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Alltrna’s unique approach focuses on addressing genetic diseases at a fundamental level, shifting the paradigm from a one-disease, one-therapy model to a mutation-specific strategy that could transform the treatment of hundreds of rare diseases. This will mark a critical step in validating the potential of engineered tRNA therapeutics.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2
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.
This means it can block estrogen receptor activity and also degrade the receptor itself, potentially offering a more effective treatment approach. Clinical Trials: Palazestrant is currently in clinical trials, including Phase 1/2 and Phase 3 studies, for the treatment of ER+, HER2- metastatic breast cancer. March 2024).
Mengzhen Gu, Xiaoqing Xu, Xiaoping Wang, Yun Wang, Yu Zhao, Xiaoxian Hu, Lu Zhu, Zhenzhong Deng, and Chao Han Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.4c02277 4c02277 Histone lysine-specific demethylase 1 (LSD1) is hyperactive in breast cancer, which is associated with the metastasis of the tumor.
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.
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. Lifescienceleader.com. Lifescienceleader.com. Lifescienceleader.com.
2024 trends with momentum Personalized/precision medicine: Tailoring treatments to individual genetic profiles and specific patient characteristics is becoming increasingly important. This aims to ensure that treatments are effective across varied demographics and to address historical gaps in clinical research.
Dr Aaron Haubner, Senior Manager of North America Medical Affairs and Market Access at Terumo Blood and Cell Technologies , reveals that while promising new treatments emerge, urgent partnerships are needed to ensure this essential blood therapy reaches the patients who need it most.
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.
As the limitations of current treatments become increasingly clear, researchers are exploring new, safer approaches. His focus, however, is on addressing a critical gap in medicine: the lack of safe, non-addictive pain treatments – a challenge that has driven the development of SRP-001. “As Dr Bazan holds a B.S.
The findings underscored that automation improves data consistency and quality, thereby supporting more accurate diagnostics and personalised treatment strategies. The findings underscored that automation improves data consistency and quality, thereby supporting more accurate diagnostics and personalised treatment strategies.
According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8 However, getting essential treatments to patients quickly and safely requires more than just technological innovation. billion in 2023 to 1.2 billion in 2035.
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.
New drug triggers rapid cell death in cancer models By Karen Zusi-Tran October 29, 2024 Breadcrumb Home New drug triggers rapid cell death in cancer models BRD-810 inhibits the MCL1 protein and reactivates apoptosis in tumor cells, displaying therapeutic potential in animal models. Online August 23, 2024. Paper cited Rauh U, et al.
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] 3] Inavolisib was approved for medical use in the United States in October 2024. [3] Hz, 1H), 7.38 (br s, 1H), 7.18 (s, 1H), 7.00 (br s, 1H), 6.71 (t, J=55.9
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. npj Imaging 2024, 2 , 20. 2024, Article ASAP. 4 Similarly, Groseclose et al. Furthermore, Yang et al.
Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] 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.
However, despite impressive advancements, many patients still do not respond to current immunotherapy treatments. Recently, mitazalimab, Alligator Bioscience’s stimulatory monospecific CD40 antibody, has demonstrated success as a potential first-line treatment for pancreatic cancer. 2024: 25(7):853-864. 2024: 84(6).
Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. Toxicities were challenging enough to cause 13% of patients to reduce dose or skip doses and 3% to discontinue treatment. months (versus 20% ORR and 8.3 Stay tuned.
LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. Join us as we break down what went wrong this time. What Happened? But, where have we heard this all before?
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].
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.
A Step Forward in Frontline Multiple Myeloma Treatment Currently, Sarclisa is approved in the European Union for the treatment of adults with relapsed or refractory multiple myeloma (R/R MM) across three distinct indications.
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. Managing director Kasper Lage shares the center’s origin story, its achievements so far, and what the future holds.
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] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] Hidetoshi Hayashi (2024).
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.
Vamorolone , sold under the brand name Agamree , is a synthetic corticosteroid , which is used for the treatment of Duchenne muscular dystrophy. [4] In December 2023, it was approved in the EU for the treatment of patients ≥4 years of age. Treatment of crude 9.3 4] [5] [6] [7] [8] It is taken by mouth. [1] with peracetic acid 9.4
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.
As of the data cutoff in October 2024, each of these participants had completed at least 12 months of follow-up. No serious adverse events were attributed to the treatment itself. The outcomes presented by Vertex demonstrate strong and durable clinical efficacy, paired with a favorable safety profile.
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.
Since their initial development, both base editing and prime editing have been used by thousands of laboratories around the world and have enabled the study and potential treatment of many genetic diseases. He is the 2022 King Faisal Prize Laureate in Medicine, and the recipient of the 2024 Gabbay Award in Biotechnology and Medicine.
At the forefront of this mission is Senior Vice President of Vitalant, Becky Butler Cap, whose career has been long defined by a passion for building new services and treatment options in emerging markets. Building new services and treatment options for patients are a passion for me.
In Issue 7 of The Altascientist , we delve into these factors, the importance of drug interaction studies, and how to limit adverse effects and maximize treatment response. The FDA, 2024 FDA guidance M12 Drug Interaction Studies , pg.
Despite its impact, there are currently no FDA-approved therapies for PMN , and treatment options are limited to non-specific and often toxic agents such as chemotherapy or general immunosuppressants. The rights to felzartamab were licensed to Human Immunology Biosciences (HI-Bio) , which Biogen acquired in July 2024. Source link
This receptor is also expressed in the brain, where it helps to regulate dopamine release, making it a therapeutic target for the treatment of insomnia, pain, depression and Parkinson’s disease. Available at: [link] (Accessed: 23 September 2024). Jones EM, et al. doi:10.7554/elife.54895.
Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024. The impact of lengthening timelines Ballooning timelines in clinical trials have a profound impact on drug development, driving up operational costs and delaying market access for promising new treatments.
Despite having a final FDA guidance issued in 2017 , evolving drug development and treatment modalities continue to prompt important dialogue to adapt existing methods. One example is the recent interest in psychedelics for the treatment of psychiatric disorders, including major depressive disorder. Published in March 2025.
According to Takeda, oveporexton hit every main and secondary trial endpoint, helping improve patient-reported scores on measures of wakefulness, excessive daytime sleepiness, muscle weakness, and other symptoms after 12 weeks of treatment. 10, 2024 Psychiatry drugs finally have pharma’s attention. Can they keep it?
2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment. 7] [8] As of 2024, long-term safety and side effects remain undetermined. [8] 7] [8] As of 2024, long-term safety and side effects remain undetermined. [8]
Participants were randomized in a 4:1 ratio to receive either intravenous ARGX-119 or placebo over a 12-week treatment period, following a screening phase of up to 28 days. Importantly, nearly all participants in the Phase 1b trial had previously enrolled in an observational natural history study initiated by argenx in 2024.
3] In 2021, it was approved by the National Medical Products Administration of China for the treatment of complicated skin and soft tissue infections (cSSTI). [3] 3] In 2021, it was approved by the National Medical Products Administration of China for the treatment of complicated skin and soft tissue infections (cSSTI). [3]
This marks a pivotal moment for the treatment of a rare and aggressive group of cancers—including gallbladder cancer (GBC) and cholangiocarcinoma (CCA)—which are often diagnosed at an advanced stage when curative surgical options are no longer feasible. of patients, with the most frequent being diarrhea, fatigue, and elevated liver enzymes.
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