Gene Therapies

Spectral Medical shifts its FDA submission for a breakthrough sepsis device, a strategic move that could strengthen its case but delays a potential lifeline.

With soaring sales for its rare obesity drug IMCIVREE, Rhythm Pharmaceuticals faces a critical March FDA decision that could unlock a massive new market.

A new collaboration is betting on tiny natural particles to deliver a gene-editing cure for a devastating neurological disorder, bypassing a key medical hurdle.

With its strategic pivot to high-growth Med Tech paying off, all eyes are on AngioDynamics' CEO presentation at the upcoming Leerink healthcare conference.

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.

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.