News
After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
FEATURED STORIES
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
THE LATEST
With a Friday advisory committee meeting looming, the sole indication for Intercept Pharmaceuticals’ Ocaliva appears to be at risk as the regulator flags issues regarding its post-marketing results.
In an effort to build its commercial capacity, Moderna on Thursday announced it is lowering research and development spending, while pushing back its target for breaking even by two years to 2028.
When doses were increased rapidly in a Phase I study, patients on Roche’s investigational oral GLP-1 receptor agonist experienced nausea, vomiting, constipation, diarrhea, as well as abdominal distension.
In this episode, Lori and guests discuss practical approaches regarding artificial intelligence and investor and industry confidence in its current state.
For the first eight months of 2024, California had the most job postings live on BioSpace—37.7% more than Massachusetts. Last year, Massachusetts ranked No.1 for the same time period.
An appreciation for practicality, independent thinking and patient care can help disrupt the bureaucracy of Big Pharma.
Join our webinar to explore strategies, best practices, and the evolving landscape of QARA in today’s dynamic market.
On the heels of Terns’ positive Phase I results that analysts compared with Lilly’s and Pfizer’s weight loss pills in development, Novo Nordisk showcased more details about its own oral candidate.
No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmeceuticals’ recently approved sickle cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.
Healthcare players are pointing fingers amid regulatory crackdowns on pharmacy benefit managers, but proposed reforms wouldn’t address a dearth of competition in the larger market.