Astellas Pharma Inc. has announced that its Phase 2 GLEAM trial evaluating zolbetuximab in combination with gemcitabine plus nab-paclitaxel for metastatic pancreatic adenocarcinoma did not meet its primary endpoint of…
AstraZeneca and Daiichi Sankyo have announced promising Phase 3 trial results for Datroway, their investigational antibody-drug conjugate (ADC) targeting triple-negative breast cancer (TNBC). The trial demonstrated that Datroway significantly slowed…
Bayer AG and its subsidiary BlueRock Therapeutics LP have announced encouraging 36-month follow-up data from the Phase I exPDite clinical trial of bemdaneprocel (BRT-DA01), an investigational cell therapy aimed at…
Capsida Biotherapeutics has suspended a clinical trial of its experimental gene therapy after the first child to receive the treatment died, sending shockwaves through the rare disease and gene therapy…
Serina Therapeutics, a clinical-stage biotechnology company, has announced a major development in the treatment of Tardive Dyskinesia (TD) by advancing its innovative POZ-VMAT2i candidate into clinical development. This move, reported…
Cogent Biosciences has announced new clinical trial results indicating that its drug BEZUCLASTINIB offers promising benefits for patients with indolent systemic mastocytosis (ISM), a rare and chronic disorder characterized by…
Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they…
The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide is effective in the treatment…
Humans have been using tobacco as a drug for more than 12,000 years. It wasn't until the early-20th century that the link between tobacco and lung cancer (and other health problems)…
Approximately one and one half million people are affected by ulcerative colitis (UC) worldwide. It is an inflammatory bowel disorder with, as of yet, no known cause. There are, however,…
Imagine that your loved one is diagnosed with a rare disease. The first thing you do is to go to the internet to understand what you can about this disease…
By: Harsha K Rajasimha, Ph.D., Founder and CEO of Jeeva Clinical Trials Challenges Unique to Cell and Gene Therapies (CGTx) Clinical Trials Cell and gene therapies (CGTx) have emerged as…
Rare disease drug development can be challenging. Countries globally developed programs to incentivize innovation and development in this space. One of these initiatives is the designation of orphan drugs, or…
Clinical-stage biopharmaceutical company Savara Inc. was founded on a desire to develop innovative therapies for people living with rare respiratory diseases. The company's work over the past few years centers…
Ocugen, Inc., a biotechnology company based in Malvern, Pa. recently reported to BioSpace that DSMB gave a favorable review of its Phase ½ ArMaDa clinical study of OCU410. Approximately 10…
Long COVID is a phenomenon emerging from the global spread of SARS-CoV-2, a strain of coronavirus causing COVID-19. Seven million people died from COVID-19, though this is considered a serious…
Mirum Pharmaceuticals has long been a leader in the rare liver disease space, driving forward with a mission of empowering patients and families, and identifying novel therapeutic interventions to help…
Three years after the U.S. Food and Drug Administration (FDA) first placed a full clinical hold on Larimar Therapeutics’ Phase 2 clinical study, the hold has officially been lifted. The…
Artificial intelligence (AI) is changing the way we live. It seems rare to find an industry where AI has not made at least a preliminary impact. In politics, AI-generated cartoons…
An estimated 25% of people with Clostridioides difficile (C. diff) infections experience recurrence following successful antibiotic intervention. After three or more recurrent infections, the risk of developing another infection sits…
People with idiopathic pulmonary fibrosis (IPF) already have two FDA-approved treatment options: Ofev (nintedanib) and Esbriet (pirfenidone). But biotech company Agomab Therapeutics ("Agomab") is working to introduce another potential therapeutic…
You might have heard of Orphan Drug designation - and maybe even Rare Pediatric Disease designation. But have you heard of Regenerative Medicine Advanced Therapy (RMAT) designation? Described in Section…
The Phase 1b/2a RESOLVE clinical study set out to identify how safe and effective EP-104GI is for people living with a chronic immune-mediated disease called eosinophilic esophagitis (EoE). People with…
Just about one month ago, clinical-stage biotechnology company AnaptysBio announced the availability of positive top-line results from two Phase 3 clinical studies: GEMINI-1 and GEMINI-2. Both studies sought to assess…
RINVOQ (upadacitinib) is a Janus Kinase inhibitor that is currently approved to treat a variety of conditions: moderate to severe atopic dermatitis, active psoriatis arthritis, moderate to severe rheumatoid arthritis,…
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