Gene Therapies

With its lead muscular dystrophy drug in late-stage trials, Edgewise Therapeutics heads to the JPM conference with high stakes for its pipeline and stock.

With its first drug for a rare disease now on the market, Stealth BioTherapeutics bets its future on a deep pipeline targeting mitochondrial dysfunction.

Early trial data for IKS014 reveals a high clinical benefit rate in pre-treated oesophageal cancer, offering a new beacon of hope for a hard-to-treat disease.

With stunning clinical data for its miv-cel therapy, Kyverna is set to outline a new era for autoimmune treatment at the J.P. Morgan conference.

With blockbuster sales from VYJUVEK, Krystal Biotech heads to J.P. Morgan with investors eager for news on its global growth and ambitious pipeline.

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

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

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

Fresh off a major drug approval, Insmed's upcoming J.P. Morgan talk is under scrutiny as investors eye pipeline updates and acquisition potential.

Ironwood Pharma forecasts a massive profit jump by lowering its top drug's price, a bold move as it advances its rare disease pipeline for future growth.

Emmaus Life Sciences outsources its sickle cell drug Endari® in North America to focus on global growth, as NeoImmuneTech gains a key U.S. market foothold.

The biopharma investment giant has now acquired 100% of the royalty stream for Roche's blockbuster SMA drug, a strategic move years in the making.

With a novel therapy from newborn heart cells, Secretome Therapeutics heads to J.P. Morgan to showcase its progress against intractable heart failure.

With promising survival data and a pivotal Phase 3 trial underway, Imunon's localized immunotherapy could finally change the game for ovarian cancer.

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.

As its cash runway shortens, Longeveron's novel stem cell therapy gets a major boost with selection for a prestigious Alzheimer's health moonshot.

With a promising schizophrenia drug stalled by FDA requirements and finances tight, Reviva Pharmaceuticals' upcoming presentation is more than just a talk.

Early data shows QurAlis' QRL-101 successfully engaged its intended target in ALS patients, a key step for a novel drug aimed at quelling neuronal storm.

Mirum raises over a quarter-billion dollars to acquire Bluejay Therapeutics, targeting a high-potential liver disease drug and signaling major growth.

A new oral drug, cadisegliatin, enters a major clinical trial in the UAE, aiming to help Type 2 diabetes patients struggling with insulin therapy.