This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
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.
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.
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.
They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Solving old problems in new ways Herne is well acquainted with the operational inefficiencies that persist across clinicaldevelopment. 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.
With more than 40 clinical and commercial-stage assets in active development, BeOne Medicines is demonstrating a scale of ambition matched by few in the field. A Global R&D Footprint Backed by Clinical Execution BeOne’s engine of innovation is supported by a vast and diversified global clinicaldevelopment network.
DARPin therapeutics were a prominent theme at AACR 2025 , with Molecular Partners presenting a trio of preclinical programmes spanning radiopharmaceuticals and immune cell engagers. “My passion for science drew me to DARPin technology, and I was eager to contribute to the development of new DARPin therapies.”
The data were presented during the 2025 European Hematology Association (EHA) Congress (Abstract #S239). Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. The most frequently encountered form within this group is diffuse large B-cell lymphoma (DLBCL).
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.
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.
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.”
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 DeLLphi-304 study was reported at ASCO earlier this month.
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.,
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.
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. President and Chief Executive Officer of Intellia Therapeutics.
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.
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.
At this premier global meeting focused on thrombosis, hemostasis, and bleeding disorders, Sanofi will present compelling data across 18 scientific abstracts, including five oral presentations. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial. In the U.S.,
(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%
Additionally, advanced data mining capabilities enable the extraction of real-world evidence and patient stratification, providing deeper insights into patient populations and treatment outcomes. This presents three core conflicts: Opaque provenance: The data used to train the model is not transparent.
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. Engaging in early dialogue with regulators can help prevent costly delays and ensure clinical trials are structured to meet approval criteria.
Strategic Expansion with INZ-701 The centerpiece of this acquisition is INZ-701, an investigational enzyme replacement therapy currently in advanced clinicaldevelopment for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency.
A Significant Milestone for Zanzalintinib The STELLAR-303 trial represents the first pivotal phase 3 study to read out in the development program for zanzalintinib, a novel compound that has shown promise across various tumor types. These therapies offer only modest benefits and are often associated with challenging side effects.
The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.
Most Popular National Resilience, a well-funded manufacturing startup, to scale back operations Concentra to acquire Elevation in bid to eliminate another biotech ‘zombie’ Vaccine makers face fresh uncertainty after firing of CDC panel Biotech funding plummets as Trump policies unnerve investors: Jefferies Library resources Podcast Pioneering Treatments (..)
Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME. The recent integrin inhibition program that was being developed by Pliant has not yet demonstrated a positive/risk benefit profile at least in pulmonary fibrosis.
1428321-10-1 Pritelivir mesylate is an antiviral drug currently under development, specifically targeting herpes simplex virus types 1 and 2 (HSV-1 and HSV-2). It is being investigated as a potential treatment for various herpes infections, including those resistant to traditional antivirals like acyclovir.
FDA Approves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S. Subsequently, KEYTRUDA is used as monotherapy following the completion of adjuvant therapy.
With a particular focus on biologics, cell, and gene therapies, Galbraiths work is centred on equipping drug developers with the tools, insights, and support needed to advance their therapies from concept to clinic. These innovative modalities present both exciting possibilities and unique challenges in drug discovery and development.
Dive Insight: Lexeo is developingtreatments for heart conditions, such as cardiomyopathy associated with Friedreich’s ataxia and plakophilin-2 arrhythmia. Recent biotech company acquisitions have put emerging schizophrenia treatments in focus. But many development hurdles still stand in the way of new medicines for the brain.
This support will accelerate the clinicaldevelopment of the company’s pioneering DUO™ System—a minimally invasive transcatheter device designed to treat severe cases of tricuspid regurgitation. CroíValve’s DUO™ System presents a novel approach to TR treatment. CroíValve Awarded €12.5M CroíValve Awarded €12.5M
The delayed nature of cerebral ischemia secondary to SAH-related vasculopathy presents a window of opportunity for the evaluation of well-tolerated neuroprotective agents administered soon after ictus. Based on allometric scaling of both toxicological and efficacy data, the therapeutic margin in man should be at least 2.
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.
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.
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.
Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . United States and virtually from July 26 to 30, 2021 (Presentation No.:
Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinicaldevelopment program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. Late Breaking Oral Presentation OALC01LB03. Abstract 2361.
Chief Executive Officer of MorphoSys, will present at the 39 th Annual J.P. EST. Live audio of the presentation can be accessed from the Media and Investors section under Conferences on MorphoSys’ website, www.morphosys.com. The presentation as well as a replay of the webcast will also be available on MorphoSys’ website.
and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinicaldevelopment programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content