Regenxbio shares new Duchenne gene therapy data ahead of big readout next year
Regenxbio has biomarker data on the first patient younger than 4 years old dosed with its Duchenne muscular dystrophy gene therapy, showing that the patient expressed levels of a shortened version of a...
View ArticleAhead of FDA decision, Alnylam faces the big question: Can it make money?
Despite pioneering RNAi drug development and getting four therapies approved, Alnylam still isn’t profitable. That may change as soon as next year. The FDA is expected to decide by Sunday whether to...
View ArticleCargo Therapeutics scraps R&D pipeline, axes bulk of workforce as it looks...
As Cargo Therapeutics looks for a permanent solution to its troubles, the biotech is dropping its remaining clinical programs and laying off the majority of staff in a second round of cuts. The San...
View ArticleTrump’s DOJ rejects effort to shed mifepristone restrictions
President Donald Trump's new administration defended Biden-era restrictions on the abortion pill mifepristone, marking one of the first times it’s voiced its stance in the ongoing lawsuits over the...
View ArticleGilead stock slips as HHS denies reports of HIV funding cuts
Gilead Sciences' stock {$GILD} dropped by more than 3% on Wednesday afternoon amid reports that the US will slash billions in CDC funding for anti-HIV prevention efforts. An HHS spokesperson told...
View ArticleNovartis touts Zolgensma data in older children
Novartis released two Phase 3 datasets on Wednesday that show a new version of its gene therapy Zolgensma led to motor function improvements in patients with spinal muscular atrophy. The company hopes...
View ArticleHow Akido Labs uses AI to run medical visits, from intake to diagnosis
For cardiologist Haritha Alla, a typical day in her Los Angeles-area clinic is ruled by mundane data collection. Patients struggle to fill out intake forms and tend to leave them unfinished. Much of...
View ArticleSanofi to pay $600M upfront for Dren Bio’s bispecific B cell depleter
Sanofi is buying a B cell-depleting antibody developed by Dren Bio as a therapy for cancer and autoimmune diseases. The pharma said on Thursday that it will pay $600 million in ...
View ArticleParatek to acquire Optinose and its nasal spray Xhance
Paratek Pharmaceuticals is expanding beyond antibiotics and infectious disease by paying up to $330 million to acquire Optinose and its drug-device combination for chronic rhinosinusitis (CRS). Paratek...
View ArticleMonte Rosa reports first data for molecular glue program
Monte Rosa Therapeutics reported Phase 1 data Thursday morning for a Novartis-partnered program that the companies believe could change the treatment paradigm for some immune-mediated diseases. Though...
View ArticleIllumina's CEO evolves strategy with multiomics push
SAN DIEGO — At the headquarters of Illumina last week, CEO Jacob Thaysen bounded up a coiled staircase that mimics a DNA strand. The architectural flourish at the company’s campus is a representation...
View ArticleUnitedHealth's Optum Rx to change its payment models
UnitedHealth Group's pharmacy benefit manager Optum Rx is making changes to its payment models. Optum Rx will tweak its payment models in full force by January 2028 to more closely reflect the high...
View ArticleElevation Oncology stops work on ADC, lays off workers; AstraZeneca’s CEPI...
Plus, news about Century Therapeutics, Akebia Therapeutics, Eyenovia, Betaliq, Alvotech, Genenta Science, Aadi Bioscience, and Exicure: Elevation Oncology drops ADC, lays off 70%: The Boston-based drug...
View ArticleMerck to lay off 163 workers in Pennsylvania; Delpharm gets $60M from...
Welcome to Endpoints News’ manufacturing briefs, where we bring you essential news on new builds, collaborations, recalls and more. Merck plans to lay off 163 staffers as it closes its facility in...
View ArticleVisen Pharmaceuticals prices $86M Hong Kong IPO
Visen Pharmaceuticals, a biotech developing much of Ascendis Pharma’s endocrine-related disease pipeline for the China market, will go public on the Hong Kong Stock Exchange on Friday. The company...
View ArticleGoogle is building AI healthcare helpers
It feels like people have gotten a lot more comfortable with using AI day to day. Every spring, I go to Google’s office in New York to see how the tech giant’s applying its technology ...
View ArticleSV Health Investors nabs $250M to fund dementia-focused biotechs
SV Health Investors has added $250 million to its hunt for new therapeutics for dementia, Alzheimer's and other neurodegenerative diseases. The London-based life sciences investor secured that amount...
View ArticleCalley Means joins White House as a special government employee
A close ally of HHS Secretary Robert F. Kennedy Jr. is joining the administration as a special government employee, according to a source familiar with the decision. Calley Means, an entrepreneur and...
View ArticleNovo Nordisk lays out new structure for early R&D unit
Novo Nordisk is reorganizing its research and early development unit in an effort to “future-proof the organization” and “significantly increase” the number of projects it brings into clinical trials,...
View ArticleMany rare disease drugs rely on just one trial for approval, FDA authors find
New research published by FDA authors found that 67% of non-oncologic rare disease marketing applications approved between 2020 and 2023 used only one clinical trial plus confirmatory evidence to...
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