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Innovation drives CROs to win new business, but unchecked customization creates complexity that undermines reproducibility, weakens renewals and leads to turnover—making standardization a strategic necessity, not a constraint.
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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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Read our takes on the biggest stories happening in the industry.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
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Despite tolerability concerns, nomlabofusp’s overall efficacy represents a “large win” for Larimar, according to analysts at William Blair, who lauded the therapy’s functional benefits.
Already approved in Japan, China and other Asian countries, Crystalys’ dotinurad works to lower serum uric acid levels.
Without naming Aurinia Pharmaceuticals, the CDER director in a now-deleted LinkedIn post claimed that for lupus nephritis, companies have not conducted post-approval studies “to demonstrate a benefit on hard clinical endpoints.”
Companies are moving from using AI for distinct operations to applying the technology for control and optimization of the whole production process.
Reshoring generic pharmaceutical production is essential in today’s era of geopolitical instability and heightened awareness surrounding national health security. And it is possible—if done right.
After Emma Walmsley steps down as GSK CEO in January, Vertex Pharma’s Reshma Kewalramani will be the sole female CEO at a top-20 pharma company. Still, there are many prominent women in pharma that could someday break through again.
Chief Commerical Officer Luke Miels will replace outgoing GSK CEO Emma Walmsley at the U.K. pharma next year.
The AAV pullback comes amid Biogen’s aggressive cost-cutting campaign, which put some 1,000 jobs on the chopping block with the goal of generating $1 billion in savings by 2025.
Applied Therapeutics has yet to confirm whether the study, posted on Clinicaltrials.gov on Thursday, means it has indeed aligned with the FDA on govorestat’s development.
The centerpiece of the acquisition is petosemtamab, Merus’ bispecific antibody targeting EGFR and LGR5, which in May demonstrated best-in-class potential for head-and-neck cancer.