An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH). Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age. Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy at the NIH Clinical Center and then followed for about six years to assess safety.

Bayer AG and Thermo Fisher Scientific Inc. today announced a collaboration to develop next-generation sequencing (NGS)-based companion diagnostic assays (CDx) together. These will help identify patients who may benefit from Bayer's growing portfolio of precision cancer therapies by offering decentralized genomic testing and rapid turnaround time.

A new cancer treatment that uses a person’s own immune cells has been approved by the U.S. Food and Drug Administration (FDA) for treating the most dangerous type of skin cancer. Now this form of cellular therapy (tumor-infiltrating lymphocyte - or TIL therapy) is showing promise in advanced lung cancers through clinical trials underway at The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC - James).

A new study has discovered that a type of bacteria commonly found in the body, which usually does not pose problems for healthy people, plays a significant role in causing stomach cancer, the fifth most common cancer in the world.

Streptococcus anginosus bacteria exist alongside other germs in the mouth, throat, intestines and vagina. Occasionally, they may cause mild infections like sore throats and skin infections.

Bayer and Aignostics GmbH announced a strategic collaboration on several artificial intelligence (AI)-powered approaches with applications in precision oncology drug research and development. Aignostics is a spin-off from one of the world's leading hospitals, Charité-Universitätsmedizin Berlin, and a global leader in using computational pathology to transform complex biomedical data into biology insights.

AstraZeneca announced that it has entered into a definitive agreement to acquire Amolyt Pharma, a clinical-stage biotechnology company focused on developing novel treatments for rare endocrine diseases.

The proposed acquisition will bolster the Alexion, AstraZeneca Rare Disease late-stage pipeline and expand on its bone metabolism franchise with the notable addition of eneboparatide (AZP-3601), a Phase III investigational therapeutic peptide with a novel mechanism of action designed to meet key therapeutic goals for hypoparathyroidism.

Pfizer Inc. (NYSE: PFE) today announced that the European Commission (EC) has granted marketing authorization for the company's 20-valent pneumococcal conjugate vaccine, marketed in the European Union under the brand name PREVENAR 20®, for active immunization for the prevention of invasive disease, pneumonia and acute otitis media caused by Streptococcus pneumoniae in infants, children and adolescents from 6 weeks to less than 18 years of age.

There are thousands of diseases worldwide with no cure or available treatments. Traditional drug discovery and development takes decades and billions of dollars and more than 90% of these drugs fail in clinical trials. The emergence of artificial intelligence (AI) holds promise for streamlining and improving the entire process. However, ushering in a new era of AI-driven drug discovery requires costly and lengthy validation in preclinical cell, tissue, and animal models and human clinical trials.

A new study has shed light on the importance of the protein STAP-1 in activating certain immune cells. Understanding the role of STAP-1 in these cells could give researchers a better glimpse into immune-related disorders and ways to treat them.

The researchers found that STAP-1 plays an important role in the activation of T cells, which are white blood cells that play a critical role in defending the body against infections and maintaining overall health.

A team of University of Michigan researchers has successfully modified a naturally occurring chemical compound in the lab, resulting in advanced lead compounds with anti-HIV activity.

Their results, published March 7 in the Journal of Medicinal Chemistry, offer a new path forward in the development of drugs that could potentially help cure - rather than treat - HIV.

In a study published in Cell on March 5, Prof. ZHANG Xiaoming at the Shanghai Institute of Immunity and Infection (SIII) of the Chinese Academy of Sciences and Profs. GAO Qiang, FAN Jia and YANG Li at Fudan University have uncovered an unexpected level of complexity hidden within neutrophils, which were previously thought to be a relatively uniform population of short-lived immune cells.

Scientists discover a new way to disarm a deadly protein that also appears in cancers of the lung, breast and colon.

UC San Francisco researchers have designed a candidate drug that could help make pancreatic cancer, which is almost always fatal, a treatable, perhaps even curable, condition.

The new molecule permanently modifies a wily cancer-causing mutation, called K-Ras G12D, that is responsible for nearly half of all pancreatic cancer cases and appears in some forms of lung, breast and colon cancer.

New Cleveland Clinic-led research points to sildenafil (Viagra) as a potential treatment for Alzheimer's disease. The study provides evidence from computational models, insurance claims data and observations from brain cells in Alzheimer's patients.

Sildenafil is the main component of drugs used to treat erectile dysfunction (Viagra) and pulmonary arterial hypertension (Revatio).

Novartis today presented new data that continue to support the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment.

Cleveland Clinic researchers have discovered a key mechanism used by Kaposi's sarcoma-associated herpesvirus (KSHV), also known as human herpesvirus 8 (HHV8), to induce cancer. The research points to effective new treatment options for KSHV-associated cancers, including Kaposi's sarcoma, primary effusion lymphoma, and HHV8-associated multicentric Castleman disease.

Pfizer Inc. (NYSE: PFE) today announced top-line ABRYSVO® vaccine efficacy and safety data for respiratory syncytial virus (RSV) in adults 60 years of age and older following a second season in the Northern and Southern Hemispheres from the ongoing pivotal Phase 3 clinical trial (NCT05035212) RENOIR (RSV vaccine Efficacy study iNOlder adults Immunized against RSV disease).

A study by the Institut de Neurociències of the UAB (INc-UAB) demonstrates in animal models the benefits of vorinostat after having suffered a stroke. The drug, used in humans to treat cutaneous T-cell lymphoma, has been proved to mitigate brain injuries and help in restoring brain tissue.

Ischemic stroke is the second leading cause of death worldwide and occurs when blood flow cannot reach the brain due to an obstruction.

Bayer announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy. BAY 2927088 is an oral, reversible tyrosine kinase inhibitor (TKI) that potently inhibits mutant human epidermal growth factor receptors 2 (HER2),

Lung cancer is not the most common form of cancer, but it is by far the deadliest.

Despite treatments such as surgery, radiation therapy and chemotherapy, only about a quarter of all people with the disease will live more than five years after diagnosis, and lung cancer kills more than 1.8 million people worldwide each year, according to the World Health Organization.

Pfizer Inc. (NYSE: PFE) announced that the European Commission (EC) has granted marketing authorization for VELSIPITY® (etrasimod) in the European Union to treat patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.