Gene Therapies

With a massive late-stage pipeline and deep pockets, the biotech aims to defeat elusive cancer targets, but soaring costs raise the stakes for pivotal trials.

A new oral drug shows remarkable success in healing stubborn herpes lesions in people with HIV, offering a much-needed breakthrough for a vulnerable population.

A nonprofit is investing millions in a novel program to fast-track academic breakthroughs for neurological diseases from the lab to patients.

New subgroup data from ITM's COMPETE trial could refine treatment for a tough-to-treat cancer and bolster the company's position in the radiopharma market.

With a key gene therapy approval imminent and major clinical data on deck, REGENXBIO's upcoming financial report will be a crucial test for the biotech.

A newly identified blood biomarker for the drug masitinib could revolutionize how doctors treat ALS, progressive MS, and Alzheimer's disease.

Dupixent's landmark FDA approval offers a new, surgery-sparing option for a debilitating sinus disease, but questions of access and cost remain.

Intellia Therapeutics prepares for a critical March, where it will outline its gene editing future to investors amid safety questions and market pressures.

With a massive Series A, Alveus Therapeutics is advancing novel drugs aiming for more durable and higher-quality weight loss than current blockbusters.

Larimar Therapeutics' nomlabofusp receives Breakthrough Therapy Designation, paving an accelerated path for a potential disease-modifying treatment.

New data on MCO-010 gene therapy identifies ideal patients for Retinitis Pigmentosa, a key step as the one-time injection advances toward FDA approval.

Swiss biotech AC Immune begins a human trial for an oral drug aiming to halt the chronic inflammation driving diseases like Alzheimer's and Parkinson's.

A pivotal trial for a rare muscle disease failed its main goal, but promising data in a specific patient group offers a new, complex path forward.

With no approved therapies for Hepatitis E, Atea Pharmaceuticals' new drug shows potent preclinical results, offering a potential lifeline for vulnerable patients.

Avacta's pre|CISION therapy shows faster, more potent, and more selective tumor targeting than Enhertu in an AI-driven study, paving the way for 2026 trials.

Beam Therapeutics is set to announce its 2025 financial results and a new liver-targeted therapy, a key moment for its base editing platform and investors.

A Korean firm says it can cure 34 diseases using cells from cloned embryos, walking a fine line between a medical breakthrough and a bioethical taboo.

A lucrative deal for a rare disease voucher positions Fortress Biotech for profitability, highlighting a successful strategy and a breakthrough for Menkes patients.

BioVie faces a pivotal moment, touting breakthrough drugs for Alzheimer's and cirrhosis while navigating clinical setbacks and financial pressures.

As cardiovascular disease affects nearly half of US adults, experts are urging a shift to existing, life-saving therapies that are widely ignored.