PDE4-B Inhibitors: A Promising Target for Idiop...

FDA Approves AstraZeneca’s Imfinzi for Biliary Tract Cancer Imfinzi, a checkpoint inhibitor developed by AstraZeneca, has been approved by the FDA as the first immunotherapy for biliary tract cancer (BTC), a rare and aggressive form of cancer with few treatment options. Imfinzi (durvalumab) has been approved by ...

There are a few major unmet needs in the primary sclerosing cholangitis (PSC) treatment market, which include a lack of effective therapies, proven surveillance strategies, tools for early detection of malignancies, impact on quality of life (QoL), and surrogate endpoints in primary sclerosing cholangitis clinical ...

Aktis Oncology Raises USD 84 Million To Advance Novel Targeted Alpha Radiopharmaceuticals Aktis Oncology has raised an additional USD 84 million in its Series A round, adding to the USD 72 million raised last year to help bring its radiopharmaceuticals to market. The extension to the first round included Merck's...

There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical...

Congenital adrenal hyperplasia (CAH) is a rare inherited autosomal recessive disorder defined by a lack of one of the enzymes required to produce specific hormones. In the United States and Europe, the most prevalent type of CAH, 21 hydroxylase deficiency, affects around 1:10,000 to 1:15,000 people. The estimated C...

Dystrophic epidermolysis bullosa (DEB) is a common skin disease with a chronic history and low healing rates. Despite current management therapies, the disease has the potential to become severe and devastating for many patients over time. Dystrophic epidermolysis bullosa is classified into six types: dominant dyst...