News
The company’s intein-based technology is initially aimed at Stargardt disease, a type of macular degeneration.
FEATURED STORIES
Facing declining valuations and funding challenges, public biotechs like bluebird bio are going private to restructure, reduce regulatory burdens and refocus on long-term growth.
Johnson & Johnson has been fighting thousands of lawsuits over its now-discontinued talc products for 16 years. A pending judge’s ruling could finally put the issue to bed once and for all.
As we reflect on five years of COVID-19, it’s clear that the impacts are still unfolding. The life sciences—and we as individuals—will never be the same again.
Job Trends
Pfizer Inc. announced that Andrew Baum, M.D., will join the company as Chief Strategy and Innovation Officer, Executive Vice President.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
When talking to some of the most impressive women in biopharma, the conversation inevitably turned to what these women wanted other entrepreneurs to know. Here’s the best of the best of that advice.
THE LATEST
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
The restrictions on Novavax’s vaccine could portend changes at the FDA. Commissioner Marty Makary suggested last week that the agency could update its vaccine approval guidelines “in the coming days.”
Analysts at BMO Capital Markets said in a weekend note that a non-invasive blood test could help boost uptake of Alzheimer’s disease therapies.
The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain.
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
While the Trump administration has painted the jettisoning of staff and regulations as good for business, there are multiple reasons it’s unlikely to work out that way.
The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
The FDA also approved the use of Zynyz as a monotherapy for patients with squamous cell carcinoma of the anal canal who are intolerant to platinum chemotherapy or whose disease has progressed.
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.
Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss.