Gene Therapies

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.

A major clinical trial setback for a promising liver disease drug leaves patients waiting and puts Bausch Health's future strategy under scrutiny.

A new strategic partnership aims to dismantle commercialization hurdles, promising faster access to life-saving therapies for rare disease patients.

Swedish biotech innovator Hansa Biopharma wins a top award for its life-saving transplant tech, facing a pivotal year with FDA review on the horizon.

PulseSight's first-in-class gene therapy for dry AMD completes its Phase I trial, offering a novel approach to a leading cause of vision loss.

With a $150M+ capital raise and a top cancer immunologist on board, the T1D Fund is making a strategic play to shift from managing T1D to curing it.

BioAge Labs is developing an oral drug for diabetic macular edema, a breakthrough that could end the need for frequent, invasive eye injections for millions.

Ascletis Pharma unveils ASC37, a next-gen, once-monthly obesity drug claiming superior potency and convenience, challenging giants like Eli Lilly.

Corvus Pharmaceuticals is set to unveil pivotal early data for its novel eczema drug, soquelitinib, a moment analysts call a critical milestone.

Crosswalk Therapeutics secures a key grant to develop a therapy targeting the debilitating skeletal disease in Morquio A, a challenge current treatments can't solve.

Adial Pharmaceuticals files a key patent to protect its personalized Alcohol Use Disorder drug, AD04, until 2045, signaling a new era for addiction medicine.

Vericel posts strong 2025 results driven by its MACI cartilage therapy. The biotech leader is now investing in a new facility and a clinical trial for ankle repair.

Pimicotinib, a once-daily pill for tenosynovial giant cell tumor, gains FDA review, offering new hope for patients beyond invasive surgery.

With a new name and a laser focus on its lead hemophilia A drug, Metagenomi Therapeutics signals a major shift from platform tech to clinical powerhouse.

Promising new 96-week data shows Vir's therapy could redefine care for severe hepatitis delta, as its oncology pipeline and financial runway also advance.

Biomea Fusion launches a high-stakes 2026 plan, advancing a novel diabetes drug to restore insulin cells and an oral pill to rival obesity giants.

With booming revenue, a potential blockbuster on the horizon, and key partnerships, Ascendis Pharma lays out a bold roadmap for global dominance.

Promising Phase 2 data shows a new DNA immunotherapy for advanced melanoma, delivered via a needle-free jet injector, significantly improves patient outcomes.