FDA Approval

Azafaros presents its promising drug, nizubaglustat, for untreatable rare neurological disorders, backed by major funding and late-stage clinical trials.

Fresh off its 'Fierce 15' win, Recludix Pharma and its billion-dollar Sanofi pact take the spotlight with a novel oral drug for inflammatory diseases.

Azitra will present its novel AI-driven skin therapies for rare diseases at Biotech Showcase, seeking crucial funding and partnerships to advance its pipeline.

A first-in-class antibody shows a strong safety profile, de-risking development and offering a new, targeted approach for severe autoimmune diseases.

A routine prenatal screen can now detect a virus that causes birth defects, offering a path to prevention and sparking calls to change medical guidelines.

A groundbreaking Duke trial combines a novel device to lure cancer cells with targeted immunotherapy, offering real-time tracking and new hope for patients.

With its rare disease drug JURVIGO® in late-stage trials, Mezzion appoints a veteran leader to navigate the path to approval and commercialization.

A validated dry EEG headset provides a scalable, objective measure of brain plasticity, promising to accelerate drug discovery for Alzheimer's and depression.

Internationally recognized fetal surgeon Dr. Jena Miller joins CHOP, signaling a new chapter in minimally invasive procedures for the world's most fragile patients.

Processa Pharmaceuticals completes enrollment for a pivotal analysis of its NGC-Cap therapy, offering new hope for a safer, more effective cancer treatment.

With promising 3-year vision data and a manufacturing breakthrough, Lineage is pioneering 'off-the-shelf' cell therapies for blindness, diabetes, and more.

Formerly Cybin, Helus Pharma's Nasdaq launch marks a pivotal shift towards commercializing its breakthrough depression and anxiety drug pipeline.

A new 10,000-sample 'skin library' collected by painless stickers aims to slash drug development time and costs for biopharmaceutical partners.

With a key drug decision looming and a refocused strategy, Rocket Pharmaceuticals faces a critical audience at the J.P. Morgan Healthcare Conference.

All eyes are on GH Research ahead of a crucial Jan. 5 announcement on the FDA status of its fast-acting drug for treatment-resistant depression.

Genelux appoints Dr. Jason Litten as CMO, a strategic move to steer its promising oncolytic virus, Olvi-Vec, through crucial late-stage cancer trials.

Hoth Therapeutics files patents for a therapy to mitigate severe skin reactions from cancer treatments, a strategic move that could transform patient care.

BridgeBio Pharma hosts a key investor webinar on its oral drug infigratinib for achondroplasia, setting the stage for crucial trial results in Q1 2026.

Ultragenyx's stock plummets after its Osteogenesis Imperfecta drug fails to reduce fractures, a major blow for patients and a test of the firm's pipeline.

INOVIO's promising non-surgical treatment for a rare disease faces FDA scrutiny, casting doubt on its fast-track approval and impacting patient hopes.