What’s next for AAV gene therapies in 2024?
Pharmaceutical Technology
MARCH 11, 2024
Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024.
Mon.Mar 11, 2024
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Biotech stock fundings headed for best quarter in 3 years, Jefferies says
Bio Pharma Dive
MARCH 11, 2024
Publicly traded companies raised nearly $10 billion in follow-on stock offerings in January and February, a financing surge that’s driving a “sector recovery,” the investment bank said.
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Boehringer and Sosei Heptares partner on schizophrenia treatment
Pharmaceutical Technology
MARCH 11, 2024
Boehringer Ingelheim has entered a deal with Sosei Heptares for the development of treatments for all schizophrenia symptoms.
Amylyx CEOs look for a path forward following major setback
Bio Pharma Dive
MARCH 11, 2024
Justin Klee and Joshua Cohen spoke to BioPharma Dive about the next steps for their company, which is considering pulling its only product from market after the drug failed a confirmatory study in ALS.
4D Molecular Therapeutics files patent for gene therapy for fabry disease using optimized alpha-galactosidase a sequence
Pharmaceutical Technology
MARCH 11, 2024
Discover how 4D Molecular Therapeutics Inc. is revolutionizing gene therapy with a patent for delivering nucleic acids to treat disorders like Fabry disease.
Boehringer, Sosei Heptares team up in schizophrenia drug deal
Bio Pharma Dive
MARCH 11, 2024
Sosei Heptares could receive more than 700 million euros in a deal to develop medicines aimed at the protein GPR52, an emerging drug target in multiple neurological diseases.
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Roche, following setbacks, turns to its next Alzheimer’s drug
Bio Pharma Dive
MARCH 11, 2024
Roche said its experimental Alzheimer’s disease drug trontinemab showed it might be able to clear brain plaques faster than other medicines.
Corporate Hospitals forge path to TB elimination at Leadership Summit in New Delhi
AuroBlog - Aurous Healthcare Clinical Trials blog
MARCH 11, 2024
In a resounding declaration of solidarity and commitment, Corporate Hospitals from across India convened in New Delhi for a seminal workshop aimed at fortifying the nation’s fight against tuberculosis (TB).
Advanced technologies in unlocking the future of drug manufacturing
Bio Pharma Dive
MARCH 11, 2024
Innovative technologies, such as flow chemistry and biocatalysis, are achieving not only sustainability goals but also unprecedented levels of quality and efficiency.
AD/PD 2024: BioVie’s novel NE3107 shows promise but confirmatory trial needed
Pharmaceutical Technology
MARCH 11, 2024
However, a significant limitation of the early data is that the majority of trial participants were excluded from the analysis following GCP violations at multiple trial sites.
The New Age of Decentralized Clinical Trials
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White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations
Can using SDoH data help identify patient populations who could benefit from weight loss drugs?
Bio Pharma Dive
MARCH 11, 2024
SDoH data can help companies understand the footprint of weight loss medications in the market and their impact across populations.
Cordless Tube Saw Delivers Precision Cuts for High Purity Pharmaceutical Applications
Pharma Mirror
MARCH 11, 2024
Reimagining conventional tube saw design has led to an innovative, cordless model that cuts the widest range of piping and tubing sizes and materials with greater precision, efficiency, and longevity. The pharmaceutical industry requires extensive piping and tubing systems to maintain stringent quality and regulatory standards. These specialized systems are designed to ensure the purity of critical fluids, such as pharmaceutical-grade water, raw materials, active pharmaceutical ingredients (APIs
Eagle Pharmaceuticals files patent for stable pemetrexed formulations for cancer treatment
Pharmaceutical Technology
MARCH 11, 2024
Discover Eagle Pharmaceuticals Inc.'s innovative patent for stable pemetrexed formulations, offering effective treatment for pleural mesothelioma and non-squamous lung cancer. Learn about the unique composition and storage options designed for patient convenience and efficacy.
FDA approves first MASH drug: Madrigal's Rezdiffra breaks ground in notorious biopharma graveyard
Fierce Pharma
MARCH 11, 2024
The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended, as the FDA has approved the first drug for the fatty liver disease. | The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended. The FDA has approved Madrigal's resmetirom under the brand name Rezdiffra as the first drug for the fatty liver disease.
Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research
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Planning on running clinical trials in Japan? How can you reliably supply these studies? Discover Catalent’s clinical supply packaging facility in Shiga, Japan. Strategically located between Tokyo and Osaka, and one of largest in Japan, this 6,000 square meter facility offers comprehensive services including primary and secondary clinical packaging and labelling, comparator sourcing, cold chain storage, local and global distribution, local language support and white glove service to support stud
Celltrion files XOLAIR biosimilar for chronic spontaneous urticaria BLA
Pharmaceutical Technology
MARCH 11, 2024
Celltrion has filed a BLA with the US FDA for a biosimilar candidate to XOLAIR (omalizumab), CT-P39, for chronic spontaneous urticaria (CSU).
J&J’s growing rare disease focus brings a potential multi-use treatment to the table
Bio Pharma Dive
MARCH 11, 2024
The head of J&J’s autoantibody programs believes nipocalimab is unique among potential rare disease drugs because it can harness a common thread among them.
AD/PD 2024: Eli Lilly’s donanemab hit with FDA delay
Pharmaceutical Technology
MARCH 11, 2024
The FDA is still likely to approve donanemab, however, the delay will benefit Eisai and Biogen in allowing Leqembi to gain greater traction in the Alzheimer's market.
Pain is reduced by almost half with cannabis-based medicines – report finds
Outsourcing Pharma
MARCH 11, 2024
On March 7, Celadon Pharmaceuticals plc, a leading UK-based pharmaceutical company specializing in cannabis-based medicines, unveiled promising findings from an early economic analysis of its chronic pain clinical trial, dubbed CANPAIN.
The New Age of Decentralized Clinical Trials
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This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.
Astellas wins FDA orphan drug status for approved antifungal Cresemba
Pharmaceutical Technology
MARCH 11, 2024
Cresemba will enjoy seven years and six months of market exclusivity for the treatment of invasive aspergillosis and invasive mucormycosis.
Amylyx plummets as confirmatory trial of ALS drug fails
pharmaphorum
MARCH 11, 2024
Shares in Amylyx have cratered after the company reported a confirmatory trial of its amyotrophic lateral sclerosis (ALS) therapy Relyvrio missed all its objectives, putting its accelerated approval in jeopardy.
Sanofi’s amlitelimab shows best-in-class response in atopic dermatitis
Pharmaceutical Technology
MARCH 11, 2024
Sanofi presented the data as part of a late-breaking session at the American Academy of Dermatology (AAD) 2024 conference in San Diego, US.
Bluebird bio inks first Medicaid coverage agreement for sickle cell gene therapy, signing on with Michigan
Fierce Pharma
MARCH 11, 2024
Bluebird Bio has secured its first Medicaid outcomes-based agreement for its sickle cell disease gene therapy Lyfgenia, signing on with Michigan.
Roles and Responsibilities of Specialized Clinical Supply Experts
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When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.
Novo expands Wegovy’s US label to reduce cardiovascular events
Pharmaceutical Technology
MARCH 11, 2024
Wegovy can now be used to reduce the risk of major cardiovascular events in obese or overweight patients with heart disease.
Medable's Colin Weller addresses challenges and future of clinical trials
Outsourcing Pharma
MARCH 11, 2024
A conversation between Colin Weller, VP and GM of the evidence platform at Medable and Liza Laws, senior editor, at Outsourcing Pharma, was held at this yearâs SCOPE 2024 in Orlando.
Macau regulator accepts Everest’s NDA for ulcerative colitis therapy
Pharmaceutical Technology
MARCH 11, 2024
Macau has accepted a NDA of Everest Medicines for VELSIPITY, a treatment for adults with active ulcerative colitis (UC).
Enhancing clinical trials: The case for algorithmic prespecification in subgroup analysis
pharmaphorum
MARCH 11, 2024
Enhancing clinical trial outcomes through algorithmic prespecification in subgroup analysis can provide valuable insights for the FDA and medical community. Learn more about this practice and its importance in clinical research.
How Machine Learning Drives Clinical Trial Efficiency
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Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.
Mesoblast eyes accelerated approval filing for heart failure cell therapy
Pharmaceutical Technology
MARCH 11, 2024
The race for the first FDA-approved cell therapy for patients with heart failure heats up.
Sarcura: Can deep tech be utilized to enhance cell therapy manufacturing?
BioPharma Reporter
MARCH 11, 2024
Austian start-up Sarcura has secured a â1.7 million grant to help the company develop a âminiaturized and autonomousâ cell therapy manufacturing platform using deep tech.
The state of oligonucleotide therapeutic manufacturing: what biopharmaceutical players need to know
Pharmaceutical Technology
MARCH 11, 2024
In the genomic medicine toolbox, oligonucleotide therapeutics offer highly specific and effective treatments
How fibrinolytic biomarkers could transform the way we approach severe COVID-19
BioPharma Reporter
MARCH 11, 2024
One of the most challenging aspects of the COVID-19 pandemic was not being able to assess how individual patients would respond to the virus and how severe their symptoms would be.
Accelerating Clinical Supply Through Integrated Drug Development
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As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinical trials. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.
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