Gene Therapies

A new RNA drug shows promise by reducing harmful belly fat while preserving muscle, but modest overall weight loss in early trials spooks investors.

Experic and Flurry Powders form a strategic alliance to accelerate complex therapies by combining advanced particle engineering with GMP manufacturing.

UK biotech Amphista to showcase its innovative Targeted Glue™ platform at AACR, revealing new data for AML, lung cancer, and mesothelioma therapies.

With a history of billion-dollar deals, Mark Altmeyer takes the helm at ReproNovo, aiming to commercialize its promising fertility and women's health pipeline.

An influential guidelines update expands options for a tough-to-treat bladder cancer, giving patients access to a novel gene therapy beyond its FDA label.

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.

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.