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The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
Nowak Annual Reports in Medicinal Chemistry , 2024 [link] Proteolysis targeting chimeras (PROTACs) are hetero-bifunctional molecules that remove disease-causing proteins through the means of targeted protein degradation (TPD). Geiger, Radosław P.
By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.
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.
integrated their GWAS findings with functional genomics data from relevant tissues, identifying 700 genes with high confidence of being involved in osteoarthritis, and eight biological processes key to the disease development, including circadian clock and glial cell functions. GWAS credible sets from Hatzikotoulas et al. GCST90566795 ).
Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.
“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. Today’s broad-spectrum drugs often miss the mark, especially for chronic diseases like COPD or heart failure.
Valentine We recently blogged about a new December 2024 draft guidance about accelerated approval (the December 2024 draft guidance). While the first topic is addressed primarily in the December 2024 draft guidance, the considerations and policies FDA articulates in the January 2025 draft guidance are also relevant.
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.
Tomonori Tamura, Masaharu Kawano, and Itaru Hamachi Chemical Reviews 2024 DOI: 10.1021/acs.chemrev.4c00745 Drugging such undruggable targets is essential to develop new therapies for diseases where current treatment options are limited or nonexistent.
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. and EU, in late 2024 as the first therapy of its kind. Qfitlia was approved in multiple regions, including the U.S.
NGS has revolutionised genomic analysis, enabling the identification of disease-related genetic variants. This ability to correlate seemingly disparate data is crucial for tackling complex targets and discovering new treatments for diseases considered difficult to treat with conventional approaches. Lifescienceleader.com.
This issue is particularly pronounced for rare diseases and trials requiring diverse patient populations. 2024 trends with momentum Personalized/precision medicine: Tailoring treatments to individual genetic profiles and specific patient characteristics is becoming increasingly important.
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. 2024 Dec 1; 8. Ball M, Romanovsky E, Schnecko F, et al.
2024) 6 demonstrated MSI’s ability to distinguish biliary toxicants by revealing distinct PKs within hepatocytes and bile duct cells, informing the development of safer alternatives. 2024, 52 (11), 1181–1186. Multimodal Imaging Distribution Assessment of a Liposomal Antibiotic in an Infectious Disease Model. Drug Metab.
The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.
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.
1] Medical uses In the US, sunvozertinib is indicated for the treatment of adults with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. [1] 9 April 2024.
no comparator arm), FOENIX-CCA2 was not capable of establishing improvement on time-to-event efficacy endpoints such as PFS or OS and, without an appropriate comparator, it is not possible to determine if the observed effect is attributable to LYTGOBI or to other factor(s), such as the natural history of the disease.
3] Tofersen was approved for medical use in the United States in April 2023, [3] [6] and in the European Union in May 2024. [4] 22 February 2024. Retrieved 24 February 2024. 3 June 2024. January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024. ^ Retrieved 16 May 2024.
3] Inavolisib was approved for medical use in the United States in October 2024. [3] 3] Inavolisib was approved for medical use in the United States in October 2024. [3] 11 October 2024. Retrieved 11 November 2024. 10 October 2024. Retrieved 11 October 2024. 1 October 2024. 3 November 2006. .
With monthly or less frequent administration , robust and sustained weight loss , significant metabolic improvements , and a favorable safety profile , MariTide may address critical gaps in current obesity treatment paradigms.
The 2024 Nobel Prize in Chemistry was awarded to Demis Hassabis and John Jumper from Google DeepMind for developing AlphaFold2, and to David Baker from the University of Washington for his work in computational protein design. These innovations have revolutionised the understanding of protein structures using artificial intelligence.
Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). The results were remarkable for this very sick patient population, with a median OS of 14 months at the dose of 10 mg given every 2 weeks: The data supported FDA approval, in May 2024, of tarlatamab for refractory ES-SCLC.
For some, however, disease management becomes even more precarious due to impaired hypoglycemia awareness and a heightened risk of severe hypoglycemic events (SHEs). As of the data cutoff in October 2024, each of these participants had completed at least 12 months of follow-up.
The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions. Available at: [link] (Accessed: 23 September 2024).
Part one of the GMMG-HD7 study was presented at the 2024 American Society of Hematology (ASH) Annual Meeting and simultaneously published in the Journal of Clinical Oncology. Sanofi is now one step closer to establishing Sarclisa as a foundational therapy in the management of multiple myeloma.
Primary membranous nephropathy is a rare and serious autoimmune kidney disease characterized by the formation of autoantibodies that damage the glomerular basement membrane, often resulting in nephrotic syndrome —a condition marked by high levels of protein in the urine, severe swelling, and a significant risk of progression to kidney failure.
Ncardia, 2024. Throughout her experience in academic research, she gained distinct knowledge and expertise in both disease modelling and biobank building and in the differentiation of iPSCs into cardiac and neuronal cell types. Conventional models often fail to predict cardiac side effects, especially with biologics.
argenx remains focused on uncovering new biological insights into misunderstood diseases to meaningfully change the lives of patients who have long-been underserved.” argenx remains focused on uncovering new biological insights into misunderstood diseases to meaningfully change the lives of patients who have long-been underserved.”
SCHEME SIDECHAIN MAIN PATENT WO2021250648 PFIZER WO2023215910 PAPER The Pfizer scientists described ibuzatrelvir’s medicinal chemistry campaign in a Journal of Medicinal Chemistry paper that was published in April 2024 (DOI: 10.1021/acs.jmedchem.3c02469). August 2024). August 2024). 2024 Mar 13;16(738):eadi0979. [4].
Durable Benefit and Positive Clinical Outcomes “Today’s results underscore the promising potential of Intellia’s approach to genome-editing therapy — a one-time treatment that has been well tolerated and offers a highly differentiated, durable effect for patients suffering from a serious disease,” said John Leonard, M.D.,
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.
Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024. Strategies and goals Demand for regulatory consulting has surged in 2024 as drug developers face a growing need for specialized support.
“Ziihera offers a targeted monotherapy with a favorable safety profile and compelling efficacy, representing a crucial advance in the fight against this challenging disease.” Arndt Vogel , professor of gastroenterology at Hannover Medical School.
The extended agreement will support joint research efforts targeting critical therapeutic areas, including oncology, cardiovascular and renal diseases, neurology, rare diseases, and immunology. In September 2024 , Bayer expanded its innovation commitment by opening a new Bayer Co.Lab site in China.
Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. These therapeutics share some characteristic measures of clinical efficacy, including high initial overall response rates but a low rate of complete responses and limited duration of response. Br J Cancer 124: 10371048.
With an extensive background in these fields, Ichim is dedicated to advancing personalised cellular therapies aimed at addressing age-related diseases, with a special focus on cancer and liver failure. Ichim’s work at Immorta Bio is focused on transforming the way we approach age-related diseases and cancer treatment.
Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. Can We Lay This Scourge to Rest?
Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.
2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 4] A medication used to reduce the amount of steroid replacement required in patients with a genetic disease that causes, amongst other symptoms, a steroid deficiency. . 1 December 2024. 1 October 2024. Retrieved 16 December 2024.
Next month, families affected by the rare genetic disease metachromatic leukodystrophy (MLD) will meet in Washington. MLD and Duchenne muscular dystrophy were slated to join the RUSP next month – neither is on the most recent update from July 2024. It can take years. But the family groups won’t back down.
Researchers identify source of a brain cancer’s deadly transformation By Corie Lok November 21, 2024 Breadcrumb Home Researchers identify source of a brain cancer’s deadly transformation Slow-growing glioma tumors become more aggressive when their cells shift identities and acquire new genetic mutations. Online November 21, 2024.
11] Zilucoplan was approved for medical use in the United States in October 2023, [6] [12] in the European Union in December 2023, [7] and in Australia in July 2024. [1] 24 September 2024. Retrieved 12 October 2024. ^ “Therapeutic Goods (Poisons Standard—June 2024) Instrument 2024” Federal Register of Legislation.
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