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Flavonoids exhibit significant potential in cancer therapy by inhibiting MMP-2 and MMP-9 activity and expression, primarily through interactions with their active sites and modulation of the MAPK signaling pathway. ABSTRACT Last few decades, extensive research efforts have been dedicated to uncovering novel cancer treatments.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. The Natural History Studies Grants Program funds natural history studies that collect gather data on how rare diseases progress over time without treatment.
The regulator will issue a decision on lovo-cel by Dec. 20, roughly two weeks after a verdict is expected on a rival gene editing treatment from Vertex and CRISPR Therapeutics.
By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.
FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.
All these features make GPCRs central to regulating many vital biological processes in the body, including immune responses and inflammation, and thus especially attractive for therapeutic intervention. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.
BIIB129 (25) demonstrated efficacy in disease-relevant preclinical in vivo models of B cell proliferation in the CNS, exhibits a favorable safety profile suitable for clinical development as an immunomodulating therapy for MS, and has a low projected total human daily dose.
Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach.
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.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.
In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. Kosten Digital was established to bridge this gap.
Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.
These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes. Regulators care a lot about controlling the Type 1 error rate of the clinical trial, Smith notes.
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Neuropsychiatric treatment is on the verge of a major transformation. Neuropsychiatric disorders, affecting millions worldwide, disrupt the brain’s intricate processes of mood regulation, cognition and behaviour. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,
This alliance aims to address mounting challenges in the development and commercialization of mAb therapies and gene therapies, particularly those involving AAV vectors. Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers.
Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system. Consequently, the accumulation of β-amyloid not only exerts stress on other brain cells but also affects endothelial cells responsible for regulating cerebral blood flow.
GBM has a poor prognosis despite aggressive treatment, in part due to lack of adequate drug permeability at the BBB. Standard of care GBM therapies include radiation and cytotoxic chemotherapy that lead to DNA damage. These observations can guide further exploration of elimusertib for the treatment of GBM, or other CNS tumors.
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. Adapted from an Allen Institute press release.
FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. For many years, systemic corticosteroids have been the mainstay of BP treatment. Dr. George D.
ABSTRACT Protein arginine methyltransferase 5 (PRMT5) is an epigenetic-related enzyme that has been shown to be a promising target for the treatment of human cancers. Finally, Western blotting was used to detect the mechanism of action of the compounds in the treatment of prostate cancer.
Approximately two in 10 patients with hematologic malignancies are estimated to be eligible and able to receive cell therapy. The REMS Burden Explained Once deemed necessary for managing the risks associated with CAR T therapies, the REMS requirements imposed significant operational burdens on treatment centers.
Multiple investigations prove that it is regulated genetically, and epigenetically, and is affected by environmental factors. The molecular and neural pathways linked to the regulation of schizophrenia have been extensively studied. Scientists are trying to use viral vectors, miRNA, siRNA, and CRISPR technology.
These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects. Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.
Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. This limited pool poses significant barriers to conducting statistically significant studies and validating the efficacy and safety of new treatments.
In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. Existing therapies, such as tocilizumab, work by blocking specific inflammatory pathways but often come with limitations.
In an era where biotherapeutics are transforming the healthcare landscape, the organisation, Vitalant , is uniquely positioned to bridge the gap between cutting-edge therapies and community access. Building new services and treatment options for patients are a passion for me.
Chan, Gui Lu European Journal of Medicinal Chemistry , 2023 [link] Aurora kinases, which belong to the serine/threonine protein family, play critical roles in the regulation of the cell cycle and mitotic spindle assembly. Despite the development of some reversible Aurora kinase inhibitors, none has been approved for clinical use yet.
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.
Acacetin may be a promising drug in complementary therapy of cancer treatment. Acacetin treatment induced bax's expression and repressed bcl-2's expression. Abstract Acacetin is a natural flavonoid compound found in diverse plants, which has strong anti-inflammatory and anti-cancer activities.
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.
Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. However, as your high-school literature teacher warned youto ace the test, you need to read the book, ahem, source regulations, guidance, or other policy documents.
They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. As we’ll see, ASCO 2025 was a breakout conference for B7-H3-targeted therapies. Advanced therapies targeting B7-H3 use different modalities, currently dominated by ADCs.
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.
Revolutionizing Treatment Through the Skin with Transdermal Drug Delivery Systems The human skin, often perceived as a passive barrier to the external environment, holds potential as a route for drug administration. This membrane regulates the diffusion of the drug from the reservoir into the skin.
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 Unlike the newly approved NaV1.8
Increasing evidence suggests that ALDH2 is a crucial regulator of human tumor development, including HCC. Therefore, clarifying the relationship between ALDH2 and HCC is helpful for formulating rational treatment strategies. Meanwhile, we envisioned viable strategies for targeting ALDH2 in the treatment of HCC.
Through relationship-building and increased engagement, regulators aim to improve their understanding of the ‘lived experience’ of patients with rare diseases and hear their top concerns.
A Patient-Centered Innovation in B-Cell Cancer Treatment Zanubrutinib, marketed as BRUKINSA, is a next-generation Bruton’s tyrosine kinase (BTK) inhibitor approved for the treatment of various B-cell malignancies. In the United States, BRUKINSA has continued to demonstrate its clinical and commercial strength.
Providing treatments for our community means understanding the different ways KAND can manifest, and learning from other disease groups with overlapping symptoms. These molecules regulate neuronal growth and survival, and mice with insufficient KIF1A show NTRK1 transport defect and reduced pain sensitivity. SCN9A/Nav1.7:
Agonist antibodies of immune checkpoint regulators These represent a groundbreaking class of immunotherapeutic agents that mimic the natural function of endogenous ligands by binding to specific cell-surface receptors. In these conditions, the goal of therapy is typically to suppress or mitigate immune activity.
KIF1A-Related Research Preferential transport of synaptic vesicles across neuronal branches is regulated by the levels of the anterograde motor UNC-104/KIF1A If axons are the highways KIF1A drive on, synapses are like the off-ramps and intersections for local deliveries.
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