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
MONDAY, June 9, 2025 — Prompt treatment with a drug combo can effectively manage chronic kidney disease in people with type 2 diabetes, a new clinical trial shows. Patients prescribed the combination of finerenone and empagliflozin.
Looking ahead, 2025 could represent a major turning point for the pharmaceutical sector. Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time. Approaches that improve data efficiency will drive this space, Smith explains.
WEDNESDAY, June 18, 2025 — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront. The deal gives Lilly a potential new treatment for heart disease, The Wall Street Journal reported.
2025 , 20 , 6 , 1181–1194 [link] Cardiac hypertrophy is usually accompanied by many forms of heart disease, including hypertension, vascular disease, ischemic disease, and heart failure, and thus effectively predicts the increased cardiovascular morbidity and mortality.
Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health
From Research to Real-World Care 🌎 Understand how AI-driven advancements are shaping personalized treatments and patient outcomes. Navigating the AI Landscape 🤖 Explore the opportunities and challenges with integrating AI in healthcare and disease prevention. Register today to save your seat!
Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders.
Zhang, and Lun Kelvin Tsou Journal of Medicinal Chemistry 2025 DOI: 10.1021/acs.jmedchem.4c02665 4c02665 Pharmacological inhibition of the cGAS-STING-controlled innate immune pathway is an emerging therapeutic strategy for a myriad of inflammatory diseases. Here, we report GHN105 as an orally bioavailable covalent STING inhibitor.
Broad Institute and Bayer extend their cardiovascular alliance By Leah Eisenstadt June 11, 2025 Breadcrumb Home Broad Institute and Bayer extend their cardiovascular alliance Partnership will continue with renewed focus on developing new cardiovascular therapies.
Its therapeutic platform aims to go beyond traditional symptom management to directly tackle the genetic roots of diseases. The company’s lead candidate, NCX-L2 , is specifically designed to slow or even halt the progression of Parkinson’s disease—a neurodegenerative condition that currently has no disease-modifying therapies on the market.
The latest technologies are empowering researchers with faster, more efficient and more accessible genomic tools, leading to richer data generation and accelerating progress in areas like cancer genomics, rare disease research and infectious disease surveillance. 2025 [cited 2025 Jun 11]. 2025 [cited 2025 Jun 11].
By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.
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.
Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. It also holds potential for neuroinflammatory conditions such as certain types of migraine.
With AI, researchers predict the location of virtually any protein within a human cell By Corie Lok May 19, 2025 Breadcrumb Home With AI, researchers predict the location of virtually any protein within a human cell Trained with a joint understanding of protein and cell behavior, the model could help with diagnosing disease and developing new drugs.
By Sarah Wicks & Dara Katcher Levy FDAs Office of Prescription Drug Promotion (OPDP) issued its second Untitled Letter of 2025 to Taiho Oncology (Taiho) for a healthcare provider branded website for its drug LYTGOBI (futibatinib). DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).
It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.
Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients aged seven years and older with narcolepsy. of participants reported treatment-emergent adverse events (TEAEs), all of which were mild or moderate and consistent with previous safety data on Xywav. Approximately 32.8%
Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
argenx Unveils Positive Phase 2 Results for Efgartigimod in Myositis and Sjogren’s Disease at EULAR 2025 argenx SE a global immunology company dedicated to advancing treatments for severe autoimmune diseases, presented encouraging new clinical data at the 2025 European Congress of Rheumatology (EULAR), held June 11–14 in Barcelona, Spain.
David Liu receives Breakthrough Prize in Life Sciences By Corie Lok April 5, 2025 Breadcrumb Home David Liu receives Breakthrough Prize in Life Sciences Liu is honored for the development of base editing and prime editing, two gene editing technologies transforming medicine.
To this end, the December 2024 draft guidance and a companion guidance published January 7, 2025, Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway (the January 2025 draft guidance ) describe FDAs latest thinking on what it means to conduct a confirmatory trial with due diligence.
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.
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.
Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., from June 21 to 25, 2025. In the U.S.,
Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. April 10, 2025. Preclinical advantage. Lifecycle planning and repurposing. Acta Pharm Sin B. 2022 Jul;12(7):3049-3062. [2]
Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.
Sunvozertinib , sold under the brand name 舒沃哲, among others is an anti-cancer medication used for the treatment of non-small-cell lung cancer. [2] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]
DARPin therapeutics were a prominent theme at AACR 2025 , with Molecular Partners presenting a trio of preclinical programmes spanning radiopharmaceuticals and immune cell engagers. ” With an IND filing and first-in-human trials expected in 2025, MP0712 is moving steadily towards clinical evaluation.
Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. months (versus 20% ORR and 8.3
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 results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., This is critical, as hypercoagulation is a known risk when manipulating clotting pathways, especially in treatments designed to restore clotting function.
mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As The dual approach represents an effort to expand treatment efficacy across a broader subset of tumours, potentially improving outcomes. “In
1] It’s a privilege to participate in such a prestigious gathering of vision science professionals, and to gain insight into the latest innovations and treatments for complex ocular diseases. 1] [link] ; This blog was originally published in June 2025. conference locations. Tags Preclinical Research Weight 14
In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.
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.
The findings were presented as part of a late-breaking abstract session (#LB0001) at the European Alliance of Associations for Rheumatology (EULAR) Congress 2025 , held June 11–14 in Barcelona, Spain. No new safety signals emerged, and the treatment continued to be well tolerated. of patients achieving an ACR20 response versus 39.4%
A New Treatment Option for a Debilitating Joint Tumor TGCT is a rare, debilitating condition that primarily affects young and middle-aged adults, often striking individuals in their prime working years. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics 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).
Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.
This study explored the effects of beginning treatment with lower starting doses of MariTide to improve tolerability while maintaining clinical benefit. These results were evaluated using the efficacy estimand, a statistical model that accounts for treatment adherence and protocol deviations.
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 data were presented today in a late-breaking oral presentation at the 2025 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress in Glasgow, UK. This is crucial information for guiding both patients and physicians in their treatment decisions.” points greater in reduction with Dupixent (p < 0.00011).
2] Fitusiran was approved for medical use in the United States in March 2025. [2] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2]
The studies shared at ECFS 2025 encompassed a broad range of clinical outcomes, including pulmonary function, exacerbation frequency, nutritional metrics, and health-related quality of life.
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