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Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.
In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.
Artificial intelligence (AI) has revolutionised many industries, yet its adoption in pharmaceutical drug development has been notably slower. Looking ahead, 2025 could represent a major turning point for the pharmaceutical sector.
A gene editing therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics can mute sickle cell disease’s most damaging symptoms. Yet treatment may not be as simple as its dramatic benefit makes it seem.
While therapies like CAR-T have shown great success in hematologic malignancies, solid tumours remain a challenging area for current treatments. The process involves extracting lymphocytes from the tumour itself, expanding them, and then using these lymphocytes as a treatment.
The decision stands in contrast to the agency’s plans for a would-be rival gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that is also under a regulatory review.
Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. This has sparked the development of a new approach to treating neuropsychiatric disorders.
As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. This is exciting because often resistance builds up to these other treatments.
A partnership between University of Oxford, the Earlham Institute, and the global pharmaceutical companies Biogen Inc and Boehringer Ingelheim is announced today to investigate a new drug target for the treatment of schizophrenia.
In an interview with PharmaVoice, Alicia Zhou, the new head of the nonprofit Cancer Research Institute, expressed a desire to foster deeper partnerships between scientists and the pharmaceutical industry.
The Dark Side of Innovation: How Companies Use Drug Patents to Block Competitors As we celebrate the breakthroughs in medical research and the development of life-saving treatments, it's essential to acknowledge the complex landscape of pharmaceutical innovation. But at what cost? Let's start a conversation in the comments below.
In the vast realm of pharmaceutical research and development, there’s a fascinating intersection between ancient wisdom and modern science. “Pharmacognosy is the bridge between traditional medicine and modern pharmaceutical science, offering a treasure trove of potential new drugs waiting to be discovered.”
Longer follow-up results from a mid-stage trial indicate the company’s treatment for a genetic cause of dwarfism may be competitive to an approved therapy from BioMarin Pharmaceutical.
Most of these conditions are genetic in origin and the majority have no effective treatment. That includes countless rare peripheral diseases, including many for which there are currently no treatments. Boosting autophagy won’t be the only road to new treatments for rare genetic diseases but it is a vital one to take.
Ebert, USA, receives this year’s Sjöberg Prize, worth one million US dollars, for his research into how lenalidomide works as a treatment for blood cancer. His discovery of the mechanism that promotes protein breakdown in cancer cells may be vitally important to the future development of new pharmaceuticals.
Kendal Chidwick NDARC’s Kendal Chidwick discusses the findings from a recently published study that examined how patterns of opioid agonist treatment medicines have changed over the past decade. Other changes around this time aimed at improving treatment accessibility included increased use of take-home doses and telehealth appointments.
The Unsung Heroes of Generic Drug Development: The Power of Partnerships As we navigate the complex landscape of pharmaceuticals, it's easy to overlook the crucial role that partnerships play in bringing affordable, life-saving medications to market. What do you think is the most significant challenge facing generic drug development today?
The Swiss pharmaceutical company has agreed to buy Televant, a company set up by Roivant and Pfizer to develop a promising inflammatory bowel disease treatment.
The rising cost of brand-name medications has left millions of people struggling to afford the treatments they need to manage their conditions. This is a win-win for everyone involved patients get the treatments they need, and healthcare systems can allocate resources more efficiently. So, what's the future hold for biosimilars?
8 With the widespread use of induced pluripotent stem cells (iPSCs), organoids can now be generated from the skin or blood cells of both healthy volunteers and disease patients, opening the door to studying various diseases and creating individually customised potential treatments. Organoids in cancer research. Nat Rev Cancer. Cell Stem Cell.
However, getting essential treatments to patients quickly and safely requires more than just technological innovation. A global network with local expertise To ensure the delivery of treatments to patients worldwide, biotech and biopharma companies also need partners that can provide comprehensive solutions from a geographic perspective.
2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1] 26 March 2025.
While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.
This approach not only helps maintain market exclusivity but also ensures a steady revenue stream for pharmaceutical companies. Understanding the Pharmaceutical Market Dynamics The pharmaceutical industry is a complex ecosystem where branded drugs and generics coexist, each playing a vital role in patient care and market dynamics.
Vancouver-based Algernon Pharmaceuticals plans to conduct a late-stage study to evaluate an orally delivered drug, NP-120 (ifenprodil), as a COVID-19 treatment. The drug was originally developed in the 1990s by Sanofi to treat peripheral circulatory disorders and it is still used in generic form in Japan.
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.
percent) reduction in the treatment group. About the author James Graham, CEO of Recce Pharmaceuticals Recce Pharmaceuticals. James Graham is the CEO of Recce Pharmaceuticals. Our preclinical studies performed in infected mice demonstrated significant bactericidal activity against N. gonorrhoeae , with a 4-log (99.99
Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&
Biosimilars, which are biologic drugs that are highly similar to an already approved biologic drug, are transforming the pharmaceutical industry. As patents for original biologic drugs expire, biosimilars are becoming increasingly important, offering more affordable treatment options for patients and healthcare systems.
3c05899 Owing to their remarkable pharmaceutical properties compared to those of noncovalent inhibitors, the development of targeted covalent inhibitors (TCIs) has emerged as a powerful method for cancer treatment.
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.
Our expertise in alopecia areata studies As of July 2023, there were more than 60 planned or ongoing clinical trials for alopecia areata, involving nearly 20 pharmaceutical or biotech organizations. Accelerate your alopecia areata treatments. Learn more The post Advancing New Treatments for Alopecia Areata appeared first on PPD.
These biologic drugs are generally administered by injection and include arthritis treatments like Humira, cosmetic drugs like Botox, and cancer drugs like Keytruda, which famously reversed Jimmy Carter’s brain metastases. The small molecules drugs of today look nothing like the molecules of the 1970s.
1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 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. under nitrogen.
Monument recently raised £2.625 million in seed funding and applies a novel drug development strategy, leveraging digital assessments of cognition to match patients with new pharmaceuticaltreatments.
The pharmaceutical industry is constantly evolving, and one of the most exciting areas of growth is the development of biosimilars. These biological products, designed to be highly similar to existing approved biologics, offer the promise of more affordable treatment options for patients.
A new shift is occurring in the pharmaceutical industry, leading to a rapidly expanding field known as digital therapeutics (DTx). DTx interventions include sensor-equipped wearable devices, remote patient monitoring tools, and virtual reality platforms integrated with conventional pharmaceuticaltreatments.
Glecaprevir, or ABT-493, was approved in combination with pibrentasvir (Mavyret) for the treatment of Hepatitis C (HCV) in 2017. RCM in the Medicinal Chemistry of Pacritinib Pacritinib, a kinase inhibitor approved in 2022 for treatment of myelofibrosis, was also synthesized with an RCM in its med. Gonzalez-Bobes, F.
Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.
Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.
Pharmaceutical researchers, in particular, have a keen interest in understanding the unfolding dynamics of this market. Inspired by its success, pharmaceutical companies are pursuing the development of drugs with similar therapeutic profiles, aiming to expand treatment options.
However, the underlying mechanisms of DCM are poorly understood, and treatment options are limited. DCM is the leading cause of heart failure in patients with chronic diabetes. Another mystery is the regulation of cytochrome P450 enzymes (CYPs) in the central nervous system.
Federal Court Orders Puerto Rico Pharmaceutical Distributor to Pay $12 Million in Connection with Alleged Failure to Report Suspicious Orders of Pharmaceutical Drugs and Other Controlled Substance Violations, Nov. 6, 2023 ( DOJ Press Release ). 21 U.S.C. § 829; 21 C.F.R. Complaint ¶ 58. Complaint ¶ 59.
Environmental Impacts of Traditional Pharmaceutical Production Traditional pharmaceutical manufacturing is complex, with each phase contributing to environmental degradation. The extraction and synthesis of active pharmaceutical ingredients (API) utilize vast amounts of water and energy.
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