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The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.

AstraZeneca will push the pill, elecoglipron, into a comprehensive late-stage program that will test the drug as a monotherapy, as part of a combination regimen and for several indications.

As next-generation antibody-drug conjugates reshape cancer care, digital pathology and artificial intelligence are transforming how HER2 is measured. The advances aim to help clinicians identify low and ultra-low expressors, match patients to the right therapies and make more precise treatment decisions.

As the field grows rapidly, companies are luring people from other nuclear industries and tapping the expanding educational talent pipeline, but are constrained by a steep learning curve and the value of real-world experience.

The deal gets Lilly access to Orna’s in vivo CAR T technology. The biotech’s lead asset, which has yet to start clinical testing, is focused on B cell–driven autoimmune diseases.

Analysts, investors and scientists are eager for Biogen’s 2026 BIIB080 readout. Even if successful, executives warn that there are many more steps before the Alzheimer’s therapy could reach the market.

Novo Nordisk has sued Hims for allegedly violating patents protecting semaglutide, seeking potentially “hundreds of millions” in damages, John Kuckelman, the pharma’s general counsel, said. The wellness platform pulled its version of the drug just days after launching it.

The gene therapy uses an AAV vector to restore healthy levels of the alpha-galactosidase enzyme, which is rendered dysfunctional in patients with Fabry disease, leading to the toxic build-up of lipids in cells.

The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing manufacturing issues. Ultragenyx Pharmaceutical resubmitted its application ten days ago, expecting a six month review time.

The FDA underwent significant changes during the first year of the second Trump administration, directly affecting business risk and opportunity. Understanding key 2025 trends will be critical to developing regulatory strategies and maximizing opportunities for success.

With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.

Sales of Johnson & Johnson’s esketamine-based nasal spray jumped in the fourth quarter last year, priming the pump for a suite of other pharmas, including AbbVie, champing at the bit with their own psychedelics.

Data Presented at Epilepsy Foundation Pipeline Conference Demonstrated Significant Reduction in Epileptiform Activity: KIO-300 is the active pharmaceutical ingredient of KIO-301, Kiora’s ongoing Phase 2 clinical program in vision restoration in patients with gene mutation-agnostic Retinitis Pigmentosa

The Company invites individual and institutional investors, as well as advisors and analysts with an interest in Phio Pharmaceuticals, to attend the live, interactive presentation and one-on-one meetings

Cancer Research UK’s Centre for Drug Development is using TrialAssure LINK AI to support medical writing in CRUK-sponsored studies

CDMO continues on path to commercial growth, expansion

New specialized treatment site expands Paradigm’s capabilities for psychiatric clinical trials and studies evaluating investigational psychedelic-assisted therapies

Key Highlights: · Luxna and BioSpring have extended their existing agreement, enabling the manufacture of next-generation oligonucleotides incorporating Luxna’s GuNA™ amidites, in addition to AmNA™, scpBNA™, and 5′-CP™. · GuNA™ further expands the range of Luxna’s proprietary Xeno Nucleic Acid (XNA) chemistries already available through BioSpring’s oligonucleotide manufacturing services. · GuNA™-modified oligonucleotides are designed to provide enhanced nuclease resistance, improved target binding, reduced immunotoxicity and neurotoxicity; properties that provide key advantages for developing better efficacy and safer therapies targeting neurodegenerative diseases.