BioMed Consulting & Training’s Post

🚨 uniQure releases updates about AMT-130’s approval pathway. The pharmaceutical company has made key progress in its pursuit of Accelerated Approval for AMT-130, a gene therapy for Huntington’s Disease, by reaching alignment with the FDA on key elements. 🎯 Highlights: - Agreement on Relevant Data: FDA has agreed that data from the Phase I/II studies, compared to a natural history external control, may serve for an upcoming regulatory submission known as Biologics License Application (BLA). - Clinical Measures Validated: The FDA recognizes the composite Unified Huntington’s Disease Rating Scale (cUHDRS) as a valid intermediate clinical endpoint for the Accelerated Approval Process. - Supporting Evidence: Reductions in neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) may serve as supportive evidence for therapeutic claims. - Regenerative Medicine Advanced Therapy (RMAT) Status: Confirmed in May 2024, highlighting AMT-130’s potential to meet critical unmet needs in HD treatment. 🔗 Learn more and read the full release here ➡️ https://lnkd.in/eMiznUxe

Today’s Conference Spotlight features the Office of Research Regulatory Affairs (ORRA) here at Nationwide Children's Hospital. Kevin Bosse, Ph.D., RAC-US, CABP(H), Director, and Elizabeth George, MS, Regulatory Specialist, will be presenting at the ISCT, International Society for Cell & Gene Therapy Annual Meeting in Vancouver next week! Elizabeth is presenting an abstract titled, “Development of a Structured Review Service for Non-GLPS Study Data Intended to Support Regulatory Submissions at an Academic Institution” on Thursday, May 30th. Kevin is chairing the “Late Breaking Abstracts” session on Thursday, May 30th at 5:00 pm, and is also presenting a roundtable session titled “Viral Versus Non-Viral: What’s the Better Vector?” on Friday, May 31st at 5:00 pm, as well as the “Technologists Training Course” all day Saturday, June 1st. For more on the ISCT program, click here: https://bit.ly/4dRckwI The ORRA advises the development and regulatory strategy of novel drugs, biologics, and devices, with a primary focus on gene and cellular therapies. They provide regulatory affairs support and consulting for numerous academic, private, and publicly traded programs working towards the clinical development of AAV-based gene and cell-based therapies. Since 2012, the office has launched over twenty investigator-initiated first-in-human clinical biologic programs and has obtained fast-track, breakthrough, orphan, and rare pediatric disease designations for many submissions. The OTC would not have produced such astonishing levels of success in licensing without the support and results generated from our partnership with the ORRA. Learn more about the ORRA: https://bit.ly/3KbeObA #InnovateWithUS #ISCT24 #GeneTherapy #RegulatoryAffairs

🚨 FDA OTP Virtual Town Hall: Best Practices for Regulatory Interactions 🚨 The upcoming OTP Town Hall on December 12, 2024, provides an excellent opportunity for developers of cell and gene therapies to gain insights into best practices for interacting with the FDA's Office of Therapeutic Products (OTP). If you're involved in the development of advanced therapies like cell and gene therapies, mark your calendars for the upcoming event on December 12, 2024 from 11:00 a.m. to 12:30 p.m. ET! This town hall will provide invaluable insights for developers navigating the IND/BLA pathway. Experts from the Office of Review Management & Regulatory Review (ORMRR) will share best practices and guidance on formal meetings with OTP and discuss how to optimize regulatory interactions throughout the product development lifecycle. 🎯 What to Expect: 1. Key advice on the IND/BLA process for advanced therapies. 2. Guidance on scheduling, preparing, and documenting formal meetings with OTP. 3. Expert insights from ORMRR staff to ensure efficient interactions and regulatory success. 🔍 Important Notes: Questions can be submitted during registration (by November 13). The focus is on general regulatory practices, not specific product inquiries. Let’s collaborate to drive the future of advanced therapies forward! C&R Research, Inc and ABC Bioscience (https://lnkd.in/gufTD7Dq) offer deep expertise and practical insights through our integrative and pragmatic consultation services. Our dedicated team, equipped with professional expertise, is committed to delivering solutions that meet global standards and cater to your specific needs for advanced therapy. 👉 Register Today: Please secure your seat. #FDA #Biotech #GeneTherapy #CellTherapy #RegulatoryAffairs #FDARegulations #AdvancedTherapies #Pharmaceuticals #DrugDevelopment #ClinicalTrials #RegulatoryScience #CNRResearch

In 2023, the number of Small-molecule New Drug Application (NDA) approvals slightly surpassed the number of Biologics License Application approvals. However, this margin was narrow, with biologics accounting for 48% of the new approvals. This percentage was only exceeded once before, in 2022. This trend underscores the ongoing growth of new biologic modalities and highlights the robust performance of the Center for Biologics Evaluation and Research (CBER), an FDA division responsible for regulating vaccines, cell and gene therapies, and blood products. #smallmolecules #biologics #bitoech

🔊 Upcoming Virtual Town Hall with the FDA’s Office of Therapeutic Products (OTP) 🔊 This Thursday, 12/12, the FDA’s Center for Biologics Evaluation and Research (CBER) will host an insightful (virtual) town hall focused on best practices for interacting with the Office of Therapeutic Products (OTP). This event is a MUST-ATTEND for those in the advanced therapy development space, including cell and gene therapies. 🔍 Key Details 🔍 Focus: Best practices for regulatory interactions with OTP, specifically for developers of advanced therapies under the IND/BLA pathway. Audience: This session is targeted at developers of biological products and regulatory affairs professionals, offering a unique opportunity to hear directly from experts from OTP's Office of Review Management & Regulatory Review (ORMRR). Event Time: 11:00 a.m. to 12:30 p.m. ET Platform: The event will be hosted via Zoom (registration required). 👩🔬 Why Attend? ORMRR will be present to answer questions about the IND/BLA submission process, providing invaluable guidance on how to efficiently engage with OTP at various stages of the product development lifecycle. Whether you’re preparing for formal meetings or seeking advice on regulatory practices, this is your chance to gain expert insights into navigating the regulatory environment. 💬 Submit Your Questions! Questions can be submitted during registration and live during the event. Please note, however, that the FDA will not address questions about specific investigational products, biologic applications, or draft guidance documents. 🔗 Register Today: https://lnkd.in/gM3rcxtZ Don't miss this opportunity to enhance your understanding of the regulatory landscape for advanced therapies and connect with FDA experts! #FDA #RegulatoryAffairs #AdvancedTherapies #CellAndGeneTherapies #IND #BLA #TherapeuticProducts #Biologics #ClinicalResearch #Biotech

Get familiarized with the new designations being ascribed to the drugs now and then only at PharmaShots!  PharmaShots brings every month a Designation report, highlighting the designations allotted to drugs in various geographies.  This month, we have covered designations from the US, China, Canada and EU regions.  For a curated report on the designation of your need, reach out to us at connect@pharmashots.com  Stay Tuned for more updates!  Ascendis Pharma Longboard Pharmaceuticals Lisata Therapeutics, Inc. Aisa Pharma, Inc. Actuate Therapeutics, Inc. Agios Pharmaceuticals Atsena Therapeutics uniQure Amarex Clinical Research, LLC, an NSF company Gibson Oncology, LLC Astria Therapeutics, Inc. Abdera Therapeutics CAMP4 Therapeutics YolTech Therapeutics Somite Therapeutics EydisBio, Inc. Skyline Therapeutics SeaBeLife Biotech SUN PHARMA Moebius Medical Ltd. Priovant Therapeutics Innovent Biologics Glycomine Inc. ImCheck Therapeutics Amplia Therapeutics Limited PTC Therapeutics, Inc. Caribou Biosciences Synthekine Elevation Oncology Simcere MSD (Shanghai) Pharmaceuticals Co Ltd Arrowhead Pharmaceuticals BridgeBio Neuraptive Therapeutics, Inc. GSK BioCity Biopharma Biogen Cartesian Therapeutics NS Pharma, Inc.   #healthcanada #nmpa #usfda #ema #us #eu #orphandrugdesignation #fasttrackdesignation #breakthroughtherapydesignation #breakthroughdevicedesignation #regenerativemedicineadvancedtherapydesignation #rarepediatricdiseasedesignation #priorityreview #designations #regulatory 

𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐚𝐧𝐝 𝐏𝐡𝐚𝐫𝐦𝐚 𝐔𝐩𝐝𝐚𝐭𝐞𝐬: September 24 - 25, 2024 • Emergent BioSolutions raking in $400M in mpox, smallpox vaccine orders • Vertex Pharmaceuticals’s Casgevy approved in Canada • Amgen notches mixed wins in atopic dermatitis and myasthenia graves • Genespire’s $52M Series B, Eurofins to build new $64M biologics site • Asklepios BioPharmaceutical, Inc. (AskBio)’s gene therapy collab with Belief BioMed • Semaglutide continues wonder-drug tirade with studies pointing to treating a chronic skin condition and opioid use disorder • 2seventy bio & Bristol Myers Squibb shutter Abecma myeloma trial • Johnson & Johnson allegedly slashing cardiovascular and metabolic disease divisions • Pfizer’s global recall of Oxbryta sickle cell med due to patient deaths More in today's TLDR Biotech newsletter: https://lnkd.in/guh7SMhn #biotech #pharma #pharmanews #biotechnews #tldrbiotech

I am very excited to share my latest article 2024 New Drug Modalities Report, co-authored by me and my BCG colleagues Brian Bush, Mike Brochu, Gian King, PharmD. It's been an exciting year since our 2023 report. New modalities represent $168 billion in projected pipeline value in 2024, up 14% from 2024. ADCs and GLP-1s have had breakout success. Gene therapies for rare diseases, microbiomes, and oncolytic virus saw their first US approvals in the past year. CAR-T and mRNA continue to mature and broaden their applications beyond the initial therapeutics. Large Biopharmas have spent nearly $200B since 2022 in deal making in new modalities. The next few years are likely to be exceptionally competitive for new modalities. Not all new modalities can be winners. The success of each new modality will hinge on its unique value proposition and its capacity to enhance the standard of care. As you review the insights, please reach out with any feedback and perspective. Always excited to bounce ideas with fellow 'new modalities enthusiasts'! https://lnkd.in/g6rYim6s

Eder Therapeutics and xcube.bio have announced a strategic cooperation to facilitate the market entry and commercialization of biotech assets in Canada and Europe. The two companies will coordinate their offerings to biopharma innovators in their respective regions and leverage the numerous synergies that exist between the healthcare systems of Europe and Canada. The collaboration will specifically focus on product profiling, regulatory processes, Health Technology Assessment (HTA), and medical communication, and commercialization - ultimately benefiting patients and biopharma partners.   "Eder Therapeutics is thrilled to combine its unique depth in the Canadian market with the breadth of a European platform like xcube.bio. This partnership will enhance access to innovative treatments for Canadian patients, which is our ultimate goal," said Jared Rhines, CEO of Eder Therapeutics.   Pierre-Henri Belin, CEO of xcube.bio, stated, "xcube.bio, a European market-entry specialist, has found a valued like-minded partner in Eder Therapeutics. We look forward to expanding this cooperation to accelerate patient access and provide added value to our biopharma partners."   An initial phase of expert cooperation will begin with Eder Therapeutics' investigational gene therapy for Lipoprotein Lipase Deficiency. #biopharma #partnering #commercialization