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Artificial intelligence (AI) has revolutionised many industries, yet its adoption in pharmaceutical drug development has been notably slower. However, recent breakthroughs in AI, such as predictive modelling, clinical trial optimisation, and personalised medicine, have demonstrated its potential.
Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
ABSTRACT Fused pyrimidine scaffolds serve as a flexible and versatile foundational heterocycle in the field of pharmaceutical development. The presence of thienopyrimidine derivatives in several FDA-approved drugs and clinical trial candidates underscores their therapeutic potential and safety profile.
At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. Why targeted radiopharmaceuticals? The SPICA Center was built to address this gap.”
As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. Accelerate your drug development and clinical trial goals and benefit from our 360° CDMO and CRO solutions and expertise. billion in 2035.
A personal journey to Alltrna Michelle Werner’s career has spanned over 20 years in the pharmaceutical industry, where she developed her expertise in oncology drug development at leading companies such as Bristol Myers Squibb, AstraZeneca, and Novartis.
Longer follow-up results from a mid-stage trial indicate the company’s treatment for a genetic cause of dwarfism may be competitive to an approved therapy from BioMarin Pharmaceutical.
The investigational ADC is built using Daiichi Sankyo’s proprietary DXd technology and represents one of the newest generation of targeted cancer therapies. Building on Early Clinical Success The Phase 3 IDeate-Prostate01 trial is based on encouraging results from the earlier IDeate-PanTumor01 Phase 1/2 study.
Knowing a patients subtype or phenotype can be used to guide obesity treatments of all kinds, including drug therapy, devices, bariatric surgery and even diet and lifestyle interventions, Bagnall explains. Without understanding these differences, treatment becomes costly and a game of trial and error, Bagnall points out.
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Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.
Deal Structuring Deal structuring in pharmaceutical M&A requires a balance of financial, legal, and strategic considerations. Intellectual Property Valuation Valuing intellectual property (IP) assets is a critical component of pharmaceutical M&A, as these assets often represent a significant portion of a company's value.
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Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
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2] History The efficacy and safety of fitusiran were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male participants with either hemophilia A or hemophilia B. [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1] Fitusiran 1711.0g/mol,
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This can present challenges when attempting to recruit an enriched patient population for clinical trials. Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? We were able to license a molecule from a multi-national pharmaceutical company.
billion to bring a new therapy to market. In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Thus, these drugs best complement, rather than replace, healthy lifestyle interventions.
For pharmaceutical innovators and drug developers working to bring oncology therapies to market, patients are the “why” behind it all. For many patients, involvement in oncology clinical trials represents a last hope for an effective therapy. Many of these patients’ conditions are disabling.
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1] History Sunvozertinib is being developed by Dizal Pharmaceutical. [5] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] 7] Efficacy was evaluated in WU-KONG1B (NCT03974022), a multinational, open-label, dose randomization trial. [1]
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects. 3D rendering of Antibody Drug Conjugate Molecules.
JCR Pharmaceuticals Celebrates 50 Years of Innovation with Global Website Launch That Reflects Its Expanding Reach and Human-Centric Mission As JCR Pharmaceuticals commemorates its 50th anniversary, the global specialty biopharmaceutical company is marking this major milestone with the launch of a newly designed global website.
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Biogen Launches Global Phase 3 Pediatric Trial of Omaveloxolone for Friedreich Ataxia Biogen has officially launched its BRAVE study a pivotal, global Phase 3 clinical trial aimed at evaluating omaveloxolone in pediatric patients with Friedreich ataxia (FA). Head of the Neuromuscular Development Unit at Biogen.
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The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C.,
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By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Previously, organizations had limited influence on the direction of basic research, clinical trial designs, and data ownership.
1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] The efficacy of Journavx was evaluated in two randomized, double-blind, placebo- and active-controlled trials of acute surgical pain, one following abdominoplasty and the other following bunionectomy.
As a proof of concept, a drug designed to target a newly discovered biological node is showing efficacy in treating rare genetic diseases in the kidney, the eye, and the brain and is now making its way to clinical trials in collaboration with a pharmaceutical partner. You can learn more about how clinical trials work here.
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