Olivia Vizier

RNA interference (RNAi), sometimes called gene silencing, is an approach to therapeutics in certain diseases that are caused by the production of abnormal proteins or an overproduction of proteins, which can be treated by turning off or silencing the gene. One of the companies working on the cutting edge of this field is Pasadena, California-based Arrowhead […]

The first wave of a new class of anti-aging drugs have begun human testing. These drugs won’t let you live longer (yet) but aim to treat specific ailments by slowing or reversing a fundamental process of aging. The drugs are called senolytics—they work by removing certain cells that accumulate as we age.

Rezafungin is a novel molecule in the echinocandin class of antifungals, which its developer Cidara believes will be an improvement among current fungal infections treatments. Rezafungin is being developed as a first-line treatment of candidemia and invasive candidiasis.

Zach, his family and other people suffering from rare diseases will be guests of honor at an event commemorating Rare Disease Day on Leap Day – the rarest day of the year – at Carnegie Hall. Rocket Pharmaceuticals, Inc., a company that researches genetic solutions for diseases like Zach’s, is organizing the event for the

Q: Your experimental protocol was given as third-line treatment, is that correct? Most patients were on their third line of treatment, but inclusion criteria were beyond first-line standard of care.

A number of ingestible technologies have been developed for the diagnosis of gastrointestinal (GI) diseases, including the use of conventional tools, such as endoscopy, radiological imaging, or biochemical tests. Ingestible diagnostics are particularly useful for the exploration of the small bowelii, since gastroscopy can only reach a limited area of the small intestine and alternative

“Their Phase I data was stellar, great Phase I, and then they had this Phase II that failed primarily because they added the wrong drug,” Asher Nathan, CEO and co-founder of NeoTX, told Endpoints News as he unveils $45 million in new financing.

A look into Cue Biopharma’s engineering-centric approach to in vivo tumor antigen specific T cell activation. Late last month, Cue Biopharma shared preclinical and translational data supporting the therapeutic potential of CUE-101 in HPV16-related malignancies.

French companies OSE Immunotherapeutics and MAbSilico are teaming up to drive the discovery of new antibody drugs using artificial intelligence. The three-year partnership will use artificial intelligence, or AI, in six programs designed to accelerate the development of therapeutic monoclonal antibodies (mAbs) that can target cancer and autoimmune diseases.

Jersey City-based Scynexis Inc., a biotechnology company delivering therapies for difficult-to-treat and often life-threatening infections, said Thursday it completed patient enrollment, ahead of schedule, in its global Phase 3 VANISH-306 study to evaluate the safety and efficacy of oral ibrexafungerp as a treatment for women with vulvovaginal candidiasis (VVC), commonly referred to as vaginal yeast infection.

Immunomedics has developed an ADC called sacituzumab govitecan, which directly delivers chemotherapy SN-38 to TNBC tumours, which highly express Trop-2 and represents an unmet need within a highly treated cancer type. The company’s chief commercial officer Brendan Delaney explains the drug’s efficacious mechanism of action in this tumour type, as well as noting its success

Updated interim data for Adverum’s intravitreal wet age-related macular degeneration gene therapy bolstered the company’s case that the product could compete with less convenient subretinal gene therapies for the indication.

Warren Huff, Reata’s president and CEO, discussed the clinical progress of omaveloxolone and bardoxolone, as well as the company’s approach to drug development, before the stock surge in an exclusive interview with GEN Edge during the recent J.P. Morgan conference in San Francisco . . .

Dr. Lawrence Blatt, CEO, Aligos Therapeutics, discusses his company’s research around antisense oligonucleotides as potential therapies for chronic hepatitis B and other diseases of the liver. Despite an available vaccine, the incidence of chronic hepatitis B (HBV) remains high — the largest prevalence of any chronic virus that causes morbidity and mortality — in some

Thorsten Stafforst found his big break at the worst possible time. In 2012, his team at the University of Tübingen in Germany discovered that by linking enzymes to engineered strands of RNA, they could change the sequences of messenger RNA molecules in cells. In essence, they could rewrite the genome’s instructions en route to making

Awareness days and months occur throughout the calendar year to generate a greater understanding about various diseases and help increase visibility about the unmet needs related to these conditions. For many advocacy groups, these activities drive awareness and highlight rare disease states or disorders with great unmet needs.

Dean Scott could hardly see two years ago, but the Spirit River, Alta., carpenter says an experimental drug helped him regain some of his vision and added colour to his life. Scott, 45, first noticed problems with his eyes when he was a child and lost his peripheral vision.

In August of last year, Alkahest reported the results from a six-month trial that saw 40 patients with mild to moderate Alzheimer’s disease infused with a proprietary human plasma “fraction”. It appeared to arrest their expected mental decline.

Frustrated with the slow pace of drug discovery and desperate to help their loved ones, untrained family members take the work into their own hands by starting biopharma companies. But few such companies have been successful.

Valneva’s vaccine, VLA15, encourages the body’s immune system to make antibodies to defend against the bacteria that cause Lyme disease. The FDA gave VLA15 fast-track designation in 2017, and the vaccine is currently in Phase 2 clinical studies.

In drug development, assets are often aimed at a specific disease target or pathway, similar to a bullet fired from a gun. The idea of going after one precise target though, may not be the best solution for treating the totality of diseases, though.