Gene Therapies

The National Bleeding Disorders Foundation taps celebrity power for a live event, aiming to spotlight underdiagnosed conditions and transform advocacy.

As its new obesity drug enters a key trial, Biohaven bets on a novel approach: reducing fat while preserving precious muscle mass. Can it redefine weight loss?

Belief BioMed's single-dose treatment for hemophilia B, BBM-H901, is now approved in Macao, offering a new horizon for patients beyond frequent injections.

New preclinical data reveals a novel gene therapy that not only kills tumor cells but also boosts immunity, offering hope against drug-resistant lung cancer.

SAB Biotherapeutics' public offering fuels late-stage trials for its promising T1D drug, SAB-142, aiming to change the treatment landscape.

After regaining Nasdaq compliance, Incannex Healthcare is leveraging its $75M war chest to advance novel treatments for sleep apnea and anxiety.

Galectin Therapeutics adds legendary neurosurgeon Dr. Henry Brem to its board, a strategic move to leverage his drug development expertise for its lead liver drug.

BioCardia's FDA milestone for its Helix catheter could unlock a new era of targeted heart therapies, but financial hurdles and clinical questions remain.

The biotech reports promising clinical data for its lead alpha-therapy program and secures funding into late 2027, navigating a high-cost, high-reward path.

As MediciNova execs meet investors at the ROTH Conference, all eyes are on pivotal updates for its ALS, MS, and NAFLD drug candidates.

New data on an oral Parkinson's drug and major strides in rare disease research signal Lundbeck's deep commitment to tackling complex neurological disorders.

As it advances its brain cancer therapies, NeOnc Technologies hires a public company finance expert to steer its next growth phase and ensure fiscal rigor.

A new iPSC-derived cell therapy from Unixell Biotech has gained FDA approval for clinical trials, offering a regenerative approach for millions with epilepsy.

After a strategic pivot and R&D setbacks, Korro Bio secures an $85M lifeline to advance its novel RNA-editing drug for rare metabolic diseases.

Data shows Beren Therapeutics' drug slows progression and boosts survival for infantile-onset NPC, a rare disorder with no approved US treatments.

One year post-approval, Neurotech's novel gene therapy implant is transforming patient lives and rewriting the playbook for biotech commercialization.

The specialty infusion provider is the first to earn a rigorous new distinction, signaling a major leap forward in patient safety and quality of care.

Ratio Therapeutics appoints Dr. Colin Hayward as CMO, a strategic move to advance its sarcoma trial and challenge leaders in the booming radiopharmaceutical market.

Positive Phase 3 results for BBP-418 offer a potential first-ever treatment for LGMD2I/R9, a progressive muscle disease with no current therapies.

A novel compound, IRX4204, shows promise in preventing triple-negative breast cancer in lab studies, offering new hope for a safe, effective preventative.