The FDA granted priority review and set a May 1, 2027 PDUFA date, and appears likely to convene an advisory committee for the first Huntington's disease gene therapy BLA, which rests on a small, externally controlled dataset.
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Democratic control of the Senate reshapes the outlook for health policy, Medicare Advantage, and insurance legislation heading into the next session.
The accelerated approval makes Voceryq the first DNA-based immunotherapy cleared for recurrent respiratory papillomatosis, sending Inovio shares sharply higher.
The setback hits Edgewise's Becker muscular dystrophy program, a condition with no approved treatments and no clear path forward in the study.
Executives kept mRNA cancer therapies central to the pipeline while acknowledging a missed endpoint and mixed lung and breast cancer signals.
The debt-laden cancer drug developer will hold a court-supervised auction after striking a forbearance deal with creditors.
The death in rural northern New York underscores gaps in vaccination coverage amid national measles resurgence.
The filing would put Compass in a tight race to win the first US approval of a psychedelic medicine, against a nonprofit competitor.
The third US bird flu fatality overall highlights ongoing H5N1 spillover risk from dairy herds to farm workers.
The hold, tied to toxicity seen in mice, halts development of the epilepsy candidate and cut Bright Minds' share price in half.
The label expansion moves the BTK inhibitor earlier in the treatment sequence for chronic lymphocytic leukemia and small lymphocytic lymphoma patients.
Personalized mRNA cancer vaccine progress is colliding with upcoming biosimilar pressure on Merck's Keytruda, reshaping oncology competition.
The result adds to evidence that tailored mRNA vaccines can reduce relapse in high-risk melanoma patients.
The approval extends the nonsteroidal MRA's use to a patient group with few kidney-protective options.
The final guidance aims to standardize potency testing for cell and gene therapies, a persistent source of regulatory friction for developers.
The think tank urged seniors to compare plans during open enrollment given wide variation in costs across Part D offerings.
Five Bay Area labs compared psilocybin's durable effects on anxiety and depression-like behavior in mice with limited results, raising questions for clinical claims.
Tentative approval for Dr. Reddy's generic version of lenacapavir is a key step toward expanding global access to the prevention drug.
The close advisory vote could change which peptides pharmacies may compound, affecting a fast-growing gray market.
On an earnings call, Vertex leadership expressed conviction in the IgA nephropathy medicine's coming launch, seen as important to the company's future.
The designation could speed development of the Korean biotech's candidate for a cancer with limited treatment options.
The resumption of substantive review puts the Korean biotech's rivoceranib back on track for a US decision next spring.
Clinical investigators wrote to the agency backing Capricor's cell therapy for Duchenne muscular dystrophy cardiomyopathy ahead of a regulatory decision.
Scientists and ethicists are rethinking the risks and benefits of mitochondrial replacement technology as its use expands globally.
AstraZeneca will redeploy resources under a new restructuring program, while Alnylam sold off on drug concerns and Regeneron beat expectations.
The delay pushes back a decision on expanding the pyruvate kinase activator into sickle cell disease.
Two separate research efforts advance focused ultrasound as a way to boost drug delivery in hard-to-treat tumors; IMGT plans US confirmatory trials for pancreatic cancer.
The company reported a regulatory milestone for its ketamine formulation targeted at suicidal depression.
Investors and physicians await mid-stage results for the experimental oral therapy positioned to rival Sanofi and Regeneron's Dupixent.
Payer and pharmacy benefit dynamics may limit uptake of biosimilar versions of Merck's blockbuster even as its patent cliff nears.
Rising funding for cell therapy contrasts with safety concerns that could slow pharmaceutical companies' expansion into autoimmune indications.
RBC noted rising uptake of the sickle cell gene therapy as investors watch for data from the company's next programs.
The Hong Kong biotech's oncology candidates are attracting international interest as Chinese drugmakers gain prominence in global development.
The deal deepens Zuellig's clinical trial supply capabilities in the Chinese market.
The FDA and CMS are exploring coordination with states on regulating AI in health care and shielding clinicians from related liability.
Employees remain uneasy less than two weeks after a round of job cuts at the Boston health system.
Four big drugmakers allegedly paid tens of millions to intermediaries to supply medicines to Russian government entities, per STAT's pharma newsletter.
An analysis questions whether AI-driven discovery is improving the quality of development candidates or simply enlarging them.
The roundup covers FDA tentative approval of a generic HIV prevention medicine and Lonza's purchase of US manufacturing sites.
Researchers identified regulatory mechanisms that may open new targets for overcoming drug resistance in tumors.
Separate academic efforts explore mRNA-based and intracellular approaches to treating pancreatic cancer and other solid tumors.
The mouse study suggests dietary composition can shape immunotherapy efficacy, though human relevance is unproven.
The startup is working on personalized cancer vaccine approaches, which an executive cautioned remain experimental rather than established cures.
An opinion piece warns that proposed program reductions could harm access to cancer care.
Contributors argue leading drivers of death differ along political geography, with implications for public health policy.
A Guardian piece examines what more effective cancer therapies mean for the lived experience of patients receiving them.
A market research report forecasts continued growth in gene therapy spending over the next decade.