U.S. FDA approves Pfizer & #039;s BEQVEZ ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with Hemophilia B
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. (Source: World Pharma News)
Source: World Pharma News - April 26, 2024 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news
AskBio receives FDA Fast Track Designation for AB-1002 investigational gene therapy program in congestive heart failure
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1. (Source: World Pharma News)
Source: World Pharma News - April 25, 2024 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Researchers publish final results of key clinical trial for gene therapy for sickle cell disease
In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) - a blockage that results in lack of oxygen and painful episodes - for at least one year, and 100% were able to remain hospitalization-free for the same length of time. (Source: World Pharma News)
Source: World Pharma News - April 24, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Optimal timing maximises Paxlovid benefits for treating COVID-19
Researchers have described the optimal timing for COVID-19 patients to take the antiviral, Paxlovid, to get the most benefit from the treatment, according to a study published April 16 in eLife.
The findings suggest that taking Paxlovid three to five days after COVID-19 symptoms emerge may maximise the drug's ability to reduce viral loads, minimise viral spread and reduce viral rebound. (Source: World Pharma News)
Source: World Pharma News - April 23, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
European Commission approves Pfizer & #039;s EMBLAVEO ® for patients with multidrug-resistant infections and limited treatment options
Pfizer Inc. (NYSE: PFE) today announced that the European Commission (EC) has granted marketing authorization for EMBLAVEO® (aztreonam-avibactam) for the treatment of adult patients with complicated intra-abdominal infections (cIAI), hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP), and complicated urinary tract infections (cUTI), including pyelonephritis. It is also indicated for the treatment of infections due to aerobic Gram-negative organisms in adult patients with limited treatment options. (Source: World Pharma News)
Source: World Pharma News - April 22, 2024 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news
Researchers develop a new way to safely boost immune cells to fight cancer
Cancer is the monster of our society. Last year alone, more than 600,000 people in the United States died from cancer, according to the American Cancer Society. The relentless pursuit of understanding this complex disease has shaped medical progress on developing treatment procedures that are less invasive while still highly effective.
Immunotherapy is on the rise as a possible solution. Immunotherapy involves harnessing the power of the body's immune system to fight against cancer cells. (Source: World Pharma News)
Source: World Pharma News - April 19, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
How the Birmingham Drug Discovery Hub created an investment-ready & #039;drug library & #039;
A novel approach to drug discovery is enabling University of Birmingham researchers to overcome the 'valley of death', where projects fail due to the funding gap between original research and commercial investment.
The approach, detailed in a feature published in the April issue of Drug Discovery Today, has attracted more than £4m in industry funding, grants and industry awards, on the back of just £0.2m investment from the University's Dynamic Investment Fund (DIF). (Source: World Pharma News)
Source: World Pharma News - April 18, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Common HIV treatments may aid Alzheimer & #039;s disease patients
Alzheimer's disease (AD) currently afflicts nearly seven million people in the U.S. With this number expected to grow to nearly 13 million by 2050, the lack of meaningful therapies represents a major unmet medical need. Scientists at Sanford Burnham Prebys have now identified promising real-world links between common HIV drugs and a reduced incidence of AD. The study, led by Jerold Chun, M.D., Ph.D., was published in Pharmaceuticals.
Chun's new research builds on his lab's landmark publication in Nature in 2018 that described how somatic gene recombination in neurons can produce thousands of new gene variants within Alz...
Source: World Pharma News - April 17, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson & #039;s disease
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson's disease (PD).(1,2) The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.
The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. (Source: World Pharma News)
Source: World Pharma News - April 16, 2024 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Next-generation treatments hitch a ride into cancer cells
Antisense oligonucleotides (ASOs) are next-generation drugs that can treat disease by blocking the transfer of harmful messages from our genes. In people with cancer, ASOs have the potential to block messages that encourage the growth and spread of the tumor. However, ASOs aren’t used for treating cancer yet. They must first get delivered inside cancer cells, but the cancer cells won’t let them in.
Finding an effective ASO delivery system is a major challenge. Cancer cells have gatekeeper molecules that stop unwanted substances from entering. (Source: World Pharma News)
Source: World Pharma News - April 15, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Groundbreaking treatment approach shows promise in hard-to-treat cancers
Researchers have developed a functional precision medicine approach that targets cancer by combining genetic testing with a new way to test individual drugs on tumor samples. The results of the clinical study were published today in Nature Medicine.
This combined approach, developed by Florida International University cancer researcher Diana Azzam, was used successfully for the first time to guide treatment of relapsed pediatric cancer patients in collaboration with First Ascent Biomedical and Dr. Maggie Fader at the Helen & Jacob Shaham Cancer & Blood Disorders Institute at Nicklaus Children's Hospital in Miami. (Sourc...
Source: World Pharma News - April 12, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
This outdated diabetes drug still has something to offer
Thiazolidinediones (TZDs) are a class of drug that can be used to treat type 2 diabetes by reversing insulin resistance, one of the main hallmarks of the disease. While TZDs were extremely popular in the 1990’s and early 2000’s, they have fallen out of use among physicians in recent decades because they were discovered to cause unwanted side effects, including weight gain and excess fluid accumulation in body tissues. (Source: World Pharma News)
Source: World Pharma News - April 11, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
New drug prevents flu-related inflammation and lung damage
Infection with the influenza virus leads to lung injury through inflammation over-activation that causes collateral damage to cells required for breathing. Such damage can be life-threatening, but scientists have a new preventative treatment. A team from St. Jude Children's Research Hospital, University of Houston, Tufts University School of Medicine and Fox Chase Cancer Center created a drug that can prevent flu-induced lung injury. In a mouse model, the drug achieves a novel balance between shutting down runaway inflammation and allowing the immune system to stop the virus. (Source: World Pharma News)
Source: World Pharma News - April 10, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Swapping red meat for herring/sardines could save up to 750,000 lives/year in 2050
Swapping red meat for 'forage fish', such as herring, sardines, and anchovies, could save up to 750,000 lives a year in 2050 and significantly reduce the prevalence of disability as a result of diet-related disease, suggests a data analysis published in the open access journal BMJ Global Health.
Adopting this type of diet would be especially helpful for low and middle income countries, where these fish are cheap and plentiful, and where the toll taken by heart disease, in particular, is high, say the researchers. (Source: World Pharma News)
Source: World Pharma News - April 9, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
Does cannabis use affect cognitive decline?
A new study by Upstate Medical University researchers shows that recreational cannabis use may offer protection against cognitive decline.
The study, done by Master of Public Health (MPH) student Zhi Chen and Professor Roger Wong, Ph.D., MPH, MSW, analyzed a large data set from the CDC and found that compared to non-users, non-medical cannabis use, such as for recreational purposes, was significantly associated with 96 percent decreased odds of subjective cognitive decline (SCD). (Source: World Pharma News)
Source: World Pharma News - April 8, 2024 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news
