Gene Therapies

A $10M investment from BVF Partners propels Entera Bio’s revolutionary oral osteoporosis drug into a pivotal Phase 3 trial, a major step for patients.

A stark new benchmark study reveals poor survival in late-stage GvHD, just as MaaT Pharma’s microbiome therapy shows dramatically better outcomes.

The biopharma firm touts transformative drug data ahead, but with cash for only month-to-month operations, its future hangs by a precarious thread.

With its lead drug for a rare pediatric liver disease entering late-stage trials, NorthSea Therapeutics adds a veteran drug developer to its board.

A novel drug aims to break down resistance in hard-to-treat lung and bile duct cancers, with key data set for a major oncology conference.

A key U.S. patent extends protection for a promising CRPS-1 drug, paving the way for a potential first-in-class treatment for the debilitating condition.

A new partnership provides early access to an investigational drug for FAP, offering a potential non-surgical therapeutic option for the first time.

A new, highly selective oral drug for the most common form of dwarfism has been cleared for U.S. trials, offering hope for a more convenient treatment.

A startling new study reveals a sharp rise in diagnosed anxiety, highlighting a growing public health crisis and an urgent need for treatment innovation.

Sangamo pushes a promising Fabry disease therapy toward the FDA while a cash crunch forces a strategic pivot to brain-targeting neurological cures.

Aurinia Pharmaceuticals' deal for Kezar Life Sciences hinges on zetomipzomib, a promising but risky drug poised to tackle a rare autoimmune liver disease.

A $15M grant fuels a revolutionary therapy for a fatal childhood disease, turning patients' own cells into 'living biofactories' to fight MPS I.

Neumora reveals promising data for Alzheimer's and schizophrenia drugs, but a delay in its obesity program highlights the high-stakes world of biotech.

With a fortified balance sheet and promising clinical data, Monopar Therapeutics is advancing its lead drug, ALXN1840, toward a pivotal NDA filing.

After its pivotal trial for ersodetug missed its primary endpoint, Rezolute gets a rare nod from the FDA to submit full data, igniting new hope.

A landmark partnership is scaling a 'one-drug, many-diseases' therapy, revealing how logistics, not just labs, are key to the future of medicine.

New data shows Dizal's ZEGFROVY achieved an 81.3% response rate in a lung cancer subgroup with limited options, signaling a potential new first-line standard.

A French biopharma's leap in synthetic lipid production de-risks its lead drug, eyes a vast sepsis market, and creates new commercial avenues.

Agenus prepares a crucial update on its BOT+BAL immunotherapy, a potential breakthrough for cancers resistant to current treatments. What will they reveal?

Tanabe Pharma Canada's 'Letters to ALS' offers a raw, intimate look into the fight against the disease, strengthening community bonds through shared stories.