Gene Therapies

Disc Medicine's promising EPP drug hits a major roadblock as the FDA seeks more data, delaying hope for patients and setting up a high-stakes trial.

The firm reports a $195M cash reserve but a wider net loss as it nears crucial data readouts for its novel depression and anxiety drug candidates.

After a major trial failure tanked its stock, Rezolute pins its hopes on a second drug program and a crucial FDA meeting to chart a path forward.

Celcuity adds Seagen commercial veteran Charles Romp to its board, a strategic move signaling its pivot from R&D to market readiness for gedatolisib.

With strong Phase 1 data and key FDA backing, Trethera's TRE-515 is advancing as a 'pipeline-in-a-product' for a wide range of diseases.

With a new chair from Genmab and a board of industry titans, BioNJ is poised to bolster New Jersey's status as a global biotech powerhouse.

A new oral drug, IMSB301, offers hope for Aicardi Goutières Syndrome, a devastating rare disease with no cure, in a pivotal new clinical trial.

Clinical-stage biotech BioVie announces its novel anti-inflammatory drug, bezisterim, will be featured at a key psychiatric conference for its potential.

The French biopharma giant posts strong 2025 results and forecasts over 13% growth, fueled by a booming pipeline and unexpected market advantages.

As Caroline Dorsa retires, Biogen elects Dr. Maria Freire as its new chair, a strategic move placing deep scientific expertise at the helm for a critical period.

Following key FDA progress for its oral osteoporosis drug, Entera Bio's CEO and board members are buying up company stock, signaling deep confidence.

At a key summit, Candel Therapeutics will showcase how its oncolytic virus, CAN-3110, uses biomarkers to fight one of the deadliest brain cancers.

With a 135% revenue surge and a key FDA submission for its kidney transplant drug, Hansa Biopharma is poised for a pivotal year of expansion.

Celularity Inc. has secured a $12.2M cash injection by selling tax losses, a move set to accelerate its cutting-edge longevity and regenerative medicine pipeline.

With a new CEO and stunning clinical data, BOOST Pharma advances a potential game-changer for children with the rare genetic disorder Osteogenesis Imperfecta.

Aardvark Therapeutics is expanding its Phase 3 trial for a novel Prader-Willi Syndrome drug to include children as young as seven, targeting a key unmet need.

Belgian biotech Agomab Therapeutics secures $200 million in its Nasdaq debut, fueling its mission to develop therapies for debilitating fibrotic conditions.

With a promising spinal cord injury drug nearing late-stage trials, NervGen appoints interim leader Dr. Adam Rogers as permanent CEO to guide the breakthrough.

Australian biotech Immutep reaches a key milestone in its Phase III trial for a novel lung cancer therapy, a major step in a multi-billion dollar market.

A new cancer drug using DNA fragments to deliver chemotherapy more safely has been cleared for US trials, offering hope for less toxic treatments.