Gene Therapies

With a new focus on eye disease and fresh funding, Surrozen will present its novel Wnt-based pipeline to investors. What's at stake for the future of sight?

Everest Medicines bets big on MT1013, a novel dual-action drug poised to transform treatment for millions with chronic kidney disease complications.

iVeena promotes key execs as it prepares for clinical trials of a novel eye drop that could reshape pediatric myopia treatment worldwide.

A world-first trial for personalized mRNA vaccines offers new hope for children with aggressive brain tumors, a mission born from a father's own fight.

As GLP-1 drugs dominate, Veru Inc. advances a novel therapy to combat muscle loss, aiming for a new standard of 'quality' weight loss in older adults.

A new biotech partnership is accelerating development of a once-daily tablet for hypoparathyroidism, a rare disease currently requiring injections.

New 4-year real-world data shows Zevra's drug stabilizes the rare, fatal 'Childhood Alzheimer's' disease, marking a milestone for patients and families.

A new bipartisan law injects vital funds into neuromuscular disease research, a victory driven by years of relentless patient advocacy. Here's what it means.

Following promising early results, RESTEM's Restem-L cell therapy enters a pivotal Phase 2/3 trial, aiming to transform treatment for IIM patients.

As X4 Pharmaceuticals preps for a key investor summit, all eyes are on its pivotal trial for chronic neutropenia and the commercial fate of XOLREMDI®.

A next-gen immunotherapy enters trials, hoping its safer design can unlock the immune system and form a potent combo against advanced solid tumors.

With a major Phase 3 trial now in Europe, Crystalys Therapeutics aims to fill a critical gap in gout care with a drug already proven in Asian markets.

Incyte's Zynyz gets a key EU panel's nod, offering a breakthrough for advanced anal canal cancer patients after decades of limited treatment options.

A new dual-action tablet shows major promise for prostate cancer patients with BRCA mutations, poised to change the standard of care in Europe.

The market for drugs like Ozempic and Wegovy is set to explode, fueled by new oral pills, expanded uses for heart and liver disease, and fierce competition.

A German biotech's novel oral drug for herpes is moving to Phase 2 trials, offering new hope against a widespread and often resistant virus.

With promising brain scan data, a deep therapeutic pipeline, and a cash runway into 2027, Radiopharm Theranostics is gaining significant momentum.

A new drug targeting a genetic form of Parkinson's shows promise as its late-stage trial nears completion, offering hope for a precision treatment.

A new study reveals albumin, the most abundant protein in blood, is a powerful weapon against mucormycosis, offering new hope for treating this fatal infection.

A first-in-class oral therapy targeting cellular metabolism has entered patient trials, offering new hope for a disease-modifying treatment for eczema.