Gene Therapies

Strong glaucoma sales and disciplined spending helped Iridex narrow its quarterly loss, signaling resilience despite retina segment woes and global headwinds.

A top scientific honor for Dr. Mariano Garcia-Blanco validates a new approach to autoimmune disease, targeting a genetic trait in 50% of the population.

A $290M war chest fuels a new public biotech aiming to help millions of migraine patients who don't respond to current blockbuster therapies.

With a new CSO known for pushing rare disease drugs to market, Arbor Biotechnologies signals a strategic shift from platform discovery to clinical execution.

Quince Therapeutics' drug LAM-001 posts strong Phase 2a data, showing meaningful functional gains for patients with severe pulmonary hypertension.

A new partnership with AI firm Basepath Health will help Children's Mercy treat five times more children with complex and rare genetic diseases.

A new bispecific antibody, TRIV-573, enters clinical trials, targeting both skin barrier and inflammation to redefine atopic dermatitis therapy.

The biotech firm raises cash via a complex warrant deal, diluting shares to fund its promising but challenging RNAi therapy for deadly KRAS-driven cancers.

Edesa Biotech pushes its pipeline forward with a new vitiligo trial and promising ARDS data, but rising R&D costs highlight the high stakes of innovation.

NFL legend Brett Favre reports gains from an experimental Parkinson's drug, but what does the science say about this potential breakthrough treatment?

The biopharma posts record Q1 revenue and profit, driven by its flagship DefenCath and a key acquisition, while a promising pipeline signals future growth.

A new partnership aims to solve the massive underdiagnosis of a severe genetic disorder by offering free testing, a move with deep strategic implications.

A $25M payment from partner Chiesi catapults Protalix to a profitable quarter, funding a promising new gout therapy and a broader rare disease strategy.

A new alliance aims to develop first-in-class drugs for Alzheimer's and Parkinson's by targeting chronic brain inflammation, a key driver of disease.

With a novel drug for schizophrenia and deep financial backing, LB Pharmaceuticals is advancing a promising new therapy for complex mental illnesses.

With its first patient enrolled and key EU approvals, Inhibikase pushes its PAH drug IKT-001 forward, balancing clinical hope with financial realities.

As its oesophageal cancer vaccine enters trials, Infinitopes hires a top CSO with a track record of bringing breakthrough therapies to market.

Actuate Therapeutics gets FDA nod for its oral cancer drug elraglusib, shifting strategy to improve patient outcomes and tackle hard-to-treat tumors.

Anaveon reveals promising ASCO data for its cancer drug ANV600, yet puts the asset on the market to fund a bold new focus on autoimmune diseases.

An innovative RNA-editing therapy for hepatocellular carcinoma has received a major FDA designation, potentially accelerating its path to patients.