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Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.
While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way.
In a Wednesday statement, the biotechnology company said it would use the new funds to advance into clinicaldevelopment an experimental treatment targeting what are known as fibroblast activation proteins. in 2013 — the field has taken off since the success of Novartis’ prostate cancer treatment Pluvicto.
Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies. My focus has always been on advancing novel medicines from research to clinicaldevelopment. TILs were specifically developed to target solid tumour cancers, Bock explained.
There are over 7,000 rare diseases affecting more than 30 million people in the United States, and despite the FDA's approval of over 600 treatments for rare diseases since signing the Orphan Drug Act into law in 1983, most rare diseases still do not have a treatment.
ML is also used to identify predictors of response to a therapy or risk for adverse events (AEs) that can be used to inform value-based contracting or treatment strategies intended to minimize “wasted” use of a drug. Ready to learn more about convergence of real-world data and technology for clinical trials?
BIIB129 (25) demonstrated efficacy in disease-relevant preclinical in vivo models of B cell proliferation in the CNS, exhibits a favorable safety profile suitable for clinicaldevelopment as an immunomodulating therapy for MS, and has a low projected total human daily dose.
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. The founding of RadioMedix was born from a recognition of unmet needs in cancer treatment.
These results further demonstrate the utility of pH-dependent neuroprotective agents and GluN2B-selective NMDAR inhibitors as potential therapeutic strategies for the treatment of aSAH. Based on allometric scaling of both toxicological and efficacy data, the therapeutic margin in man should be at least 2.
Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinicaldevelopment, offering promising advancements in innovative cancer treatments.
This shift raises ethical questions around prioritizing supply for diabetes patients versus those seeking treatment for obesity or cosmetic weight loss. The balance is complex, as obesity carries its own serious health risks.
Herein, we report our structure-based drug design approach that led to the identification of 14, AZD4747, a clinicaldevelopment candidate for the treatment of KRASG12C-positive tumors, including the treatment of central nervous system (CNS) metastases.
More than 7,000 rare diseases – conditions that affect fewer than 1 in 2,000 people – have been identified, and1 in 17 people will develop a rare disease in their lifetime, with children disproportionately affected. Most of these conditions are genetic in origin and the majority have no effective treatment.
They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Unlearn’s digital twins are now in use in both early and late-stage clinical trials, with adoption continuing to grow. Integration is straightforward,” Herne notes.
For these patients, clinical onset means severe symptoms and a rapid decline in overall health. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. The Next Wave: Targeting B7H3 – a drug development tsunami in the making?
Like many aspects of life in the 21st century, this era of scientific advancement and increased partnership is largely driven by next-generation technologies that are streamlining workflows, breaking down silos and empowering scientists to extract the most prescient insights from their data throughout the drug development process.
Accelerating time to treatment. Don't miss this opportunity to gain valuable insights and explore the future of clinicaldevelopment. png Listing Introduction Join us for an engaging webinar that explores how innovative automation capabilities are transforming the landscape of clinicaldevelopment in the life sciences industry.
Celltrion has constructed a COVID-19 antibody portfolio and clinicaldevelopment platform to help combat the spread of the virus mutations CT-P59 demonstrated neutralising capability against key emerging mutations, including the UK variant in addition to six variant genome mutations of SARS-CoV-2.
Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. As a provider of end-to-end clinicaldevelopment solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis.
Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD. These data were viewed as both mechanistically and clinically meaningful , laying the groundwork for future mid- and late-stage development.
Instead of the standard approach where studies are designed to address a primary focus, a parallel study method — a format that incorporates multiple endpoints and objectives, modifications, treatment arms and dose selections into a single protocol — is being rapidly adopted.
The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.
The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. Throughout the 26-week study period, the investigational therapy was well-tolerated.
Recent Applications of Ring-Closing Metathesis in Drug Discovery and Development A selection of representative macrocycles in drug discovery where ring-closing metathesis has been applied to their synthesis are shown below, highlighting chemically diverse macrocycles from programs that have reached clinicaldevelopment.
As the biotech sector races to improve the tolerability of these revolutionary treatments, Poolbeg Pharma has a novel solution: an oral therapy, POLB 001, to block the development of CRS, by limiting inflammation without affecting the anti-cancer immune responses that are vital for effective immunotherapies.
To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinicaldevelopment.
This leadership transition arrives at a pivotal moment in Alnylam’s evolution, as the company builds upon the recent commercial success of AMVUTTRA® (vutrisiran) —a landmark treatment for transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
However, the FDA’s recent decision to remove the Risk Evaluation and Mitigation Strategy (REMS) requirements for certain approved Chimeric Antigen Receptor T-cell (CAR T) therapies could dramatically shift these numbers, making life-saving treatments more accessible to those in need.
This significant financial infusion is designed to fuel Revolution Medicines’ ambitious clinicaldevelopment and commercialization plans, with a particular focus on its groundbreaking RAS(ON) inhibitor programs for patients with RAS-addicted cancers.
Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies.
The encouraging data builds on the foundation established by earlier studies and further strengthens the clinical case for MR-141 as a potential non-invasive, once-daily ophthalmic solution for millions affected by the age-related decline in near vision. According to the company, MR-141 met its primary endpoint with statistical significance.
Here, we examined the preclinical and clinical effects of the first-generation FXR agonist, Px-102 (4-[2-[2-chloro-4-[[5-cyclopropyl-3-(2,6-dichlorophenyl)-4-isoxazolyl]methoxy]phenyl]cyclopropyl] benzoic acid), to enable the selection of an analog, cilofexor, with unique properties that reduced side effects yet maintained efficacy.
Metastatic castration-resistant prostate cancer represents the most advanced and lethal form of the disease, defined by progression despite androgen deprivation therapy and subsequent ARPI treatment. This has created an urgent need for more effective and tolerable treatments for this population.
a subsidiary of the Roche Group, NXT007 represents a next-generation therapeutic approach that builds upon the foundation established by Hemlibra® (emicizumab), Roche’s first-in-class bispecific antibody approved for prophylactic treatment of hemophilia A. Engineered by Chugai Pharmaceutical Co.,
The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc.
Developingtreatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. These conditions pose substantial challenges for patients, families and health care systems.
Our respective expertise — their extensive experience in clinicaldevelopment and navigating the Chinese market , alongside our own strength in biologic innovation and growing pipeline of novel oncology treatments — forms a powerful synergy.
Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. Aflibercept is a key treatment in ophthalmology.
The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.
By integrating AI into the manufacturing process, the developer achieved higher consistency across batches, improved overall product quality, and reduced time-to-market – all critical factors in ensuring patients receive timely and effective treatment.
.” Beyond that, the second MSLN Radio-DARPin is advancing and Switch-DARPin development is progressing further. “We will communicate specific next steps, including clinicaldevelopment plans… in due time,” says Croset. ” And if AACR 2025 was any indication, those treatments are closer than we think.
A Global R&D Footprint Backed by Clinical Execution BeOne’s engine of innovation is supported by a vast and diversified global clinicaldevelopment network. To date, the company has conducted more than 170 clinical trials across 40 countries, enrolling over 25,000 patients. Global Head of R&D. “We
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