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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.
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.
The journey of cell and gene therapies from preclinical discovery to clinical trials is complex and challenging, impacting every team member involved, from researchers in the lab to patients receiving treatment. IRT systems dynamically generate and track expiration dates, ensuring treatments reach patients within their viable windows.
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.
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.
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.
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.
Antibody therapies have transformed cancer treatment, yet their limits are becoming increasingly clear – particularly when tumours evolve, evade immune detection or develop resistance to existing drugs. The presence of p95HER2 – a shortened version of the receptor that still drives tumour growth – makes treatment even more difficult.
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.
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.
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.
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?
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 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.
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.
Immuno-oncology has transformed cancer treatment, but for many patients, tumours remain resistant to even the most advanced immune checkpoint inhibitors. DT-7012 retains its depletion activity even in high CCL1-concentrated environments – an essential property not shared by most competitor antibodies currently in clinicaldevelopment.”
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.,
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).
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.
.” 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.
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.
This partnership brings together Altasciences’ extensive expertise in preclinical research and early clinicaldevelopment with VoxCell’s groundbreaking tissue engineering technology, promising to deliver a more human-relevant, predictive, and efficient drug development paradigm. Source Link
(NASDAQ: ONC; HKEX: 06160; SSE: 688235), an emerging global player in oncology therapeutics, has reached a critical milestone in its mission to advance novel cancer treatments. Despite advances in treatment, including the advent of BTK inhibitors like ibrutinib, patients with WM often face relapse or treatment resistance over time.
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.
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.
Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Nature Medicine, 28, 1656–1661 (2022) [link]
This study explored the effects of beginning treatment with lower starting doses of MariTide to improve tolerability while maintaining clinical benefit. Both data sets now provide foundational insight into the design and direction of Amgen’s upcoming Phase 3 MARITIME clinicaldevelopment program.
The agreement centers around the investigational RNA interference (RNAi) therapy, plozasiran, which targets apolipoprotein C-III (APOC3) and is under development for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). President and CEO of Arrowhead. “We
In contrast to a conventional pharma partnership, this large scale and flexible funding agreement enables Revolution Medicines to retain control of the clinicaldevelopment of daraxonrasib, as well as the ability to capture significant value creation that would result from the successful clinicaldevelopment and commercialization of its pipeline.”
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.
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.
The study represents a major step toward addressing the unmet medical needs of individuals affected by this rare and severe neurodevelopmental disorder, which currently has no approved disease-modifying treatments. Those assigned to the active treatment arm will receive quarterly doses of ION582 at either 40 mg or 80 mg.
The approval authorizes AUCATZYL® for the treatment of adult patients aged 26 and older diagnosed with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL) in all 27 European Union member states. Food and Drug Administration (FDA). Source Link
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.
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.
Together, these data reflect a significant step forward in the treatment of hematologic malignancies — a group of aggressive, complex disorders for which many patients still face limited treatment options and poor outcomes — and demonstrate the potential for these novel mechanisms of action to make a profound difference in patient care.
(NASDAQ: BIIB) presented comprehensive new findings from their Phase 3 PHOENYCS GO clinical trial evaluating dapirolizumab pegol (DZP) at the 2025 Annual Congress of the European Alliance of Associations for Rheumatology (EULAR) in Barcelona, Spain. Treatment-Emergent Adverse Events (TEAEs) : Occurred in 82.6% in the SOC group.
These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. Rilzabrutinib is currently under regulatory review in major markets including the United States, European Union, and China for the treatment of ITP.
Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.
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.
Before a drug can enter human trials, researchers must submit an IND to regulatory agencies, providing preclinical toxicology data, proposed clinical trial designs, and manufacturing details. This application is the foundation for obtaining approval to proceed with Phase I human trials.
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