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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.
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.
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.
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.
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.
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.
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.
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.
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
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.
However, as we suggested in a recent blog post , the statute did not actually provide any limitations on FDAs authority to grant rare pediatric disease designations , only its authority to grant vouchers. In the meantime, the Pink Sheet confirmed that FDA was continuing to review rare pediatric disease designation requests.
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.,
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] 26 March 2025.
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.
They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). This leads us to Amgen’s T cell-engager therapeutic, tarlatamab.
Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. Clinical and Public Health Implications The data presented at SLEEP 2025 could mark a turning point in how clinicians approach long-term care in narcolepsy.
2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 6] In the United States, it has been designated by the Food and Drug Administration as a breakthrough therapy for patients with locally advanced or metastatic NSCLCs with an EGFR exon 20 insertion mutation. [7]
Expanding Targeted Protein Degradation in Hematologic Malignancies Targeted protein degradation is a powerful therapeutic approach designed to destroy disease-driving proteins within cells, thereby inhibiting their ability to contribute to disease progression.
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.
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.
An emerging concept is to localize the inhibition of VEGF or the inhibition of TGF- within the disease microenvironment, e.g. in the context of cancer and the TME. The interest from analysts is driven by near-term readouts from the COMPANION-002 Phase 2/3 Study including ORR in March and median PFS later in 2025.
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.
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).
Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up. mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As
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.
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.
Moreover, the Nantong facility plays a vital role in supporting WuXi AppTec’s mission to shorten drug discovery and development cycles, ultimately bringing critical therapies to market faster. By integrating operational excellence with architectural brilliance, the site has become a blueprint for the future of industrial science campuses.
Liso-cel achieved high, lasting response rates in patients with relapsed or refractory marginal zone lymphoma, underscoring its potential to significantly improve patient outcomes in this hard-to-treat disease,” said M. Lia Palomba, M.D. , With a follow-up of 21.6 months (median), 23.8 months, and 24.5
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. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.
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].
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.
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.
BioMarin Finalizes Acquisition of Inozyme Pharma, Strengthening Rare Disease Portfolio with INZ-701 BioMarin Pharmaceutical Inc. per share and brings Inozyme’s promising late-stage therapeutic candidate, INZ-701, into BioMarin’s growing pipeline of innovative enzyme therapies. Eastern Daylight Time, without any extension.
Taletrectinib CAS 1505514-27-1 as salt: 1505515-69-4 , Taletrectinib adipate FDA 6/11/2025, Ibtrozi, To treat locally advanced or metastatic ROS1-positive non-small cell lung cancer AB-106, DS-6051a 405.5 1] Taletrectinib was approved for medical use in the United States in June 2025. [3] April 2025. Retrieved 14 June 2025.
The ADA’s annual meeting is one of the most influential forums for showcasing scientific and clinical advancements in diabetes and metabolic disease research. The therapy is administered via subcutaneous injection and is being studied for its ability to produce and sustain significant weight loss over extended periods. to 3:00 p.m.
Successful Scale-Up Of An Upstream Viral Vector Process Using An Adherent Platform swheeler Thu, 02/27/2025 - 11:40 Thu, 04/10/2025 - 11:00 Resource Type Webinar Brian Gardell Cell and gene therapies offer hope for previously untreatable diseases, with viral vectors currently favored as the primary gene delivery method.
Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.
The data underscored the significant clinical benefits associated with cystic fibrosis transmembrane conductance regulator (CFTR) modulators—particularly its next-generation triple therapy, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor).
It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.
In this edition, our CEO and co-founder, Ariel Katz, along with Regional VP of Trial Landscape, Ryan Brown, share their top predictions for 2025. Ryan Brown , Regional VP, Trial Landscape Global standards for diversity plans will emerge in 2025. Ariel Katz , CEO & Co-Founder AI will be used to combat rare diseases.
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., Importantly, the therapy demonstrated a favorable safety profile, with no thromboembolic events reported so far. Engineered by Chugai Pharmaceutical Co.,
AMVUTTRA’s approval has not only underscored the therapeutic potential of RNAi-based medicines but also signaled Alnylam’s readiness to scale its capabilities and deepen its impact across a wider range of disease areas. I believe we are just beginning to scratch the surface of what RNAi therapeutics can do to improve human health.”
These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. Importantly, the combination therapy maintains a safety profile that aligns with what we’ve observed in monotherapy studies.” years in prior clinical studies.
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