDA continues to take proactive measures to improve communication, capture additional data, and Improve access and safety. Watching the industry grow while bringing hope and benefit to diseases without previous solutions will be exciting while providing incredible opportunities for career growth. https://lnkd.in/eYv7A_xe #collaboration #biotechnology #riskmanagement #patientadvocate #patientoutcomes #postmarketsurveillance #fda
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As part of a recent roundtable discussion, Pharma’s Almanac asked industry leaders what the most significant regulatory decisions on the horizon were. The one that doesn’t happen, quipped Janet Johnson (pictured), the Vice President of Global Regulatory Affairs and Labeling at Terumo Blood and Cell Technologies. “The FDA has long predicted it will approve between 10-20 cell and gene therapies (CGTs) a year by 2025,” she said. “But it will have approved no more than seven by the end of 2023, with at least two decisions coming in after their initial deadlines.” The impact? Sluggish momentum in rolling out critical #CurativeTherapies. Read her commentary and learn what other industry leaders say will be essential regulatory decisions in 2024 and beyond. Check it out now: https://bit.ly/3TxXm73 #FDA #CellTherapies #Healthcare
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Good exchange yesterday evening in Basel with an enthusiastic group of industry executives. The question: How to close the commercial viability gap in cell and gene therapies? Growth in CGT continues to significantly outpace the pharma market and there will be six blockbuster CGTs in 2028. However, to unlock the full potential, we must 1) become better at identifying the #patients who can benefit, 2) define effective market access and reimbursement strategies to ensure #accessibility, and 3) optimize manufacturing - and start doing so earlier in development. Dr. Oliver Eitelwein, Andreas Eggert, Dr. Clemens Freytag, Allegra Hohn-Honari, Lea Schaad
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In this ‘Patti’s People’ episode, Patti Peeples, PhD of the The Peeples Collaborative speaks with Walter Toro Jimenez, MD, PhD, Executive Director, Global HEOR/RWE, Novartis Gene Therapies. Deep diving into patient involvement in the #HTA process. Watch at The Evidence Base® #heor #healtheconomics #outcomesresearch #rwe #rwd #realworldevidence #realworlddata #marketaccess #pharma #biopharma #medicalaffairs #healthtechnologyassessment #healthdata #healthequity #healthpolicy #patientengagement
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Clinical research
There are more than 500 gene therapies in the clinical research pipeline, which is driving demand for lab capacity that's dedicated to specialized testing and services to help advance these therapies through the approval and commercialization process. To help address this need, we held a ribbon-cutting ceremony at our Middleton, Wisconsin site this week to celebrate the opening of new lab space that provides additional analytical testing capacity for our biopharma partners who are developing novel therapies for patients who need them most. Thank you to Wisconsin Governor Tony Evers and other local officials who were able to join us at today’s event. https://lnkd.in/gWBbKV6X
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WEBINAR 💻: Unlocking the Future of Pharmacy: Key topics and trends for the year ahead! Please join Strategic Benefits of Cincinnati and MedOne Pharmacy Benefit Solutions for an educational presentation on the state of pharmacy and what you can expect in the year ahead. In this 1-hour webinar, Dr. Nate Harold, VP of Clinical Services at MedOne, will discuss trending topics and provide stats, facts, and tips to help with understanding and interpreting the complex pharmacy space. Some of the discussion topics include: - Trending weight loss medications and new-to-market biosimilars - Inspection into transparency and legislation reform - Gene therapies & the impact on future generations - The movement on migraines - And more…. There is constant movement and new topics and trends taking place in pharmacy. As we approach the end of 2023 and look towards 2024, we hope this webinar proves valuable for you. Please register: https://lnkd.in/gcDAChnX #employeebenefits #pharmacybenefits
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Elevate your understanding of Cell and Gene Therapies (CGTs) through our exclusive expert roundtable summary. https://ow.ly/aTzQ50Q9TvX CGTs, such as CAR T-cells and autologous and allogeneic therapies, continue revolutionizing how we treat serious and rare diseases. However, the clinical trial landscape still proves to be complex. In this expert roundtable, panelists explore the key strategies CGT companies use to solve operational challenges in lifecycle management in expanding markets. It features insights from the following: • Matt Britz, Chief Operating Officer, AffyImmune Therapeutics • Mantej (Nimi) Chhina, Head of Global R&D and Regulatory Policy, BioMarin Pharmaceutical Inc. •Tamie Joeckel, Global Business Lead, Cell and Gene Therapy Centre of Excellence, ICON plc • Kilian Kelly, CEO, Managing Director, Cynata Therapeutics • Greg Kunst, CEO, Aurion Biotech • Curran Simpson, Chief Operating Officer, REGENXBIO Inc. • Krishnan Viswanadhan, Pharm.D, President, Chief Operating Officer, Be Biopharma Download the free summary today! #ClinicalTrials #CGTs #CitelineCommercial
Roundtable summary: Lifecycle Management for CGTs: Solving Operational Challenges in an Expanding Market
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Definitely worth a read if you value some insight on Cell and Gene Therapy clinical trials. I mean, what a panel of experts!
Elevate your understanding of Cell and Gene Therapies (CGTs) through our exclusive expert roundtable summary. https://ow.ly/aTzQ50Q9TvX CGTs, such as CAR T-cells and autologous and allogeneic therapies, continue revolutionizing how we treat serious and rare diseases. However, the clinical trial landscape still proves to be complex. In this expert roundtable, panelists explore the key strategies CGT companies use to solve operational challenges in lifecycle management in expanding markets. It features insights from the following: • Matt Britz, Chief Operating Officer, AffyImmune Therapeutics • Mantej (Nimi) Chhina, Head of Global R&D and Regulatory Policy, BioMarin Pharmaceutical Inc. •Tamie Joeckel, Global Business Lead, Cell and Gene Therapy Centre of Excellence, ICON plc • Kilian Kelly, CEO, Managing Director, Cynata Therapeutics • Greg Kunst, CEO, Aurion Biotech • Curran Simpson, Chief Operating Officer, REGENXBIO Inc. • Krishnan Viswanadhan, Pharm.D, President, Chief Operating Officer, Be Biopharma Download the free summary today! #ClinicalTrials #CGTs #CitelineCommercial
Roundtable summary: Lifecycle Management for CGTs: Solving Operational Challenges in an Expanding Market
discover.pharmaignite.com
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FDA Keynote Speaker in the Spotlight, CAR-T Industry Leaders Gather for Discussion-Join the 2024 GenScript Biotech Global Forum for a New Journey in Cell and Gene Therapy: NANJING, China, Dec. 24, 2023 /PRNewswire/ -- The innovative development of biopharmaceuticals has ushered in a new era for disease treatment, bringing unprecedented hope to countless patients. However, the transition from basic research to clinical commercialization presents formidable...
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Join us on June 25th in Woburn, MA for an engaging session at the 𝐂𝐞𝐥𝐥 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐌𝐚𝐧𝐮𝐟𝐚𝐜𝐭𝐮𝐫𝐢𝐧𝐠 𝐗𝐜𝐡𝐚𝐧𝐠𝐞 with our VP of Commercial, Robert Margolin! He'll be leading a roundtable discussion focusing on the Manufacturing Challenges with Gene-Editing Technologies for iPSC Therapeutics. Don't miss out on this valuable opportunity to: · Explore the manufacturing implications of the recent FDA guidelines from January 2024 on “Human Gene Therapy Products Incorporating Human Genome Editing”. · Understand the nuances in manufacturing requirements for gene-editing of master cell banks versus drug substances/drug products. · Discover strategies to manufacture gene-editing technologies that meet appropriate quality attributes. See you there! #CellTherapy #ManufacturingXchange #iPSCTherapeutic
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