Fulcrum Therapeutics’ Post

https://buff.ly/3VsgMLb 𝐅𝐃𝐀'𝐬 𝐎𝐩𝐞𝐫𝐚𝐭𝐢𝐨𝐧 𝐖𝐚𝐫𝐩 𝐒𝐩𝐞𝐞𝐝 2.0: 𝐀𝐜𝐜𝐞𝐥𝐞𝐫𝐚𝐭𝐢𝐧𝐠 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 🚀🔬 💠 The FDA is taking a page from Operation Warp Speed with its new pilot program, START, designed to accelerate the development of rare disease therapies. This initiative brings together the FDA and seven leading biotech companies to streamline the approval process and bring much-needed treatments to patients faster. 💠 By fostering close collaboration between regulators and drug developers, START aims to optimize clinical trial design, refine patient selection, and enhance communication throughout the development process. 💠 Companies like Denali Therapeutics, Larimar Therapeutics, Grace Science, Neurogene, and Myrtelle are already participating in the program, working on groundbreaking therapies for a variety of rare conditions. 💠 I see this as a much-needed step in the right direction to help sponsors deal with the difficulties in drug development in this area as a result of small patient populations, lack of established endpoints, and the complexities of patient recruitment and retention. #FDA #RareDisease #Biotech #Innovation #DrugDevelopment #orphandrugs

With the approval of OGSIVEO™ (nirogacestat), patients must contact their healthcare provider to see if the drug is right for them. SpringWorks Therapeutics, the pharmaceutical company that developed the drug, has several resources available to desmoid tumor patients. ✔️ SpringWorks Therapeutics has a patient support services program called SpringWorks CareConnections™ for people prescribed OGSIVEO™ (nirogacestat). Physicians and patients can contact 1-844-CARES-55 (1-844-227-3755) or visit springworkstxcares.com. This is a free, personalized patient support program to help navigate insurance coverage, answer questions about treatment, and help patients start and stay on track with OGSIVEO™ (nirogacestat). ✔️ Official website: Ogsiveo.com ✔️ Official Facebook page: facebook.com/OGSIVEO ✔️ Official Facebook group for health care providers: facebook.com/ogsiveoHCP #Desmoid #DesmoidTumors #DesmoidTumor #fibromatosis #aggressivefibromatosis #DTRF #DesmoidTumorResearchFoundation #Desmoidian #desmoidresearch #FAP #familialadenomatouspolyposis #OrphanDisease #raredisease #oncology #research #hope #OGSIVEO #nirogacestat #expandedaccess

🎥 Welcome to Amador's latest video on #ADC drug clinical development solutions! In this episode, Dr. Meina Liang, CTO of Amador, provides an insightful explanation on the definition and classification of #biomarkers, as well as effective biomarker strategies for ADC drug development. Join us to deepen your understanding of how biomarkers play a crucial role in advancing ADC therapeutics. Don't miss this opportunity to learn from an industry expert. Watch the video now and share your thoughts with us!👇 #ADCDrugs #PKPD #Pharmacology #ClinicalResearch #AmadorBiosciences

The countdown for the Covalent Drug Discovery Summit is on! Bringing 80 covalency experts to Boston for the very first time, this exciting forum is uniting industry veterans, pioneering biotechs, and academic thought leaders to hear the latest insights on novel covalent drug discovery programs, hit-to-lead acceleration and triaging, novel chemistry targeting non-cysteine amino acids, and so much more. Download the program here: https://ter.li/mnhm14 Confirmed companies in the room include: Abbvie, Amgen, AstraZeneca, Blueprint Medicines, Covant Therapeutics, FL94, Frontier Medicines, Hyku Biosciences, Light Horse Therapeutics, Matchpoint Therapeutics, MD Anderson Cancer Center, Novartis, Nurix Therapeutics, Pfizer, Pledge Therapeutics, Revolution Medicines, Scorpion Therapeutics, Totus Medicines and more! There's still time to secure your place and join your fellow VPs, Directors and Heads of #DiscoverySciences, #MedicinalChemistry, #BiologicalChemistry and more as they unite to advance covalent medicines and expand the boundaries of drug discovery for patients in need. Register your pass before it's too late: https://ter.li/gr4rq8 #covalent #covalency #drugdiscovery #smallmolecules #leadoptimization #hitscreening

Are you attending the Complement-Based Drug Development Summit in Boston? Tomorrow, on Tuesday, 9/12, at 12:00 (EDT), don't miss Dr. Michael Schwenkert, Chief Product & Technology Officer (https://hubs.ly/Q020Nhvy0) as he presents "The Complement System in Drug Development – step-by-step towards the Bigger Picture." His presentation will highlight: • Quantitative biomarker evaluation • Functional anaphylatoxin detection • Assessment of pathway and convertase activity Interested in learning more about us? Visit our booth to meet our team of experts, ready to answer your questions and provide valuable insights into the challenges of developing next-generation therapeutics. #svarlifescience #answersinlifescience #expertpartner #partnerwithus #complementterapeuthics #complement #complementsystem #complementinglifescience"

🔬 **Advancing Healthcare Innovation!** 🔬 Exciting news from Basilea Pharmaceutica! 🌟 They are diving into their second venture to strengthen their anti-infective pipeline by investing $2 million to secure Gravitas Therapeutics, Inc.’ clinical-stage antifungal drug. This pharmaceutical, once known as GR-2397, is tailored to combat invasive mold infections caused by the Aspergillus species. Invasive aspergillosis presents a significant risk, especially for individuals with weakened immune systems, such as those undergoing blood cancer treatment. Basilea's strategic investment underlines their dedication to addressing critical medical challenges, potentially saving lives in the process. #HealthcareInnovation #AntiInfectivePipeline #BasileaPharmaceutica #GravitasTherapeutics #MedicalResearch 🦠🩺

🚀 Kicking off Day 2 of the 20th BioPharma Drug Discovery Nexus Conference with a bang! Join Mauro Mileni Founder and CEO, Abilita Therapeutics, Inc. as he unveils insights into 'Enabling the Discovery of Therapeutic Antibodies Targeting Challenging Membrane Proteins'. Don't miss out on this enlightening session! #BioPharma #DrugDiscovery #Innovation #Antibodies #Therapeutics #Membraneproteins #Epitopes #Conformational #Protiens #NexusConference #SanDiego #USconference #ScientificConference

|| Pharma Fusion Series 1.8 || 🌟 Vertex Pharmaceuticals's Mega $4.9B Deal with Alpine Immune Sciences, Inc. Signals Immunotherapy Surge 💼💥🚀 Vertex Pharmaceuticals has acquired Alpine Immune Sciences, Inc., for $4.9 billion. This strategic move aims to accelerate the development and potential commercialization of povetacicept, a promising treatment for IgA nephropathy and other serious autoimmune diseases. 💼💡 The acquisition enhances Vertex's capabilities with Alpine's expertise in protein engineering and immunotherapy, signaling a notable advancement in protein-based immunotherapies. This acquisition has the potential to significantly impact the future of immunotherapy and the treatment of serious autoimmune conditions, ultimately benefiting patients in need. 🌟🩺 #PharmaceuticalInnovation #VertexPharmaceuticals #AlpineImmuneSciences #IgANephropathy #AutoimmuneDisease #ImmunoTherapy #acquisition #merger #pharmanews #information #povetaciept #proteinengineering #niper #dopm #pharmamba #pharmaprofessionals #FiercePharma ##reuterseventspharma #Reuters

PHARMA M&A Newsletter - MERCK ACQUIRES CARAWAY THERAPEUTICS Merck's acquisition of Caraway Therapeutics represents a calculated expansion in the biopharmaceutical landscape, showcasing Merck's vision for future therapies. The agreement brings forward several key outcomes: 🧬 Neurodegenerative Disease Emphasis: Caraway Therapeutics brings a promising pipeline to Merck, notably featuring TRPML1 and TMEM175 modulators. These compounds are at the frontier of targeting lysosomal dysfunction, which is implicated in neurodegenerative diseases such as Parkinson's disease and ALS. 💡 Strategic Assets: TRPML1 and TMEM175 are integral in cellular homeostasis. By modulating these pathways, Caraway's approach could lead to transformative treatments, potentially slowing or reversing disease progression. 💰 Financial implications: The acquisition, which could be worth up to $610 million including milestone payments, represents a strategic integration of Caraway's innovative work in neurodegenerative diseases and genetically defined rare diseases, with the aim of developing much-needed disease-modifying therapies. Stay tuned by subscribing now. Don't miss out on the next issue for more insights! #Pharma #Roche #Caraway

Probability of success for cancer drugs between 2011-2020 by Biotechnology Innovation Organization, Informa and QLS Advisors. A total of 12,728 clinical and regulatory phase transitions were recorded and analyzed from 9,704 development programs over the last decade (2011–2020). The overall likelihood of approval from Phase I for all developmental candidates over 2011–2020 was 7.9%. Phase II development remains the largest hurdle in drug development, with just 28.9% of candidates achieving this critical phase transition. On average, it takes 10.5 years for a Phase I asset to progress to regulatory approval. Disease areas with above-average LOAs tend to have the shortest development timelines. #cancerresearch #clinicaltrial #fdaapproval https://lnkd.in/gaaRKj7